<![CDATA[Leading psychiatric organizations respond to federal push for deprescribing. ]]>
Here’s what you need to know about the cruise ship hantavirus outbreak
MIT Technology Review Explains: Let our writers untangle the complex, messy world of technology to help you understand what’s coming next. You can read more from the series here.
Eight passengers aboard a Dutch-flagged cruise ship have contracted a type of hantavirus, a rare virus transmitted by rats. Three of them have died. As the ship prepares to dock in the Canary Islands, plans are being finalized to let the remaining passengers and crew disembark safely.
The virus in question appears to have a high fatality rate. Read on for answers to the big questions surrounding the outbreak—and to hear why health experts don’t expect a rerun of the covid-19 pandemic.
What is hantavirus?
Hantaviruses are a group of viruses that typically infect rodents but can be transmitted to humans through exposure to the animals or their droppings, urine, or saliva. The viruses don’t seem to cause illness in rodents, but they can make people very unwell. The symptoms can depend on the type of hantavirus a person has been exposed to. Varieties found in the Americas can cause hantavirus cardiopulmonary syndrome, which affects the lungs and heart and has a fatality rate of up to 50%.
That condition made headlines last year when it caused the death of pianist Betsy Arakawa, the wife of actor Gene Hackman.
How many cases have there been so far?
On April 6, a man aboard the MV Hondius developed respiratory symptoms. He became very unwell and died just five days later. His wife, who left the ship at the island of Saint Helena, also developed symptoms. Her health deteriorated during a flight to Johannesburg, South Africa, and she died the following day, on April 26. South Africa’s National Institute of Communicable Diseases tested samples taken from the woman and confirmed that she had hantavirus.
A third person aboard the ship, who developed symptoms on April 28, died on May 2. Four other passengers who became ill were evacuated—one to South Africa and three to the Netherlands.
An eighth person had disembarked in Saint Helena and reported similar symptoms once he was in Zurich, Switzerland. A team at Geneva University Hospitals confirmed that he had become ill from the Andes virus—a form of hantavirus that can be spread between people.
Could this be the start of the next pandemic?
Health experts don’t believe so. They stress that the situation is nothing like the one the coronavirus that causes covid-19 presented in 2020. For a start, the Andes virus is not a mysterious new virus—scientists already have an understanding of it, and Argentina is sharing diagnostic kits it has already developed.
The virus also doesn’t spread in the same way. Officials at the World Health Organization emphasized that the spread of hantavirus requires close contact—the kind a person might have with a partner, household member, or medical caregiver.
The cruise ship outbreak represents “a specific confined setting where people are interacting in a prolonged close contact,” Abdirahman Mahamud, the alert and response director for the WHO’s health emergency program, said at a press event on Thursday. “With the experience our member states have, and the actions they have taken, we believe that this will not lead to a subsequent chain of transmission.”
What about the rest of the people onboard the ship?
All the remaining passengers have been asked to stay in their cabins, which the WHO says are being disinfected. Doctors and health professionals from the WHO and the European Center for Disease Prevention and Control have boarded the ship and are assessing everyone on board.
So far, no one else on board has developed symptoms, Maria Van Kerkhove, WHO acting director for epidemic and pandemic management, said at the press event. That’s “a good sign,” she said, but she added that the Andes virus has a long incubation period (around six weeks). Passengers are being advised to wear a medical mask when they leave their rooms.
At the same event, WHO director general Tedros Adhanom Ghebreyesus said he was in regular contact with the ship’s captain, who was reporting that “morale had increased significantly” since the ship started its journey to the Canary Islands.
What do we know about the Andes virus?
The Andes virus is the only hantavirus that is known to be transmitted between people. That transmission seems to rely on prolonged, intimate contact.
There was an Andes virus outbreak in Argentina around eight years ago. Between November 2018 and February 2019, there were 34 confirmed cases of infection, and 11 deaths. That outbreak was triggered when a person with symptoms attended a social gathering, said Tedros. “We are in a similar situation right now,” he said. “A cluster in a confined space with close contact.”
The fact that the 2018 outbreak was limited to 34 cases should be somewhat reassuring, he implied. “We believe this will be a limited outbreak if the public health measures are implemented and solidarity is shown across all countries,” he said.
How is hantavirus treated?
Unfortunately, we don’t have any specific antiviral treatments or vaccines for hantavirus. The WHO recommends early intensive care for people who develop symptoms. “This can save lives,” Anaïs Legand, WHO technical lead on viral hemorrhagic fevers, said on Thursday.
