Recent Funding:
Tacit Therapeutics (SF) Raises $19M to Fix Genetic Diseases by Swapping Out Faulty RNA
Tacit is developing a new approach called “trans-splicing” that replaces large broken sections of RNA with corrected versions, rather than editing DNA directly. Their first program targets Huntington’s disease, where they’ve successfully repaired 70% of faulty molecules in mouse brains. The company is also working on a treatment for Dravet syndrome (severe epilepsy) and plans to start human trials within two years. Their method uses gene therapy delivery but acts on RNA, which they believe is safer than permanent DNA editing.
Recent Layoffs:
Theravance Biopharma (SF) Cuts 50% of Staff, Shuts Down All R&D After Phase 3 Failure
San Francisco-based Theravance is slashing half its workforce and completely shutting down R&D operations after its blood pressure drug ampreloxetine failed a pivotal Phase 3 trial in neurogenic orthostatic hypotension (a condition causing dangerous drops in blood pressure when standing). This marks the program’s second Phase 3 failure since 2021. The biotech is abandoning drug development entirely to focus solely on commercializing Yupelri, its already-approved COPD treatment, which is expected to generate $60-$70 million in annual cash flow. A board committee is exploring strategic options, including a potential sale of the company.
M&A, Deals, Partnerships:
Tenaya Therapeutics (SF) Partners with Alnylam in $1.13B Deal to Discover New Heart Disease Targets
San Francisco-based Tenaya is receiving $10 million upfront from RNA giant Alnylam, with potential milestone payments reaching $1.13 billion, to share genetic targets from their platform that could lead to new cardiovascular treatments. Tenaya has identified over 150 genetic targets since spinning out of UCSF’s Gladstone Institute in 2016. The cash-strapped biotech, which has conducted multiple layoffs and raised over $100 million last year, plans to begin pivotal trials in the second half of 2025 for two gene therapies targeting rare inherited heart conditions affecting roughly 190,000 US patients combined.
Day One Biopharmaceuticals (SF) Acquired by Servier for $2.5 Billion
French pharma Servier is paying $21.50 per share (68% premium) to acquire South San Francisco-based Day One, gaining the FDA-approved brain cancer drug Ojemda, which brought in $155 million in 2024 and is projected to hit $225-$250 million in 2026. Both companies now have leadership positions in treating rare brain tumors. Day One also brings two antibody-drug conjugates (ADCs) to the deal—one acquired from Mersana Therapeutics in January for $129 million and another from China’s MabCare—positioning Servier in the hot ADC space. The acquisition closes in Q2 2025 and marks one of Servier’s largest deals in its 72-year history.
Other Interesting News:
FDA’s Vinay Prasad Leaving Post After Clashes with Rare Disease Community
Vinay Prasad, M.D., director of the FDA’s biologics center (CBER) overseeing vaccines and gene therapies, will depart at the end of April to return to UCSF after a turbulent year marked by conflicts with rare disease advocates and biotech companies. He was briefly removed last summer during a dispute over Sarepta’s Duchenne gene therapy. Prasad drew criticism for rejecting multiple rare disease treatments, including reversing course on uniQure’s Huntington’s gene therapy after previously agreeing to their trial design, and initially blocking Moderna’s mRNA flu vaccine application before White House intervention led to a reversal.
