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.
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