How did people get infected in the first place?
We don’t yet have an answer to that. But we do know that the couple who died had traveled through Argentina, Chile, and Uruguay on a birdwatching trip before they boarded the ship. That trip included visits to areas where species of rats that carry the Andes virus are known to live. The WHO is working with authorities in Argentina to try to retrace the couple’s movements on that trip.
Has the virus spread beyond the ship?
We don’t yet know for sure. The WHO is receiving reports of “potential suspect cases,” Van Kerkhove said at the Thursday briefing. Some of them have links to the ship or its passengers. Each “alert” will be followed up by health authorities in the relevant country, she said.
Has the US withdrawal from WHO affected anything?
Five US states have said they are monitoring US nationals who have disembarked from the ship. WHO officials are stressing that they are still sharing technical information with the US Centers for Disease Control and Prevention. “Things are … as they used to be,” Tedros said. “WHO’s mission is to help the world to be safe … and we want the American people to be safe as well.”
But it’s worth noting that cuts made by the Trump administration aren’t exactly putting the US in a good position for events like these. Last year, all full-time employees in the CDC’s Vessel Sanitation Program—which helps prevent and control illness outbreaks on cruise ships—were laid off. Further cuts to the CDC have left public health experts worried about how ill prepared the US is to deal with future disease outbreaks.
What will happen next?
Any suspected cases will be monitored by health authorities. Passengers are due to disembark in Tenerife in the Canary Islands on Sunday, May 10, and the WHO has said it will work with the Spanish government to ensure that the risk to residents remains low and that the passengers are treated with dignity and respect.
In the meantime, scientists are working to fully sequence the genome of the virus from patient samples. They want to find out if it is different from the viruses involved in the previous cases. “So far, we haven’t seen anything unusual,” said Van Kerkhove.
<![CDATA[ CT-1812 shows promise in Lewy body dementia, stabilizing hallucinations and delusions while easing caregiver distress.]]>
Remembering J. Craig Venter, PhD
J. Craig Venter, PhD recently passed away at the age of 79 from complications following a cancer diagnosis. He was well known in both science and industry and was an integral part of sequencing the human genome in the late 90s, competing with the government organized Human Genome Project. Throughout his career, he made many other important contributions in microbiology, with the “minimal cell,” in synthetic biology, and in personalized medicine. GEN editors share anecdotes of their experiences with him, reflect on the impact that his work has had on various fields in biology, in biotech, and in how the world has responded to the disruptions caused by Venter.
Listed below are links to the GEN stories referenced in this episode of Touching Base:
Genomics Pioneer and Life Sciences Entrepreneur J. Craig Venter Dies at 79
GEN, April 30, 2026
J. Craig Venter Describes a Human Genomics Revolution Still In Progress
By J. Craig Venter, PhD, GEN, June 13, 2025
Lessons from the Minimal Cell
By Hana El-Samad, PhD, GEN, August 21, 2023
From Sequencing to Sailing: Three Decades of Adventure with Craig Venter
By Fay Lin, PhD, GEN, March 8, 2023
“Cracking the Genome”
By Kevin Davies, PhD
Touching Base Podcast
Hosted by Corinna Singleman, PhD
Behind the Breakthroughs
Hosted by Jonathan D. Grinstein, PhD
The post Remembering J. Craig Venter, PhD appeared first on GEN – Genetic Engineering and Biotechnology News.
Opinion: I’m fighting misinformation online. False hantavirus claims follow a now-familiar playbook
I learned about hantavirus misinformation this week in the same way I now learn about most public health misinformation: My followers sent it to me.
Within hours of the first headlines about a hantavirus outbreak linked to the expedition cruise ship MV Hondius, my DMs started filling with screenshots. One was from the account of a Texas doctor who became well known during Covid for promoting ivermectin. She was already telling followers that ivermectin would work against hantavirus, too.
<![CDATA[Renaming borderline personality disorder fails to address the condition’s complexity; keeping the term preserves key clinical meaning, including structural traits and stress-related psychotic symptoms.]]>
ASGCT CEO David Barrett Previews the Upcoming Conference in Boston
The 29th American Society of Gene & Cell Therapy (ASGCT) meeting kicks off in Boston next week. The annual event will be a whirlwind of sessions, keynotes, fireside chats, posters, and exhibitors.
