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Carolina Aguilar: Brain-Computer Interfaces that Heal Neural Circuits

Jonathan D. Grinstein, PhD, North American Editor of Inside Precision Medicine, hosts a new series called Behind the Breakthroughs that features the people shaping the future of medicine. With each episode, Jonathan gives listeners access to his guests’ motivational tales and visions for this emerging, game-changing field.

Brain-computer interfaces have captured global attention in recent years, but most public discussion has focused on assistive technologies—systems designed to help patients control computers, prosthetics, or digital devices through thought alone. Carolina Aguilar, co-founder and CEO of INBRAIN Neuroelectronics, believes the larger opportunity lies elsewhere: using advanced neural interfaces not just to decode the brain but to treat disease directly. Built around graphene-based technology, INBRAIN is developing implantable systems capable of reading and writing neural signals with far greater precision than traditional metal-based devices.

Before founding INBRAIN, Aguilar spent 13 years at Medtronic, including a decade leading the company’s global neuromodulation business. During that time, she saw both the extraordinary potential of brain stimulation therapies and the limitations of incremental innovation inside large medical technology companies. Her background in neuroscience research, including early work studying the relationship between pesticides and Parkinson’s disease, shaped a long-term interest in circuit modulation and neurotherapeutics. That experience eventually converged with the emergence of graphene as a promising material for next-generation neural interfaces.

In this conversation, Aguilar discusses why INBRAIN chose to focus on therapeutic BCI applications rather than assistive computing, how graphene may enable higher-resolution neural decoding and stimulation, and why Parkinson’s disease became the company’s first major target. She also outlines INBRAIN’s broader vision for personalized neurotechnology, AI-driven therapies, and future applications ranging from epilepsy and memory restoration to bioelectronic treatments for cardiometabolic disease.

This interview has been edited for length and clarity.

 

IPM: You spent more than a decade at Medtronic before founding INBRAIN. What made you realize that incremental innovation was no longer enough, and why did graphene feel like the right breakthrough technology at the right time?

Aguilar: INBRAIN is a graphene-based brain-computer interface (BCI) company developing the most intelligent and adaptive interface between the neural system and AI to solve health for billions.

I started my career in consumer goods, but then I spent 13 years at Medtronic, where, for ten of those 13 years, I was leading the brain stimulation, or neuromodulation, business globally. I was always extremely impressed by what the company vision was and the number of disease areas and patients that we could help. However, I saw that every year we were innovating, but it was a bit incremental versus breakthrough. It’s normal for big companies to prioritize preserving shareholder value and revenue opportunities.

Investing in breakthroughs is harder; they usually just acquire those breakthroughs and then integrate them. And, of course, they revitalized their innovation. So I thought at that point that I could be one of those breakthrough innovators that could take a little bit more risk but then bring much bigger value into the field.

When the right opportunity presented itself to us, after the European Union put €1 billion into bringing graphene to market and after having gone through different speeds as pieces, we realized that it was the time to build INBRAIN and make that breakthrough a step instead of the incremental innovation of the past.

It’s interesting because I actually studied neuroscience at Virginia Tech, and at Virginia Tech, I had to define my thesis to study for my master’s degree. I picked up a combination of pesticides in the study. The study focused on the effects of pesticides and their combinations on brain chemistry in Parkinson’s disease. Right. So it was already quite oriented toward what we are solving today. And I got a grant award for the best vision on circuit modulation. So it was not about targeting the brain. It was about the circuit that was actually managing some of these dynamics of Parkinson’s disease.

At that early stage, I was already very attracted to the problem we are solving today, even though I did not know I would have a company dedicated to that field and to this innovation. However, it was always driving me in that direction. Medtronic was the company that turned that ambition into a real product and broad platform, which I also helped launch globally. So I guess it was always there. It was the seed that had been growing over time.

 

IPM: Medtronic exposed you to commercial, upstream product development, and engineering challenges. What did you learn that prepared you to build INBRAIN, and how did graphene change your engineering perspective?

Aguilar: It also evolves over time. So we start with a problem, such as pesticides in the development of Parkinson’s. Then I think I stepped into Medtronic as a solution to the problem that existed at that time. I was like, great. Now we have a problem. We have a solution. Let’s fix Parkinson’s disease.

At Medtronic, I was exposed to new experiences even while working in a commercial role, as I became a global director. I was exposed to the upstream. At Medtronic, there is a downstream process from which all employees receive their compensation. Then there’s the upstream piece, which is the product development and the planning of the next platforms. I was not in charge of that, but I was exposed to the process, the thinking, what is coming next, how we can make it happen, and where the constraints are. And that intrigued me a lot.

I discovered that I had a little bit of an engineering part inside of me that I never exploited. When the right opportunity came, I received a call from an investor friend of mine, who sometimes asked me to do some small due diligence. And he said, “Hey, there are some guys who have some graphene technology here that you might understand better than me. Do you want to come to the pitch?” I said, “Perfect.”

Then I saw that some of these constraints that we were dealing with in Medtronic in terms of miniaturization, higher-resolution interfaces, charge injection limits, and many of these engineering problems, partially, we could also solve by going graphene and going a different kind of electronics and a different kind of platform. When these consolidated and we created INBRAIN, I had to put a huge amount of effort into understanding semiconductors, microelectronics, mechanical configurations, and data architectures. So it’s been a huge learning journey for me.

 

IPM: INBRAIN appears to be building a platform strategy across multiple applications and devices. What are the product verticals and why does graphene offer capabilities that traditional neural interfaces cannot?

Aguilar: So it’s effectively a platform with three product verticals. When Morgan Stanley released the BCI industry report, it stated that the market was a $400 billion opportunity. We already knew that with one device, you could not capture all that immense market. So we were already working on a stepwise approach to the biggest opportunity. And this is what we have today on the table in the sense that we have a first product. The first product I can show you is a cortical interface. Starting with about 100 contacts, we can grow to a thousand. But in this case, it was not necessary.

It’s made of graphene. So it’s not about the number of contacts but the quality of what these contacts can decode. What is the precision of anatomy and the resolution of disease-related biomarkers that we can decode at high resolution in a way that that resolution is superior to a standard medical technology?

In some cases, standard platinum and iridium are used; in some cases, iridium oxide. We decided to create this first product for use for less than 30 days once it’s approved. It’s not yet approved, but we are getting closer, and it is the one that we actually took first-in-human. The results of those three reports are being generated using this device to demonstrate both the safety of graphene and its decoding superiority compared to metal technology. And, of course, within that first-in-human study, we also decoded speech at the phoneme level.

It was about creating the first step toward making graphene and consolidating advanced materials in clinical practice. So from there, the second product is actually an implantable platform that uses some of that configuration. So we use a cortical and subcortical interface to actually decode a circuit.

In our case, the microcircuit that is involved in Parkinson’s disease. And that is the second one. And the third product is actually the same platform, where instead of these two cortical and subcortical interfaces, we put in the central nervous system. We are truly connected to a vagus nerve interface that decodes the fibers within the vagus nerve, which go into the different modulations of the different organs of the body.

