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Transcripta Bio Raises $24M for AI-Driven Neurological Disease Therapies

Chris Moxham, PhD, has been a “drug hunter” for three decades, building data-driven platforms as vice president of quantitative biology at Eli Lilly, and CSO of Folcrum Therapeutics, in recent years. 

“Technology is now advancing to allow us to interrogate the transcriptome, which is a phenomenal blueprint for cell state and fate,” he told GEN Edge.  

Moxham currently leads Transcripta Bio as founder and CEO. The AI-driven drug discovery start-up is developing small molecule therapeutics to modulate gene expression disease signatures. 

Transcripta has now announced a $24 million funding raise to advance IND-enabling studies and clinical preparation of its neurological disease portfolio, including autism spectrum disorder (ASD) and facioscapulohumeral muscular dystrophy (FSHD). Mayo Clinic and Omnimed will join JAZZ Venture Partners, BlueYard Capital, and a group of life sciences family offices, as investors. 

Phase II clinical trials are “where the rubber meets the road,” says Moxham. He emphasizes that de-risking therapies early is key to improving drug discovery success rates, which often fall below 10%. 

Founded in 2023, the Palo Alto-based company currently houses fifteen employees. “This isn’t a company you could have built five years ago,” highlighted Moxham. He cites the intersection of scalable sequencing technology, compute power, and lab automation among the factors enabling the rise of AI-driven biology. 

Three-pronged approach 

While much of the field has defined transcriptome AI models as the “virtual cell,” Moxham emphasizes Transcripta’s translational focus. 

The company’s proprietary platform takes a three-pronged approach. First, disease signatures are identified using patient-derived single cell RNA-seq (scRNA-seq) data. An in-house generated “drug atlas” then measures the effects of small molecule perturbations across 80% of the transcriptome, capturing full dose-response profiles in diverse cellular contexts, including glutamatergic and motor neurons, fibroblasts, and keratinocytes. These data power AI models that identify promising compounds that can therapeutically modulate gene expression.

Transcripta’s neurological disease pipeline is structured as a tiered portfolio of novel molecules and repurposed clinical-stage assets, with the latter benefiting from existing human safety data that can shorten development timelines and lower costs. 

In 19q12 syndrome, a form of ASD, the team demonstrated that entrectinib, an FDA-approved oncology drug, could reverse disease when given at low concentrations. One patient case demonstrated clinical benefit within nine months after taking the drug.  

In Huntington’s disease, Transcripta’s platform identified novel molecules that could downregulate DNA mismatch repair protein and validated therapeutic target, MSH3. The company plans to file an IND next year. Transcripta is also pursuing pre-IND research in FSHD and myonic dystrophy.  

Moxham emphasizes the generalizability of the platform. “We are now looking at hundreds of diseases with this type of approach,” he says.  

Transcripta plans to introduce another cohort of therapeutic programs by Q2 of 2027.

The post Transcripta Bio Raises $24M for AI-Driven Neurological Disease Therapies appeared first on GEN – Genetic Engineering and Biotechnology News.

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AI Protein Engineering Model Designed for Biomanufacturing

A cell engineering model designed specifically for the biomanufacturing environment could go a long way toward optimizing host cell performance, reducing development timelines, and streamlining biomanufacturing platform design.

“There’s a real challenge in determining how to engineer a host cell’s genome to maximize its performance in a biomanufacturing application.” That’s the difficulty laid out by Shawn Manchester, PhD, CEO, Triplebar Bio, and a view that’s shared by Yun Song, PhD, professor, University of California, Berkeley, and the industry innovation organization BioMADE. The three, with support from the National Science Foundation, are developing such an AI-informed cell engineering model to solve that challenge.

What’s most notable is that this AI-informed predictive and optimization model focuses on the biomanufacturing environment. The large-scale, application-specific training database is being designed to handle bench- to commercial-scale biomanufacturing.

