Recent Funding:
Aeovian secured $55M to complete a Phase 2 trial of AV078, a brain-penetrant, selective mTORC1 inhibitor designed to reduce seizures in tuberous sclerosis complex (TSC), aiming for improved efficacy and fewer side effects than older drugs like rapamycin and everolimus. The funding also supports preclinical work on additional programs, including a peripheral mTORC1 inhibitor for metabolic diseases and CD38 inhibitors that could expand into Duchenne, obesity, and MASH.
Ambros Therapeutics (OC) Launches with $125M to Bring Decades-Old Pain Drug to the U.S.
Ambros raised $125M to run a Phase 3 trial of neridronate for complex regional pain syndrome (CRPS-1), aiming to bring the Italy-approved therapy to U.S. patients who currently have no FDA-approved treatment options. Backed by notable investors including Vivek Ramaswamy and RA Capital, the company will focus on earlier-stage CRPS-1 patients, with FDA alignment on a single registrational study and potential U.S. patent protection into the 2040s.
Edison Scientific (SF) Raises $70M to Scale AI “Autonomous Science” Platform
Edison Scientific, the for-profit spinout of FutureHouse, raised a $70M seed round to expand its AI tools—including Kosmos, an agent that can autonomously run multihour research workflows equivalent to months of lab work. The company plans to commercialize its subscription-based platform for scientific literature reviews, data analysis, and autonomous experiment design, reflecting growing investor interest in “AI scientists” and automated research systems.
Addition Therapeutics (SF) Raises $106.5M to Advance Retrotransposon-Based Gene Insertion
The UC Berkeley–born biotech raised $106.5M to develop PRINT, a retrotransposon-powered platform that inserts full therapeutic genes using RNA + LNPs as a potential alternative to viral gene therapy and CRISPR. Addition aims to move beyond rare diseases into broader indications, with early programs (including an HIV-targeting antibody) progressing toward monkey studies next year.
M&A, Deals, Partnerships:
Clavis Bio (SF) Forms $7.25B Multi-Program Collaboration with Fosun Pharma
Aditum Bio launched Clavis Bio through a five-year partnership giving access to up to 20 drug programs, with milestone potential reaching $7.25B — one of the largest collaboration packages of the year. Clavis can nominate up to four targets per year and may develop and commercialize resulting medicines outside China, marking a major expansion beyond Aditum’s usual single-asset company model.
BioMarin (SF) Acquires Amicus Therapeutics for $4.8B to Expand Rare Disease Portfolio
BioMarin is buying Amicus for $4.8B in cash, adding two approved rare disease drugs — Galafold (Fabry) and Pombiliti + Opfolda (Pompe) — to accelerate its strategic turnaround and bolster revenue growth. The deal strengthens BioMarin’s enzyme therapy franchise and secures key patent settlements extending exclusivity into 2037, giving the company a larger, more durable rare-disease footprint.
Assembly Biosciences (SF) Hands Two Herpes Drug Candidates to Gilead in $35M Option Deal
Gilead paid $35M upfront to license Assembly’s two Phase 1b herpes treatments, ABI-1179 and ABI-5366, after early data showed strong antiviral activity and once-weekly oral dosing potential. Assembly remains eligible for up to $330M in milestones plus royalties, marking Gilead’s first opt-in move since its 2023 pipeline-wide partnership with the biotech.
Other Interesting News:
Verge Genomics (SF) Ends Development of ALS Drug After Phase 1b Failure
Verge’s small-molecule PIKfyve inhibitor VRG50635 failed to show efficacy in ALS, prompting the biotech to terminate the program and halt all internal drug development. The company will now pivot fully to an AI-platform partnership model, leveraging its patient-derived molecular datasets as it adopts a leaner structure and extends its cash runway.
Vistagen Therapeutics (SF) Misses Phase 3 Endpoint for Social Anxiety Drug Fasedienol
Vistagen’s late-stage study of intranasal fasedienol for social anxiety disorder failed to show benefit over placebo, despite earlier Phase 2 and Phase 3 success, raising uncertainty ahead of a final pivotal readout expected in 2026. The company is now implementing cash-preservation measures to extend runway into 2027 while investigating why placebo response was unusually high and how it may impact ongoing development.