We go from reading the brain to reading the body. This sequence of product verticals opens up the immense possibility of a $400 billion opportunity. But even more important is the number of patients we could actually touch and improve therapeutically with such a platform.

 

IPM: Many BCI companies focus on assistive applications like computer and prosthetic control. INBRAIN appears to be therapeutic. Why did you choose direct disease treatment, and what makes it harder?

Aguilar: People say “invasive” or “noninvasive.” I call them implantable and non-implantable. And within the implantable, there might be different levels of invasiveness, but we were looking at the problem to solve, and especially coming from the field we come from and having experience for ten years, the use cases we saw are assistive BCI, meaning I transfer thought to action in the computer as solving a very important problem, but actually in a small population because, at the end, paraplegia and ALS. They are very important disease areas, but compared to other areas, there are smaller populations.

There were many people and companies, great companies, already aiming to solve that. So Neuralink and Synchron and Paradromics were always positioned there. And we thought, well, we have a similar approach, but maybe with a material that is much better suited to actually read and write bidirectionally, therapeutically. In assistive BCI, you do a ton of decoding. So, you read it and then transfer it to the computer. On the therapeutic side, where we are doing it, you have to read, write, and do that computing already within the implanted system.

It’s a much more complex architecture and a harder problem to solve. Now the benefit is solving the disease for as long as the system is active. And Parkinson’s was only the beginning. We are currently examining the validation of this program. We’re looking at memory restoration. We are looking at a set of disease areas on the cardiometabolic side that I cannot disclose without an NDA that we are developing with Merck KGaA. Those deals were unexplored or suboptimally explored by some of the low-resolution companies in the field.

The post Carolina Aguilar: Brain-Computer Interfaces that Heal Neural Circuits appeared first on Inside Precision Medicine.

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STAT+: European Union inks a draft deal to boost local production of medicines and avoid shortages

The European Union reached a provisional deal to strengthen the supply of essential medicines — such as antibiotics, insulin, vaccines, and painkillers — and avoid shortages by ‌boosting domestic production and reducing reliance on imports.

Known as the Critical Medicines Act, the initiative seeks to avoid the kinds of withering drug shortages that were seen during the Covid-19 pandemic. Specifically, the proposal targets supply-chain weaknesses for more than 200 medicines that are considered critical ​to the region’s health security, including treatments for rare diseases.

“With today’s agreement, we are taking practical action to reduce our vulnerabilities, diversify supply chains and strengthen Europe’s capacity to produce critical medicines and their ingredients closer to home,” said Neophytos Charalambides, the health minister for Cyprus, in a statement.

Continue to STAT+ to read the full story…

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Gene Therapy Briefs: Regeneron Wins FDA Approval for First Neurosensory Gene Therapy

The FDA has granted accelerated approval to Regeneron Pharmaceuticals’ Otarmeni™ (lunsotogene parvec-cwha) as the first gene therapy designed to restore a neurosensory function to normal levels.

Otarmeni is an adeno-associated virus vector-based gene therapy indicated for treating children and adults with severe-to-profound and profound sensorineural hearing loss, defined as any frequency >90 decibel hearing level [dB HL], associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear.

Otarmeni (formerly DB-OTO) is the first and only in vivo gene therapy indicated for OTOF-related hearing loss. Regeneron said it will make Otarmeni available for free in the U.S.

The FDA based its accelerated approval decision on the improvement of hearing sensitivity as measured by average pure tone audiometry (PTA) at week 24 during the Phase I/II CHORD trial (NCT05788536). Twenty participants ages 10 months to 16 years received a single dose of Otarmeni via intracochlear infusion—10 patients in one ear, the other 10 in both ears. Data from CHORD showed:

  • 80% of participants (16 of 20) reported hearing improvements per pure tone audiometry assessments at ≤70 dB HL at 24 weeks, achieving the trial’s primary endpoint, while one additional participant achieved the threshold by week 48.
  • 70% (14 of 20) showed an auditory brainstem response (ABR) at ≤90 decibels at 24 weeks, achieving the trial’s key secondary endpoint.
  • Among participants followed to 48 weeks, all prior responders maintained a response to therapy, and 42% of all participants (five of 12) achieved normal hearing that included whispers (≤25 dB HL).

“This unprecedented breakthrough in gene therapy has already proven to be life-changing for many of the children in our clinical trial and their families,” said George D. Yancopoulos, MD, PhD, board co-chair, president and chief scientific officer of Regeneron. 1

The FDA said its accelerated approval may hinge upon verification and description of clinical benefit in the confirmatory portion of the CHOIRD trial, a first-in-human, multicenter, open-label trial designed to assess the safety, tolerability and preliminary efficacy of DB-OTO in infants, children, and adolescents with otoferlin variants.

Otarmeni is the first gene therapy, and second new molecular entity, to win FDA approval under the agency’s Commissioner’s National Priority Voucher (CNPV) pilot program.

Launched in October by FDA Commissioner Martin A. Makary, MD, CNPV awards vouchers to drug developers whose work is deemed to address a health crisis in the U.S., deliver more innovative cures, address unmet public health needs, and increase domestic drug manufacturing as a national security issue. The vouchers entitle companies to reviews of their final applications within a target timeframe of 1–2 months rather than the standard 10–12 months.

Intellia’s Lonvo-Z begins rolling BLA following positive Phase III data

Intellia Therapeutics has launched a rolling Biologics License Application (BLA) submission to the FDA seeking regulatory approval of lonvoguran ziclumeran (lonvo-z), after announcing positive topline results from the global Phase III HAELO trial (NCT06634420) in hereditary angioedema (HAE)—the first Phase III data reported for an in vivo gene editing therapy.

HAELO is a randomized, double-blind, placebo-controlled Phase III trial designed to evaluate the efficacy and safety of a one-time 50 mg dose of lonvo-z in adults and adolescents aged 16 years and older with Type I or Type II HAE. The trial’s key endpoints focused on the number of HAE attacks experienced by patients, quality of life, safety and tolerability. Eighty patients were enrolled with 52 receiving lonvo-z and 28, placebo.

HAELO met its primary endpoint. For the six-month efficacy evaluation period (weeks 5 to 28), a one-time infusion of lonvo-z reduced attacks by 87% vs. placebo, with a mean monthly attack rate of 0.26 in the lonvo-z arm vs. 2.10 in the placebo arm.

Other key findings from HAELO:

  • The trial met all key secondary endpoints with statistical significance (p<0.0001). These included a 62% rate of patients who were entirely attack free and therapy free in the lonvo-z arm for the six-month efficacy evaluation period, vs. 11% of patients in the placebo arm.
  • Lonvo-z showed favorable safety and tolerability data. The most common treatment emergent adverse events (TEAEs) during the primary observation period (infusion through week 28) were infusion-related reactions, headache and fatigue. All TEAEs reported as of the data cutoff (February 10, 2026) were mild or moderate, with no serious adverse events observed in the lonvo-z arm.
  • As of the data cutoff, all patients who received lonvo-z at baseline or in crossover after week 28 remained long-term prophylaxis (LTP) free.