Biomanufacturing-relevant environment

As Manchester says, “There aren’t a lot of organisms that have naturally evolved to make proteins they’ve never seen before, in an environment they’re not well-evolved for. Our ability to generate data in a setting that is relevant to biomanufacturing and to do so at the scale of hundreds of thousands of cells and data points is innovative…and it’s not something that many others are looking at. Most people are working on AI for protein engineering of therapeutics and other molecules as opposed to cell engineering for use in biomanufacturing.”

This project focuses on data capture from Pichia pastoris as the host cell producing five different proteins that are relevant for bioprocessing, food production, and defense. The learnings will be incorporated into Triplebar’s other programs, too, including optimization for its Chinese hamster ovary (CHO) cells, Manchester says.

“Improving the scalability of these proteins directly benefits [not just bioindustrial or biopharmaceutical applications, but a broad range of] biomanufacturing,” Brandon Simmons-Rawls, program manager, BioMADE, emphasizes.

This AI project combines two core technologies deployed at Triplebar:

  • Droplet microfluidics, in which a cell is encapsulated in a water and oil emulsion alongside a fluorescent-based sensor to quantify protein production
  • Multimodal transformer-based AI model

The genomes and transcriptomes of cells that make either more or less protein than baseline are sequenced and fed into the AI’s very large, labeled training sets to identify correlations between genotypes and phenotypes. These correlations are important patterns about how the genome works, Manchester explains, and can be used to generate new genomic designs for optimization of the host cell to the biomanufacturing process.

“We first pre-train models on all relevant sequencing data from thousands of genomes, which gives us an underlying structure to the genome. Then we fine-tune those models with the genotype-phenotype data we generate in the biomanufacturing-relevant environment. This allows us to understand which of those patterns in the genome relate to biomanufacturing-specific performance,” Manchester says.

Once this AI-enabled model rolls out, Manchester says he envisions it being used by bioindustrial and biopharma companies that “need to improve the productivity or efficiency of the organism they use in manufacturing. Results should be faster and more efficient than those derived using guess-and-check methods based on our current understanding of how these organisms work.

“The goal is for people to log onto this AI tool, identify what they’re making and what they’ve done, and ask the AI what else they can do to improve performance,” he continues. “The model will serve them specific genetic designs that they can deploy in their organism.”

Manchester predicts that more than 10% of the designs will produce meaningful improvements—significantly more than traditional, early-stage design-build-test cycles. This streamlines the cell engineering cycle by focusing on modifications likely to yield performance improvements that are most meaningful for biomanufacturing.

The post AI Protein Engineering Model Designed for Biomanufacturing appeared first on GEN – Genetic Engineering and Biotechnology News.

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Cell-Therapy Manufacturers Get Creative on Checking Particulates

Manufacturers of cell-containing products are adopting new techniques to work around unclear regulatory guidance on particulates. That’s the experience of Diana Colleluori, PhD, principal chemistry, manufacturing and controls (CMC) consultant at Biologics Consulting.

According to Colleluori, cell-containing products can be harder to visually inspect for particulates, as they’re not clear and are often stored in opaque bags.

“Processing and testing cell-containing products already has challenges because they can’t be terminally sterilized,” she explains. “It’s also harder to make a visual assessment to meet regulatory requirements because they already contain cells and the final product is usually in a cell-freezing bag.”

The particulates are mostly (90%) plastic that enter the product from contact with the inside of single-use equipment used during manufacturing, she says, adding that other particulates can enter from product manipulation and, thus, it’s best to try to minimize this.

Companies that Colleluori has worked with have tackled this problem by running their process with the formulation buffer, but minus cells, she continues. This allows them to assess particulates in a clear solution, outside of a bag, which they can then extrapolate to running the same process with cells.

“By testing visible and sub-visible particles in those samples, you can prove your product contact materials are expected to meet the limits for your cell-containing products,” according to Colleluori.