For the second year in a row, GEN spoke with David Barrett, JD, who has been the CEO of ASGCT since 2016. In this interview, we discuss his perspective on the event, if there is anything new that attendees should be looking out for, and what he, personally, is most looking forward to.
This interview has been edited for length and clarity.
LeMieux: The ASGCT meeting is an annual event. What are some of the things that will make this year’s meeting special?
Barrett: There is a lot that is special this year. First and foremost, it feels like a bit of a homecoming which is really exciting. The last time we were in Boston was in 2008. And Boston is a city and community where gene therapy, biotech, and research are all located. You can feel it when you’re in Cambridge and I think you are absolutely going to feel that when you’re inside the convention center.
The fact that the meeting is in Boston this year is also special for me because one of the very first things I did when I joined ASGCT in 2016, was to source the location for the 2020 annual meeting at the Hynes Convention Center in Boston. I was very excited and it was the first time we were going to take up an entire convention center. But that meeting, of course, did not happen; it had to be canceled because of COVID. So that makes this meeting in Boston particularly special. We finally get to have the meeting in Boston that I’ve been hoping for since 2016!
And we are growing. We are at the bigger of the two convention centers in Boston. We are going to surpass the total number of people that we had last year and I have every expectation that we’ll see significant growth year over year.
As far as other things that are that are new and interesting this year… I said this last year, but it’s worth adding it again—the science is always different. It is very consistent that we will have great science every year, and it is a wonderfully fun question mark of what exactly that science is going to look like. It’s always exciting because the science is always different year after year. So, by its very nature, it will be an exciting new conference this year.
Also, we’ll have a puppy park in the exhibit hall, so that’s really fun!
LeMieux: What are some things that will be highlighted at the meeting that ASGCT has been working on over the past year?
Barrett: ASGCT has done a lot this year. There is a lot that we have been very vocal about so far, and there is a lot that we’ll be sharing during the annual meeting.
Number one is that we partnered with Orphan Therapeutics Accelerator (OTXL) to found CGTxchange—the first and only clearing house and marketplace of its kind for cell and gene therapy assets. It is being built as we speak and we’ll have some exciting announcements during the annual meeting about assets that will hopefully be in the CGTxchange by that point. It is the culmination of a lot of work on what to do about commercially pre-viable (not non-viable) cell and gene therapies and the work that we’re doing to make those more commercially possible.
Also, ASGCT is hosting its Momentum Gala—the first formal gala at our annual meeting. That event has resonated really well with sponsors and donors. In fact, it is sold out! That event is going to be used to celebrate the launch of ASGCT Foundation, which is a separately incorporated 501C3 charitable foundation to support ASGCT’s mission to advance early career researchers and enable the development of cell and gene therapies. Also at the gala, we’ll be announcing some new initiatives to support patient access and reduce barriers to diagnosis, clinical trial participation, and treatment with cell and gene therapies.
Another major thing that’s going on is a considerable expansion of our educational activities. We recently launched a new e-learning tool and platform—the ASGCT Learning Center—a really fun project that we’ve been working on to expand how we we are getting new content to our new and expanding audiences.
We recognize that we have a really broad audience at ASGCT that is made up of cell and gene therapy basic science researchers, translational researchers, physician scientists and others in the ecosystem of drug development and administration for cell and gene therapies. And we’re looking at new ways to provide content that can help satisfy the learning needs of that really broad audience. The learning center is a big tool in our quiver to be able to do that.
LeMieux: What do you hope people take away from the meeting?
Barrett: I hope they take away a couple of things… number one, I hope they take knowledge, education, and awareness of what’s going on in the space and what has been happening over the course of the last 12 months. I hope that they take that back to their individual place of work. And I hope that, generally speaking, we fulfill our mission by expanding that knowledge base among all of the stakeholders in cell and gene therapy. Another thing that I hope people take away from this is that, after a lot of ups and downs and undulations in this field over the course of the past two to three years, that there is an extraordinary sense of excitement about the next phases in the development of cell and gene therapy drugs.
We have some really exciting new regulatory pathways. We have a lot of development of personalized gene editing technologies and techniques that can bring gene therapies much more quickly and effectively to patients who need them. We have seen significant advancements in more traditional or classic AAV gene therapies that are allowing these to be safer and more efficacious. And we’re seeing an expansion of cell-based gene therapies through an ever-expanding portfolio of indications that are reached by CAR Ts, primarily in cancer, but in an expanding outlook for the use of CAR Ts outside of cancer as well. So, I am hopeful that attendees come away with a renewed energy and vigor for the development of satellite gene therapies.