“Today’s HAELO results represent a profound milestone for Intellia, the broader CRISPR and precision medicine fields and, most importantly, the HAE community,” said John Leonard, MD, Intellia’s president and CEO. “These data affirm lonvo-z’s potential, with one dose, to offer prolonged freedom from both attacks and the need for ongoing therapy.” 2

Intellia said researchers plan to present additional clinical data from HAELO at the 2026 European Academy of Allergy and Clinical Immunology Congress (EAACI), set for June 12-15 in Istanbul, Turkey (Abstract #100217).

Lonvo-z is designed to inactivate the kallikrein B1 (KLKB1) gene in order to permanently lower kallikrein and bradykinin levels. Lonvo-z is designed as a one-time treatment that is administered in an outpatient setting.

Intellia said it is preparing for a potential U.S. launch of lonvo-z in the first half of 2027.

J. Craig Venter Dies: Pioneer in gene discovery, genomics, and synthetic biology

Craig Venter, PhD, a pioneer in gene discovery, human genomics, and synthetic biology, died April 29 in San Diego after a brief hospitalization for unexpected side effects that arose from treatment of recently diagnosed cancer. He was 79.

Venter was founder, board chair, and CEO of the institute that bears his name in La Jolla, CA. Earlier at the National Institutes of Health (NIH), he helped pioneer gene discovery using expressed sequence tags (ESTs), enabling rapid identification of large numbers of human genes and accelerating genome mapping efforts.

He went on to lead efforts that produced the first draft sequences of the human genome. He and colleagues later published the first high-quality diploid human genome, a scientific milestone that demonstrated the importance of capturing genetic variation inherited from both parents.

Venter’s work helped define and advance modern genomics, as well as launched the field of synthetic biology, where he and his teams constructed the first self-replicating bacterial cell controlled by a chemically synthesized genome—proof that genomes could be designed digitally, built from chemical components, and like a computer, booted up to run a living cell.

Through the Sorcerer II Global Ocean Sampling Expedition, Venter and his teams used metagenomics to reveal microbial diversity, reporting the discovery of millions of new genes and expanding the known universe of protein families—work that added to knowledge and insight of the ocean microbiome and its role in planetary systems.

“Venter was controversial and often challenged the scientific orthodoxy, with critics accusing him of hype and going overboard on privatization,” said John Sterling, Editor in Chief of Genetic Engineering & Biotechnology News, who has known and worked editorially with Venter over the past 35 years. “To many, he was a visionary focusing on technological acceleration and blending academic science with the zeal of an entrepreneur. Supporters saw him as a pioneer who sped up genomics by years.” 3

In addition to founding the J. Craig Venter Institute (JCVI), Venter was a serial entrepreneur who co-founded Synthetic Genomics, Human Longevity, and most recently Diploid Genomics, advancing efforts designed to translate genomics and synthetic biology into tools for health and society.

He was also a fierce advocate for robust federal science funding, as well as for partnerships that accelerate progress across government, academia, and industry.

“Craig believed that science moves forward when people are willing to think differently, move decisively, and build what doesn’t yet exist,” said Anders Dale, president of JCVI. “His leadership and vision reshaped genomics and helped ignite synthetic biology. We will honor his legacy by continuing the mission he built—advancing genomic science, championing the public investments that make discovery possible, and partnering broadly to turn knowledge into impact.” 4

Rocket Pharma selling Priority Review Voucher for Kresladi™ for $180M

Rocket Pharmaceuticals has agreed to sell for $180 million the Rare Pediatric Disease Priority Review Voucher (PRV) it was awarded by the FDA after the agency granted accelerated approval of Kresladi™ (marnetegragene autotemcel).

Kresladi is an autologous hematopoietic stem cell-based gene therapy indicated to treat children with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.

The indication was approved in March under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression. The accelerated approval of Kresladi is subject to confirmation of its clinical benefit, to be based on an evaluation of longer-term follow-up data of treated patients in the ongoing Phase I/II trial (NCT03812263) and through a post-marketing registry. The study generated positive topline data showing 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all nine LAD-I patients with 18 to 42 months of available follow-up.

The accelerated approval followed a resubmission of Rocket’s Biologics License Application for Kresladi. The original submission was rejected by the FDA in 2024 through a Complete Response Letter that requested additional Chemistry, Manufacturing, and Controls (CMC) information, but did not raise safety or efficacy issues about the gene therapy.

Rocket said it plans to use proceeds from the PRV sale toward advancing its prioritized cardiovascular gene therapy pipeline, including clinical-stage programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM).

“The monetization of our PRV, following the FDA approval of Kresladi, provides meaningful non-dilutive capital and extends our cash runway into the second quarter of 2028,” said Gaurav Shah, MD, Rocket Pharmaceuticals CEO. “This strengthens our ability to advance key clinical milestones across our cardiovascular gene therapy pipeline, with all programs on track.” 5

Passage Bio cuts staff 75%, launches strategic review

Passage Bio said it will eliminate 75% of its staff in a cost-cutting restructuring that is part of the company’s effort to review strategic alternatives.

“The Company expects that the aggregate severance and exit costs for the Restructuring Plan will be approximately $3.3 million, which will be recorded primarily in the second quarter of 2026,” Passage Bio said in a regulatory filing. 6

Passage Bio has said it plans to review strategic alternatives that may include merger or acquisition transactions, a reverse merger, a sale of assets of the company, strategic partnerships, licensing opportunities, or other potential paths.

The restructuring followed Passage Bio receiving feedback during a Type C meeting with officials at the FDA that indicated that the company will be required to complete a randomized controlled registrational trial evaluating its lead pipeline candidate PBFT02 as a treatment for frontotemporal dementia (FTD) with granulin (GRN) mutations.

PBFT02 is a gene replacement therapy that uses an adeno-associated virus serotype 1 (AAV1) viral vector to deliver, through intra cisterna magna (ICM) administration, a functional GRN gene that encodes the progranulin protein (PGRN).

The 75% workforce cut amounts to approximately 18 people, based on the 24 full-time employees it reported as of December 31, 2025, according to its annual report.

LEO Pharma acquires Replay for $50M upfront, milestones

LEO Pharma has agreed to acquire Replay, a developer of gene therapies for rare genetic dermatological conditions, in a deal that the buyer said will add deep expertise and a next-generation gene therapy platform to its pipeline, namely Replay’s high‑payload herpes simplex virus (HSV) delivery vector.

LEO Pharma plans to acquire Replay for $50 million upfront, plus milestone payments and tiered single-digit royalties.

Replay’s gene therapy platform is designed to leverage HSV’s capacity to deliver large genes, which according to LEO makes it well suited for addressing rare, genetically driven dermatological conditions. The genetically modified HSV therapy is formulated as a topical gel that targets the deficient gene when applied directly to the skin.