The company can also run quality control tests on a small sample of the final product. Among the tests carried out are those based on the United States Pharmacopeia (USP) chapters 790 for visible particulates and injections,1790 for visual inspections of injections, and 788 for the sub-visible particles.

In addition, EU guidance on visual inspection of particles can also be relied upon. Which tests are run should depend on the method of administration, adds Colleluori.

“If you’re doing an intramuscular injection, it’s probably a little less rigorous than an intrathecal injection of products that have stricter regulations on the number of particles and sub-particles that can be present,” she says.

The post Cell-Therapy Manufacturers Get Creative on Checking Particulates appeared first on GEN – Genetic Engineering and Biotechnology News.

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Scaling Cell and Gene Therapy Manufacturing

As cell and gene therapies (CGTs) move closer to mainstream clinical use, manufacturers are under growing pressure to bridge the gap between scientific breakthroughs and commercial reality. To explore this transition, GEN talked with three industry leaders, and here’s what we learned.

GEN: In today’s bioprocessing for cell and gene therapies, what is the most crucial business challenge and why?

Jonathan Wofford, COO, Title21 Health Solutions: Currently, the most crucial business challenge to solve in cell and gene therapy bioprocessing is scalable, cost-effective manufacturing. While scientific advances and clinical outcomes have accelerated the demand for therapy development, producing consistent, high-quality products at commercial scale remains difficult due to the increasing complexity of processes, limited automation, underdeveloped data-management infrastructures, supply-chain constraints, and patient-specific workflows for autologous therapies.

Justin Irizarry, CEO, OrganaBio: The disconnect between starting material and everything downstream. Despite heavy investment in manufacturing and analytics, the quality and consistency of raw biological material still determines whether a process succeeds. Variable, fragmented sourcing becomes failed runs, slipped milestones, and regulatory risk, and you can’t engineer that variability back out of a living product.

Carol Houts, CEO, Germfree: The most pressing challenge in CGT bioprocessing today is the gap between clinical promise and manufacturing readiness. Developers are advancing therapies faster than the infrastructure, workforce, and supply chain can scale to support them, and that mismatch is costing time and patients.

GEN: What is the most effective way to address these challenges?

Wofford: In theory, the approach should focus on end-to-end process standardization and automation, supported by closed-system manufacturing, digital process infrastructure, and scalable platform technologies. This reduces variability, decreases labor costs, improves regulatory compliance, increases throughput, and enables consistent product quality from clinical development through commercial production. I say in theory because adoption may be limited by the significant upfront investment needed to implement advanced manufacturing technologies.

Irizarry: Vertical integration and co-location: control the chain from donor through processing and testing under one quality system, with a recallable donor pool, identical SOPs across RUO and GMP, and processing near collection sites.

Houts: The most effective response is committing to manufacturing strategy early, not as an afterthought to clinical development. That means purpose-built environments, modular infrastructure that can grow with the program, and partners who understand GMP from day one. The groundwork determines whether a therapy ever reaches the people who need it.

The post Scaling Cell and Gene Therapy Manufacturing appeared first on GEN – Genetic Engineering and Biotechnology News.

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Hybrid Single-Use and Stainless-Steel Plants the Greener Option

Hybrid biopharmaceutical manufacturing facilities that combine stainless steel (SSTs) and single-use (SUTs) technologies are the “sweet spot” from an environmental sustainability perspective, according to new research. The research compared the environmental impact—specifically, the carbon footprint— of hybrid facilities with those where SUTs are used, arguing that ongoing efforts to decarbonize energy generation may have shifted the metrics.

And the key findings are that the CO2 footprint of SUTs is significantly higher than previously assumed and that key SST process steps can now have a lower carbon footprint than their single-use counterparts.

Lead author Jan Reiners, rer. nat. , from Roche, tells GEN, “For a long time, SUT was considered more sustainable because it avoids the energy needed to clean and steam-sterilize stainless steel. However, this paradigm has now inverted due to the rapid decarbonization worldwide due to the expansion of renewable energy.