LeMieux: Is there anything specific planned at the meeting to touch on the concerns of the challenges that the scientific community is facing right now—with funding or other barriers?
Barrett: We are very excited to have Katherine Szarama, PhD—who was recently named acting director of FDA’s Center for Biologics Evaluation and Research (CBER)—participating in a fireside chat, addressing regulatory uncertainties. [Szarama replaced Vinay Prasad, MD, MPH, on May 1st.]
We have two other fireside chats focused on regulation, as well. The three fireside chats will offer attendees an opportunity to learn a little more, ask some questions, and hear from some of the individuals in those sessions specifically.
But I think that people will also see, more broadly, the ongoing work that ASGCT is doing to continue to create a partnership and a positive working relationship with the FDA to support those regulatory concerns.
LeMieux: What are you most looking forward to?
Barrett: I think I said this last year, but it really is one of my favorite components of the annual meeting. Every year, I look forward to taking some time to watch the exhibit hall being built. When the rope drops and people enter the exhibit hall for that very first reception, the hall is in pristine condition. And one of my favorite parts is watching it get to that pristine condition because it is just so exciting to see everything being built and come to a head, to have the whole field enter all at one space, and to be able to see an industry live and in person. Because so much of what we do is at our computer screens—and what we read about, hear about, or listen to people talk about. But when you actually see the field of gene and cell therapy on display, it is really exciting and satisfying.
Lastly, I will add that I’m looking forward to eating too much clam chowder while in Boston (chuckling).
The post ASGCT CEO David Barrett Previews the Upcoming Conference in Boston appeared first on GEN – Genetic Engineering and Biotechnology News.
ParcelBio Unveils Programmable mRNA Platform Backed by $13M Financing
Biotechnology company ParcelBio said this week that it has raised $13 million in a seed financing round led by Breyer Capital with participation from General Catalyst, Y Combinator, Metaplanet, SurgePoint Capital, ZAKA VC, and other investors. The financing will support the development of the company’s proprietary Amplified and Prolonged EXpression mRNA (APEXm
) platform and advance its pipeline including an in vivo CAR T program for autoimmune disease, as well as additional programs in oncology and encoded protein therapeutics.
The company, which is developing what it describes as a new class of durable mRNA medicines, will debut APEXm and share some preclinical data at the American Society of Gene and Cell Therapy annual meeting. This year’s meeting is being held in Boston, Massachusetts and will run from May 11-15. The company claims that its data will demonstrate that ParcelBio’s APEXm RNA drives significantly higher and more durable protein expression compared to another clinical mRNA design, and yields more complete target cell depletion in in vivo CAR T models.
“mRNA has transformed medicine, but today’s technologies are fundamentally limited in how much protein they can produce and for how long,” said David Weinberg, PhD, chief executive officer and co-founder of ParcelBio. His company’s proprietary technology addresses this problem by engineering RNA molecules to recruit the cell’s native RNA-stabilizing machinery, which enables higher and more durable protein expression. The company claims that its approach will result in medicines that reach thresholds that have historically been challenging for mRNA-based therapeutics. “We engineered RNA to work with the cell’s machinery rather than against it, enabling meaningful improvements in both expression and durability that we believe are essential for true disease modification,” Weinberg said.
Furthermore, ParcelBio’s platform maintains a simple, linear RNA architecture unlike circular RNA and other approaches, whose structure introduces manufacturing complexity or reduces output. Its broad applicability across proteins and cell types makes it suited for various therapeutic applications including immune programming and protein replacement.
“Most RNA platforms force a tradeoff between potency, durability, and manufacturability,” said Chris Carlson, PhD, chief scientific officer and co-founder of ParcelBio. “Our approach eliminates that tradeoff, enabling both higher peak expression and longer duration within a manufacturable system, and opening the door to entirely new classes of medicines.”
ParcelBio’s lead program focuses on in vivo CAR T therapies that target pathogenic B cells across autoimmune diseases, with the goal of achieving deep B-cell depletion for durable, drug-free remission. By enabling sustained CAR expression without viral delivery or ex vivo manufacturing, the company aims to develop scalable, off-the-shelf therapies. Additional programs leveraging the technology are currently in development in oncology and encoded protein therapeutics.
The post ParcelBio Unveils Programmable mRNA Platform Backed by $13M Financing appeared first on GEN – Genetic Engineering and Biotechnology News.
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)
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