“Replay’s HSV gene therapy platform holds significant promise for patients with rare genetic skin diseases, and realizing its full potential requires focused expertise in medical dermatology—an area where LEO Pharma brings decades of leadership, scale and proven execution,” LEO Pharma CEO Christophe Bourdon said. “The acquisition aligns with our strategy of investing in the most impactful opportunities in dermatology and positions LEO Pharma at the forefront of next‑generation gene therapy.” 7

LEO Pharma agreed to acquire Replay after identifying Replay as a high‑potential opportunity using its artificial intelligence (AI) scouting platform, Innoviewer™. Replay’s lead pipeline drug program is a preclinical phase candidate designed to treat dystrophic epidermolysis bullosa (DEB).

MeiraGTx buys rights to XLRP treatment from J&J for $25M upfront

MeiraGTx Holdings has agreed to acquire from Johnson & Johnson (J&J) all interests in botaretigene sparoparvovec (bota-vec), a gene therapy being developed to treat X-linked retinitis pigmentosa (XLRP).

Under the companies’ asset purchase agreement, MeiraGTx agreed to pay J&J $25 million cash upfront, a one-time regulatory and commercial milestone payment tied to U.S. approval and U.S. sales performance of bota-vec for the treatment of XLRP, plus what MeiraGTx called a high double-digit royalty on global net sales starting in mid-2029.

The sale comes nearly a year after bota-vec failed the 95-patient, Phase III LUMEOS trial (NCT04671433)  by missing the study’s primary endpoint of demonstrating statistically significant vision-guided mobility in patients with XLRP, as measured by a Visual Mobility Assessment (VMA) or maze.

However, MeiraGTx has emphasized results showing that subjects treated with bota-vec were 2.4x more likely to respond than untreated subjects. A Low Luminance Questionnaire – Patient-Reported Outcome (LLQ PRO) showed significant benefit in mobility and dim light function, qualities tested by the VMA—thus indication, according to MeiraGTx, that the maze was not sensitive enough to capture these benefits.

The company characterized data from the LUMEOS trial’s secondary endpoints as very strong, with clinically meaningful and statistically significant improvements shown in each of three domains of vision.

MeiraGTx is the commercial manufacturer of bota-vec and had collaborated in its development with J&J from Phase I development onward. The FDA has granted Fast Track and Orphan Drug Designations to bota-vec, while the European Medicines Agency has granted Priority Medicines (PRIME), Advanced Therapy Medicinal Product (ATMP), and Orphan Drug designations to bota-vec.

“This is a unique opportunity to gain an asset at this stage in development with data supporting a meaningful benefit in patients with no alternative treatment, many of whom are waiting for this life changing therapy and hoping for expeditious approval,” said Alexandria Forbes, PhD, MeiraGTX’s president and CEO. 8

She added that MeiraGTx intends to start filing a Biologics License Agreement (BLA) with the FDA and applications for regulatory approval in the European Union and Japan as soon as possible.

 

References

1. Regeneron Pharmaceuticals. Otarmeni™ (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S. April 23, 2026. (Last accessed May 1, 2026)

2. Intellia Therapeutics. Intellia Therapeutics Reports Positive Phase 3 Results in Hereditary Angioedema, Marking a Global First for In Vivo Gene Editing. April 27, 2026. (Last accessed May 1, 2026).

3. Genetic Engineering & Biotechnology News. Genomics Pioneer and Life Sciences Entrepreneur J. Craig Venter Dies at 79. April 30, 2026. (Last accessed May 1, 2026)

4. Craig Venter Institute. J. Craig Venter, genomics pioneer and founder of JCVI and Diploid Genomics, Inc., dies at 79. April 29, 2026. (Last accessed May 1, 2026)

5. Rocket Pharmaceuticals. Rocket Pharmaceuticals Announces $180 Million Sale of Priority Review Voucher. April 28, 2026. (Last accessed May 3, 2026)

6. Passage Bio. Form 8-K, filed April 28, 2026. (Last accessed May 5,2026)

7. LEO Pharma. LEO Pharma bolsters rare skin disease focus through acquisition of Replay gene therapy platform. April 30, 2026. (Last accessed May 3, 2026)

8. MeiraGTx Holdings. MeiraGTx Announces the Acquisition of Botaretigene Sparoparvovec (bota-vec) for the Treatment of X-linked Retinitis Pigmentosa (XLRP). April 16, 2026. (Last accessed May 3, 2026)

The post Gene Therapy Briefs: Regeneron Wins FDA Approval for First Neurosensory Gene Therapy appeared first on GEN – Genetic Engineering and Biotechnology News.

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Top 10 Best Selling Gene Therapies

The groundbreaking partnership that successfully treated a rare metabolic disorder in KJ Muldoon, or “Baby KJ,” with personalized CRISPR therapy last year has led therapy developers, researchers, and regulators, including the FDA, to craft a pathway for expanding the universe of gene therapies to advance the development of N-of-1 gene-editing therapies.

In February, the FDA unveiled its Plausible Mechanism Pathway draft guidance, a series of initiatives designed to increase regulatory flexibility and spur the development of bespoke gene-editing therapies for rare and ultra-rare disorders, which collectively total about 30 million individuals in the United States.

“The Agency anticipates that substantial evidence of effectiveness for individualized therapies could be established based on a single adequate and well-controlled clinical investigation with confirmatory evidence,” the draft guidance stated.

Last June, at a historic roundtable of cell and gene therapy researchers and clinicians hosted by the FDA, base editing pioneer David Liu, PhD, of Harvard University and the Broad Institute of MIT and Harvard, stated: “With sufficient organization and federal support and partnership with the FDA, I believe it will be possible by 2030 to treat at least 1,000 patients with personalized genetic treatments.”

Meanwhile, conventional gene therapy development continued in 2025. Last year saw four U.S. gene therapy approvals, bringing the number of FDA-approved gene and cell therapies up to 26, according to the American Society of Gene and Cell Therapies (ASGCT)—more than half of the 40 tallied by the organization as being approved worldwide.

Of those 26, 18 were gene therapies, of which 10 had disclosed sales high enough to be included on this A-List, which ranks top-selling gene therapies based on sales and net product revenue figures furnished by the companies in regulatory filings, annual reports, and/or press releases. Each gene therapy is listed with its sponsor(s), type, indication, and initial FDA approval date.

Not included are gene therapies with sales below the top 10, a category that includes two gene therapies approved in 2025: Precigen’s Papzimeos™ (zopapogene imadenovec-drba), which generated $3.4 million in net product revenue last year after becoming the first-and-only FDA-approved treatment for adults with recurrent respiratory papillomatosis (RRP) in August; and Abeona Therapeutics’ Zevaskyn® (prademagene zamikeracel), an autologous cell sheet-based gene therapy approved to treat wounds in adults and children with recessive dystrophic epidermolysis bullosa (RDEB).