“A single-use run at the 2,000L scale generates up to 6.5 tons of CO2e per batch only in plastic and packaging waste. We didn’t do this assessment for a full stainless-steel system, but the hybrid facility used less than half of plastics,” he adds.

Reiners and colleagues used “market-based emissions accounting” data for the analysis, noting it is becoming common for drug companies to decouple emissions from local energy supplies through tools like power purchase agreements and on-site photovoltaic (PV) installations.

“For instance, our own organization, Roche, has recently achieved its global goal of sourcing 100% sustainable electricity across all worldwide operations. And we are not alone: AstraZeneca is reducing energy emissions by 98% by 2026 and transitioning to 100% renewable energy for heat and power. Novartis claims to have become carbon neutral for energy in 2025, as do many other companies,” he says.

The researchers also found that some long-standing assumptions about SUT systems— for example, that they have lower HVAC costs—no longer hold.

“In modern biomanufacturing, cleanroom ISO classifications remain identical for both SUT and SST due to functionally closed processing, while the massive logistics and warehousing required to store SUT consumables actually expand physical space requirements,” continues Reiners. “And finally, the mass of fossil-fuel-based plastics was underestimated and its CO2 impact actually increased by ~30% in updated global life cycle databases like Ecoinvent v3.12.”

Hybrid alternatives

Considering the findings, Reiners and co-authors suggest that, to minimize emissions and costs, biopharmaceutical manufacturers need to combine stainless-steel systems with SUTs.

“Although SUT is less sustainable, it has other advantages like faster product changeover, more flexibility, less investment, but higher running costs. A hybrid facility is the ‘sweet spot’ of combining SUT and stainless steel to reduce the CO2 impact,” he maintains. “Depending on the facility layout, various measures can reduce the footprint, but per se it is about retaining stainless steel for simple, high-volume, resource-heavy operations—like buffer and media preparation—while keeping single-use for the complex, flexible core process like bioreactors.

“In our study, comparing a full-SUT facility to a hybrid facility reduced the material-related carbon footprint by 62%, dropping emissions from 6.5 tons of CO2e to just 2.8 tons of CO2e per batch.”

The post Hybrid Single-Use and Stainless-Steel Plants the Greener Option appeared first on GEN – Genetic Engineering and Biotechnology News.

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Congo’s Ebola outbreak has killed over 1,000 people

BUNIA, Congo — More than 1,000 people have died in the Ebola outbreak in Congo, according to Africa’s top health body, a grim toll in the fastest Ebola outbreak in history, as conflict, community resistance and an uneven response fuel its spread.

Speaking at a health summit in Ghana on Wednesday, Dr. Jean Kaseya, director-general of the Africa Centres for Disease Control and Prevention, said 1,031 deaths have been confirmed.

Read the rest…

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STAT+: Pharma tries to tamp down broad China restrictions

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Good morning. All my social media feeds have been filled with Jimothy the raccoon, and I hope your feeds now will be too.

The need-to-know this morning

  • Celldex Therapeutics said its experimental drug barzolvolimab failed in a Phase 2 study in the skin condition prurigo nodularis, sending its shares down in premarket trading. The company is continuing to test the drug in a number of other indications, with data expected in September or October from Phase 3 trials in chronic spontaneous urticaria, which causes chronic hives. 
  • Summit Therapeutics released updated survival data from its Phase 3 HARMONi trial testing the drug ivonescimab in a form of non-small cell lung cancer. The FDA is set to make an approval decision on the drug in the disease by Nov. 14.  

Pharma tries to tamp down broad China restrictions

From STAT’s Daniel Payne: Pharmaceutical leaders are trying to convince policymakers to back off pushes for aggressive restrictions on investments in Chinese firms.

Continue to STAT+ to read the full story…

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