Three gene therapies did not have disclosed sales in 2025, including:

  • Encelto™ (revakinagene taroretcel-lwey), an allogeneic encapsulated cell-based gene therapy marketed by Neurotech Pharmaceuticals and indicated for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel).
  • Imlygic® (talimogene laherparepvec), a genetically modified oncolytic viral therapy marketed by BioVex (Amgen) and indicated for local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery.
  • Waskyra™ (etuvetidigene autotemcel), a cell-based gene therapy and the first FDA-approved treatment for Wiskott-Aldrich syndrome (WAS). Developer Fondazione Telethon is the first non-profit organization to have successfully led full development of an ex vivo gene therapy from lab research (at Milan’s San Raffaele Telethon Institute for Gene Therapy or SR-Tiget) to regulatory approval.

Also not included this year are sales of three gene therapies that had been marketed by Bluebird Bio: Beta thalassemia treatment Zynteglo™ (betibeglogene autotemcel), sickle cell disease treatment Lyfgenia® (lovotibeglogene autotemcel), and cerebral adrenoleukodystrophy (CALD) treatment Skysona® (elivaldogene autotemcel).

Last year, Bluebird Bio went private after being acquired by funds managed by Carlyle and SK Capital Partners, then rebranded in September as Genetix Biotherapeutics. Genetix does not disclose sales but did announce on March 2 that more than 100 patients received infusions of the three gene therapies during 2025.

Also last year, Pfizer halted development and commercialization of Beqvez™ (fidanacogene elaparvovec-dzkt), which had been co-marketed with Roche-owned Spark Therapeutics, after it generated no sales in 2024. Last August, Pfizer terminated its license agreement with Spark for Beqvez, an adeno-associated virus (AAV) vector-based gene therapy indicated for forms of moderate to severe hemophilia B in adults.

 

 

 

Top 10 Best Selling Gene Therapies
 

1. Zolgensma®  (onasemnogene abeparvovec-xioi)

2025 Sales: $1.232 billion 1

Sponsor(s): Novartis2

Type: AAV vector-based gene therapy

Indication(s): Treatment of pediatric patients less than two years of age with spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene.

Initial FDA Approval Date: May 24, 2019
 

2. Elevidys®  (delandistrogene moxeparvovec-rokl)

2025 Sales: $898.7 million

Sponsor(s): Sarepta Therapeutics

Type: AAV vector-based gene therapy

Indication(s): Treatment of ambulatory pediatric patients aged four through five years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.3

Initial FDA Approval Date: June 22, 2023 (Accelerated Approval)
 

3. Vyjuvek®  (beremagene geperpavec-svdt)

2025 Sales: $389.13 million

Sponsor(s): Krystal Biotech

Type: Herpes-simplex virus type 1 (HSV-1) vector-based gene therapy

Indication(s): Treatment of wounds in patients six months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

Initial FDA Approval Date: May 19, 2023
 

4. Adstiladrin®  (nadofaragene firadenovec-vncg)

2025 Sales: €172.673 million ($199.329 million)

Sponsor(s): Ferring Pharmaceuticals

Type: Non-replicating adenoviral vector-based gene therapy

Indication(s): Treatment of adults with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

Initial FDA Approval Date: December 16, 2022
 

5. Casgevy®  (exagamglogene autotemcel; “exa-cel”)

2025 Sales: $115.8 million

Sponsor(s): Vertex Pharmaceuticals and CRISPR Therapeutics

Type: Autologous genome-edited hematopoietic stem cell-based gene therapy

Indication(s): Treatment of patients aged 12 years and older with sickle cell disease with recurrent vaso-occlusive crises (VOCs), or transfusion-dependent β-thalassemia (TDT).

Initial FDA Approval Date: December 8, 2023
 

6. Hemgenix®  (etranacogene dezaparvovec-drlb)

2025 Sales: A$92 million ($64.9 million)4

Sponsor(s): CSL Behring

Type: AAV vector-based gene therapy

Indication(s): Treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Initial FDA Approval Date: November 22, 2022
 

7. Kebilidi™ / Upstaza®  (eladocagene exuparvovec-tneq)5

2025 Sales: $56.626 million

Sponsor(s): PTC Therapeutics

Type: AAV vector-based gene therapy

Indication(s): Treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.

Initial FDA Approval Date: November 13, 2024
 

8. Luxturna®  (voretigene neparvovec-rzyl)

2025 Sales: CHF 41 million ($51.8 million)

Sponsor(s): Spark Therapeutics (Roche)

Type: Adeno-associated virus vector-based gene therapy

Indication(s): Treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).

Initial FDA Approval Date: December 18, 2017
 

9. Lenmeldy™ / Libmeldy™  (atidarsagene autotemcel)6

2025 Sales: ¥6.4 billion ($40.2 million)

Sponsor(s): Orchard Therapeutics (a wholly owned subsidiary of Kyowa Kirin)

Type: Autologous hematopoietic stem cell-based gene therapy

Indication(s): Treatment for children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).

Initial FDA Approval Date: March 18, 2024
 

10. Roctavian®  (valoctocogene roxaparvovec-rvox; “val-rox”)

2025 Sales: $36 million

Sponsor(s): BioMarin Pharmaceutical

Type: AAV vector-based gene therapy

Indication(s): Treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to AAV serotype 5 detected by an FDA-approved test.

Initial FDA Approval Date: June 30, 2023
References

  1. Includes sales of ITVISMA® (onasemnogene abeparvovec-brve), approved by the FDA in November 2025 to treat SMA in adult and pediatric patients two years of age and older with a confirmed mutation in the SMN1 gene. ITVISMA has the same active ingredient as Zolgensma but is administered via a single intrathecal injection, while Zolgensma is administered intravenously.
  2. Novartis is the successor to AveXis, which successfully completed the development of Zolgensma in 2019 by receiving FDA approval for the therapy. In 2014, AveXis licensed from REGENXBIO the AAV9 vector used in the Phase I SMA clinical trial at Nationwide Children’s Hospital. REGENXBIO licensed exclusive rights to key intellectual property covering novel recombinant AAV vectors discovered at the University of Pennsylvania in the lab of James M. Wilson, MD, PhD.
  3. Following the deaths of two DMD patients receiving Elevidys last year, Sarepta halted shipments of Elevidys for non-ambulatory patients and paused the Phase III ENVISION trial (NCT05881408). The study remained paused at deadline. Following a third death, that of an eight-year-old Brazilian boy, the FDA demanded Sarepta pause shipments of Elevidys to ambulant patients. Sarepta initially refused before agreeing in July 2025. A few days later, after an FDA reversal, Sarepta resumed Elevidys shipments to ambulant patients, after Brazilian authorities ruled out treatment with the gene therapy as a factor in the boy’s death.
  4. Sales figure is for the fiscal year ending June 30, 2025. CSL Behring has since disclosed sales of $57 million ($40.2 million) for July–December 2025 but has only furnished a comparison to the year-ago period in terms of constant currency without disclosing a specific sales figure.
  5. Eladocagene exuparvovec-tneq is marketed as Kebilidi in the U.S. and as Upstaza outside the U.S.
  6. Atidarsagene autotemcel is marketed as Lenmeldy in the U.S. and as Libmeldy within the European Union.

 

 

 

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The Download: storing nuclear waste and orchestrating agents

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology.

It’s time to make a plan for nuclear waste

Today, nuclear energy enjoys rare support across the political spectrum. Public approval has spiked, and Big Tech is throwing money around to meet rising electricity demand. That newfound interest is exactly why it’s time to talk about an old problem: nuclear waste.

In the US, nuclear reactors produce about 2,000 metric tons of high-level waste each year—and there’s nowhere to put it. Now, the need for a permanent storage solution is becoming urgent. Here’s what’s at stake.

—Casey Crownhart

This article is from The Spark, MIT Technology Review’s weekly climate newsletter. Sign up to receive it in your inbox every Wednesday.

Orchestrated agents are coming for white-collar work

When people say AI will transform industries, what they have in mind—whether they know it or not—are AI agents. ChatGPT showed AI can talk. But to change the world, it needs to do stuff.

The real power comes when agents work as teams, coordinating multiple roles to tackle complex tasks. Apps like Codex and Claude Cowork offer a glimpse of this shift, bringing multi-agent general-purpose productivity tools.

In theory, networks of AI agents could do to white-collar knowledge work what assembly lines did to manufacturing. That’s the vision. But as agents move into real-world systems, the risks grow too. Read the full story.

—Will Douglas Heaven

Agent Orchestration is one of the 10 Things That Matter in AI Right Now, MIT Technology Review’s guide to what’s really worth your attention in the busy, buzzy world of AI. We’re unpacking one item from the list each day here in The Download, so stay tuned.

MIT Technology Review Narrated: no one’s sure if synthetic mirror life will kill us all

In February 2019, a group of scientists proposed a high-risk, cutting-edge, irresistibly exciting idea that the National Science Foundation should fund: making “mirror” bacteria.

These lab-created microbes would be organized like ordinary bacteria, but their proteins and sugars would be mirror images of those found in nature. Researchers believed they could reveal new insights into building cells, designing drugs, and even the origins of life.

But now, many of them have reversed course. They’ve become convinced that mirror organisms could trigger a catastrophic event threatening every form of life on Earth. Find out why.

—Stephen Ornes

This is our latest story to be turned into an MIT Technology Review Narrated podcast, which we publish each week on Spotify and Apple Podcasts. Just navigate to MIT Technology Review Narrated on either platform, and follow us to get all our new content as it’s released.

The must-reads

I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.

1 Elon Musk says Sam Altman “stole a charity” at the OpenAI trial
Musk testified for the first time yesterday in the landmark legal showdown. (FT $)
+ He said OpenAI was founded as a non-profit to avoid a “Terminator outcome.” (Wired $)
+ And claimed he came up with the idea for the company. (Reuters $)
+ The trial could upend the global AI race. (MIT Technology Review)

2 The White House has plans to bypass Anthropic’s blacklisting
It’s drafting guidance to sidestep the supply-chain risk designation. (Axios)
+ The White House is also meeting other tech firms to discuss AI risks. (Politico)
+ The Pentagon’s culture war against Anthropic has backfired. (MIT Technology Review)

3 OpenAI is tightening ties with Amazon after retreating from Microsoft
AWS customers are getting extra access to OpenAI systems. (NBC News)
+ While OpenAI gets new users and cloud-computing capabilities. (CNBC)

4 AI bots told scientists how to create biological weapons
And unleash them in public spaces. (NYT $)
+ AI will change war forever. (MIT Technology Review)

5 China has suspended robotaxi licenses after a scary outage
Dozens of Baidu vehicles suddenly stopped last month. (The Verge)
+ Chinese robotaxi firms are planning global expansions. (Guardian)

6 Meta has been found in breach of EU rules on protecting children
After failing to block access to Facebook and Instagram. (Guardian)
+ Parents are forcing schools to roll back classroom tech use. (NYT $)

7 AI is spotting pancreatic cancer years before symptoms appear
 A study found it could catch the tumor early enough to treat. (Bloomberg)

8 The Iran war is disrupting data center rollouts
Oaktree-owned Pure DC is the latest firm to pause investments. (CNBC)

9 SpaceX is tying Elon Musk’s pay to Mars colonization goals
It’s set lofty goals for his jaw-dropping compensation. (Reuters $)

10 AI has reconstructed the face of an ancient Pompeii victim
 Technology is reshaping our understanding of the distant past (NPR)

Quote of the day

“Overnight, without you even knowing it, your own life chances, the life chances of your children, will be dependent on people continuing to prop up Musk’s visions of how the world should look.”

—Elon Musk biographer Michel Martin tells NPR how the Tesla tycoon is shaping our lives.

One More Thing

a person with luggage walks through and airport setting

NEIL WEBB


Inside Clear’s ambitions to manage your identity beyond the airport

If you’ve ever been through a large US airport, you’re probably aware of Clear, the identity verification service that uses biometric scans to whisk travelers past standard security checks.

Now Clear wants to expand that “face-first” experience from airports to just about everywhere, from retailers and banks to even your doctor’s office. Its CEO has designs on making Clear the “identity layer of the internet” and the “universal identity platform” of the physical world.

All you have to do is show up—and show your face. But as biometric identity systems go mainstream, concerns about privacy, security, and control are becoming harder to ignore. And the cost of convenience may not be shared equally. Discover what’s at stake. 


—Eileen Guo

We can still have nice things

A place for comfort, fun and distraction to brighten up your day. (Got any ideas? Drop me a line.)

+ Discover why the eight-hour night is a modern invention.
+ This artist creates masterpieces using only a vintage typewriter and a lot of patience.
+ Test your local knowledge with this game that drops you in a random Street View location.
+ Watch this incredible feat of precision piloting as a race aircraft touches down on a 120km/h cargo train.

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The Download: Musk and Altman’s legal showdown, and AI’s profit problem

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology.

Elon Musk and Sam Altman are going to court over OpenAI’s future

Elon Musk and OpenAI CEO Sam Altman head to trial this week in a case with sweeping consequences. Ahead of OpenAI’s IPO, the court could rule on whether the company can exist as a for-profit enterprise. It could even oust its leadership.

Musk, an OpenAI co-founder, claims he was deceived into bankrolling the firm under false pretenses. He’s seeking $134 billion in damages, the removal of Altman and president Greg Brockman, and the company’s restoration to a non-profit.

Find out how the trial could upend the global AI race.

—Michelle Kim

The missing step between hype and profit

In a celebrated South Park episode, a community of gnomes sneak out at night to steal underpants. Why? The gnomes present their pitch deck. “Phase 1: Collect underpants. Phase 2: ? Phase 3: Profit.” It’s a business plan that captures the current state of AI. 

Companies have built the tech (Step 1) and promised transformation (Step 3). But how they get there is still a big question mark. Read about the potential paths forward.


—Will Douglas Heaven

This story originally appeared in The Algorithm, our weekly newsletter giving you the inside track on all things AI. Sign up to receive it in your inbox every Monday.

Welcome to the era of weaponized deepfakes

For years, experts have warned that deepfakes could be deployed in malicious ways. These dangers are now here.

Cheap, accessible models now produce weaponized deepfakes—from sexually explicit images to political propaganda—that look startlingly real. They’re already inciting violence, changing minds, and sowing mistrust, with women and marginalized groups disproportionately affected.

Experts fear that they’re cratering trust and critical thinking. Here’s why they’re alarmed.

—Eileen Guo

Weaponized deepfakes are on our list of the 10 Things That Matter in AI Right Now, MIT Technology Review’s guide to what’s really worth your attention in the busy, buzzy world of AI. 

The must-reads

I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.

1 OpenAI has ended its exclusive partnership with Microsoft
The new deal allows OpenAI to court rivals such as Amazon. (Reuters $)
+ Microsoft will still license OpenAI’s tech, but no longer exclusively. (NYT $)
+ OpenAI is missing key growth targets ahead of its IPO. (WSJ $)

2 Google has signed a classified AI deal with the Pentagon
It permits AI use for “any lawful government purpose.” (The Information $)
+ Over 600 Google workers had called for a block on the deal. (QZ)
+ AI firms are set to train military versions of their models on classified data. (MIT Technology Review)

3 The EU has told Google to open Android to AI rivals
It wants to end Gemini’s built-in advantage. (Ars Technica)
+ Google calls the move an “unwarranted intervention.” (WSJ $)
+ A final decision is expected by the end of July. (Reuters $)

4 OpenAI is reportedly developing an AI-first smartphone
It would replace apps with agents. (TechCrunch)
+ Qualcomm and MediaTek may be developing its processors. (Gizmodo)

5 A brain implant for depression is moving into human testing
The FDA has approved a human study of the device. (Wired $)
+ BCIs have thus far struggled to reach the market. (MIT Technology Review)

6 A populist backlash against AI is gaining momentum in rural America
From Indiana to Idaho, voters are pushing back against the technology. (NYT $)
+ Anti-AI protests are expanding worldwide. (MIT Technology Review)

7 DeepSeek has priced its new model 97% below OpenAI’s GPT-5.5
It aims to attract more enterprises, developers, and agent-based users. (SCMP)
+ Here are three reasons why DeepSeek V4 matters. (MIT Technology Review)

8 AI now generates a third of new websites
A study found it’s making the web more cheery and less verbose. (404 Media)

9 Top talent is leaving Big Tech to launch their own AI startups
Meta, Google, and OpenAI are facing a brain drain. (CNBC)

10 Taylor Swift is trademarking her voice and image
The Grammy winner has been the target of numerous deepfakes. (NBC News)
+ A growing number of celebrities are fighting AI with trademarks. (BBC)

Quote of the day

“The reality is people don’t like him.”

—Judge Yvonne Gonzalez Rogers reacts to prospective jurors confessing their negative views of Elon Musk ahead of his legal battle with Sam Altman, The Verge reports.

One More Thing


How covid conspiracy theories led to an alarming resurgence in AIDS denialism

When Joe Rogan falsely declared that “party drugs” were an “important factor in AIDS,” several million people were listening. He also asserted that AZT, the earliest drug used to treat AIDS, killed people “quicker” than the disease itself—another claim that has been disproven.

Such comments illustrate an unmistakable resurgence in AIDS denialism: a false collection of theories arguing either that HIV does not cause AIDS or that there is no such thing as HIV at all. By the dawn of the millennium, these claims had largely fallen out of favour. That changed when the coronavirus arrived.

Follow the digital path from Covid skepticism to the return of a deadly conspiracy theory.


—Anna Merlan

We can still have nice things

A place for comfort, fun and distraction to brighten up your day. (Got any ideas? Drop me a line.)

+ Explore the planets from your laptop with this live sky map.
+ This marathon DJ set from Daphni is an incredible journey through electronic music.
+ NASA’s stunning Artemis II wallpapers bring a high-res piece of deep space to your phone.
+ This fascinating GPS explainer breaks down how your phone figures out exactly where you are.

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Sun Pharma Aims for Top 3 in Women’s Health with $11.75B Organon Purchase

Sun Pharmaceutical Industries has agreed to acquire Organon, the women’s health drug developer spun out of Merck & Co., for $11.75 billion in a deal intended to catapult the buyer into a top 25 global biopharma—top three in women’s health—by growing its innovative medicines business and expanding its product offerings into biosimilar drugs, the companies said today.

Headquartered in Jersey City, NJ, Organon was spun out of Merck in 2021 and has since then grown its portfolio to more than 70 women’s health and general medicines products, including biosimilars, that have been commercialized in the U.S. and some 140 countries worldwide. In addition to the U.S., Organon’s largest markets include Brazil, Canada, China, and the countries of the European Union. Organon said it has six manufacturing facilities across the EU and emerging markets.

Sun Pharma said the combined company created by the deal will have annual revenue of $12.4 billion, a figure the company said would propel it into a top 25 global pharma—though the company was ranked No. 14 in GEN’s most recent A-List of Top 25 Biotech Companies Heading Into 2026, compiled last December, based on its market capitalization (share price times the number of outstanding shares) of INR 4.31 trillion ($50.8 billion).

Sun Pharma said Organon’s portfolio was similar to its own, and that the acquisition of Organon was aligned with its strategies of growing its Innovative Medicines business (to a 27% revenue share) and expanding into biosimilars as a Top 10 global company.

The combined company, Sun Pharma and Organon said, would be top three in global women’s health, creating a commercial platform for future growth; the seventh largest global biosimilar player; and a presence in 150 countries worldwide, with 18 large markets that would each generate more than $100 million in revenues.

“This transaction represents a significant opportunity for Sun Pharma to build on its vision of Reaching People and Touching Lives,” Sun Pharma executive chairman Dilip Shanghvi said in a statement. “Organon’s portfolio, capabilities, and global reach are highly complementary to our own, and we believe that bringing the two organizations together can create a stronger and more diversified platform. We have deep respect for Organon’s mission and look forward to building on its legacy while driving sustainable long‑term growth.”

Deal speculation

The deal ends two weeks of speculation that began with an April 10 report in the Indian news outlet The Economic Times stating that Sun Pharma had submitted a $12 billion all-cash offer for Organon. On Friday, the news outlet followed up with a report stating that Sun Pharma had submitted a revised $13 billion offer.

Investors appeared to support the deal, as Sun Pharma shares on India’s National Stock Exchange rose about 7% to INR 1,733.50 ($18.41) at the close of trading today.

Sun Pharma has agreed to acquire 100% of Organon’s issued and outstanding shares for cash. Sun said it planned to fund the acquisition through a combination of available cash resources and committed financing from banks.

“Together, we will become a partner of choice for acquiring and launching new products,” stated Kirti Ganorkar, managing director of Sun Pharma. “Our immediate priorities will be business continuity, disciplined integration, and responsible value creation. We see strong potential in leveraging Organon’s talent pool. In addition, there is a scope for synergies including significant revenue upside opportunities to be realized over the coming years.”

Those synergies were later quantified by Sun Pharma as approximately $350 million within two to four years of the deal’s completion.

Sun Pharma did say, however, that the acquisition of Organon will strengthen its generation of cash, with its earnings before interest, taxes, depreciation, and amortization (EBITDA) and cash flow set to nearly double, supporting future efforts to reduce the net debt/EBITDA of 2.3x resulting from the deal.

Sun Pharma finished the first nine months of its fiscal year ending March 31, 2026, with a net profit of INR 87.654 billion ($931.5 million) and EBITDA of INR 137.772 billion ($1.464 billion; up 19.2% from the year-ago period), on sales of INR 436.604 billion ($4.64 billion), up 11.3% year over year.

During its fiscal year ending March 31, 2025, Sun Pharma reported adjusted net profit (excluding one-time items) of INR 119.844 billion ($1.274 billion), up 19% from a year earlier, on sales of INR 520.412 billion (about $5.53 billion). Reported net profit for FY 2025 was INR 109.290 billion ($1.161 billion), vs. Rs. 95.764 billion ($1.017 billion) during FY 2024.

Organon finished last year with adjusted EBITDA of $1.9 billion on revenue of $6.2 billion. The company reported debt of $8.64 billion—down from the $9.5 billion in debt it reported when it separated from Merck—and a cash balance of $574 million.

Planned sale

In November, Organon announced plans to sell its JADA® System, designed to control and treat abnormal postpartum uterine bleeding or hemorrhage, to Laborie Medical Technologies for up to $465 million—$440 million to be paid at closing, subject to adjustments, and up to $25 million tied to achieving 2026 revenue targets. Net proceeds from the divestiture will contribute to Organon’s cash balance as of March 31, 2026.

Organon will merge with a subsidiary of Sun Pharma, with Organon surviving the merger. The transaction is expected to close in early 2027 subject to customary conditions, including regulatory approvals and Organon stockholder approval.

The boards of both Sun Pharma and Organon have approved the deal.

“Following a comprehensive review of strategic alternatives, our Board determined that this all‑cash transaction offers compelling and immediate value to Organon stockholders,” stated Carrie Cox, executive chair of Organon. “We believe Sun Pharma is well positioned to support Organon’s businesses, employees, and patients globally, and to further advance our commitment to delivering impactful medicines and solutions.”

The post Sun Pharma Aims for Top 3 in Women’s Health with $11.75B Organon Purchase appeared first on GEN – Genetic Engineering and Biotechnology News.

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The Download: murderous ‘mirror’ bacteria, and Chinese workers fighting AI doubles

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology.

No one’s sure if synthetic mirror life will kill us all

In February 2019, a group of scientists proposed a high-risk, cutting-edge, irresistibly exciting idea that the National Science Foundation should fund: making “mirror” bacteria.

These lab-created microbes would be organized like ordinary bacteria, but their proteins and sugars would be mirror images of those found in nature. Researchers believed they could reveal new insights into building cells, designing drugs, and even the origins of life.

But now, many of them have reversed course. They’ve become convinced that mirror organisms could trigger a catastrophic event threatening every form of life on Earth. Find out why they’re ringing alarm bells.

—Stephen Ornes

This story is from the next issue of our print magazine, which is all about nature. Subscribe now to read it when it lands this Wednesday.

Chinese tech workers are starting to train their AI doubles—and pushing back

Earlier this month, a GitHub project called Colleague Skill struck a nerve by claiming to “distill” a worker’s skills and personality—and replicate them with an AI agent. Though the project was a spoof, it prompted a wave of soul-searching among otherwise enthusiastic early adopters.

A number of tech workers told MIT Technology Review that their bosses are already encouraging them to document their workflows for automation via tools like OpenClaw. Many now fear that they are being flattened into code and losing their professional identity.

In response, some are fighting back with tools designed to sabotage the automation process.

Read the full story.

—Caiwei Chen

The must-reads

I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.

1 The White House and Anthropic are working toward a compromise
The Trump administration says they had a “productive meeting.” (Reuters $)
+ Trump had ordered US agencies to phase out Anthropic’s tech. (Guardian)
+ Despite the blacklist, the NSA is using Anthropic’s new Mythos model. (Axios)

2 Palantir has unveiled a manifesto calling for universal national service
While denouncing inclusivity and “regressive” cultures. (TechCrunch)
+ It’s a summary of CEO Alex Karp’s book “The Technological Republic.” (Engadget)
+ One critic called the book “a piece of corporate sales material.“ (Bloomberg $)

3 Germany’s chancellor and largest company want looser AI rules
Chancellor Merz said industrial AI needs ‌more regulatory freedom. (Reuters $)
+ Siemens says it plans to shift investments to the US if EU rules don’t change. (Bloomberg $)
+ Fractures over AI regulation are also emerging in the US. (MIT Technology Review)  

4 Nvidia’s once-tight bond with gamers is cracking over AI  
Consumer graphics cards are no longer the priority. (CNBC)
+ But generative AI could reinvent what it means to play. (MIT Technology Review)

5 Insurers are trying to exclude AI-related harms from their coverage
And escape legal liability for AI’s mistakes. (FT $)
+ AI images are being used in insurance scams. (BBC)

6 AI is about to make the global e-waste crisis much worse
And most of the trash will end up in non-Western countries. (Rest of World)
+ Here’s what we can do about it. (MIT Technology Review)

7 Tinder and Zoom have partnered with Sam Altman’s eye-scanning firm
To offer a “proof of humanity” badge to users. (BBC)

8 Islamist insurgents in West Africa are driving surging demand for drones
A Nigerian UAV startup is opening its first factory abroad in Ghana. (Bloomberg $)

9 Hundreds of fake pro-Trump AI influencers are flooding social media
In an apparent bid to hook conservative voters. (NYT)

10 A Chinese humanoid has smashed the human half-marathon record
Despite crashing into a railing near the end of the race. (NBC News)
+ Chinese tech firm Honor swept the podium spots. (Engadget)
+ Last year, humans won the race by a mile. (CNN)

Quote of the day

“This is the only issue where you’ve got Steve Bannon and Ralph Nader, Glenn Beck and Bernie Sanders fighting for the same thing.”

—Ben Cumming, head of communications at the AI safety nonprofit Future of Life Institute, tells the Washington Post that diverse public figures are endorsing a declaration of AI policy priorities.

One More Thing

International Space Station photographed from space with Earth in the distance

NASA


The great commercial takeover of low Earth orbit

The International Space Station will be decommissioned as soon as 2030, but the story of America in low Earth orbit (LEO) will continue. 

Using lessons from the ISS, NASA has partnered with private companies to develop new commercial space stations for research, manufacturing, and tourism. If they are successful, these businesses will bring about a new era of space exploration: private rockets flying to private destinations.

They will also demonstrate a new model in which NASA builds infrastructure and the private sector takes it from there—freeing the agency to explore deeper and deeper into space. Read the full story.


—David W. Brown

We can still have nice things

A place for comfort, fun and distraction to brighten up your day. (Got any ideas? Drop me a line.)

+ Bask in thisadorable test of a dog’s devotion.
+ This vocal pitch trainer improves your singing straight from your browser.
+ Master international etiquette with this interactive guide to the world’s cultures.
+ Explore the networks of public figures with this intriguing interactive graph.