biotech12.825 min read
Lp(a) sparks biotech's next wave of cardiovascular drug development
17 hours ago•Source: Fierce Biotech
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2.595 min•3 days ago
Life Biosciences secures $80M to test aging-reversal gene therapy
David Sinclair’s Life Biosciences raised $80 million to move a one-time anti-aging gene therapy into clinical testing, pursuing partial epigenetic reprogramming with Yamanaka factors (Oct4, Sox2, Klf4) delivered by AAV to rewind cellular aging.
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Petrelintide Delivers Up to 10.7% Weight Loss in Zealand‑Roche Phase 2 Trial
Fierce Biotech•1 month ago
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Grail's Galleri multi-cancer test misses primary endpoint in NHS study, fueling skepticism
Grail's Galleri blood test for early cancer detection failed to meet its primary endpoint in a large NHS-backed study, renewing questions about its clinical utility despite some detected benefits. Grail reported selling 185,000 tests in 2025 for $136.8 million, and its stock fell about 47% after hours following the setback.
Compass’s psilocybin therapy advances toward FDA nod in severe depression
Compass Pathways reports positive Phase 3 results for its psilocybin-based treatment COMP360 in two trials for severe depression, suggesting potential FDA approval, though full data are needed to confirm the magnitude of benefit; if approved, it could become the first psilocybin product on the market, following Spravato (ketamine derivative).
FDA rejects Regenxbio’s Hunter syndrome gene therapy over biomarker doubts
The FDA has rejected Regenxbio’s RGX-121 gene therapy for mucopolysaccharidosis type II (Hunter syndrome), citing concerns about the surrogate cerebrospinal fluid biomarker used to predict longer-term cognitive benefit and casting doubt on the accelerated-approval pathway.
Lilly bets on in vivo CAR-T with $2.4B Orna Therapeutics deal
Eli Lilly agreed to acquire Orna Therapeutics for up to $2.4 billion to gain an in vivo CAR-T platform using engineered circular RNA delivered by lipid nanoparticles, led by ORN-252 targeting CD19 for autoimmune diseases; terms include upfront and milestone-based payments. The deal follows a wave of pharma acquisitions in this space as Lilly expands its genetic medicines pipeline and Orna has drawn funding and collaborations since its 2021 launch.
Capricor Advances Deramiocel BLA: FDA Seeks Full HOPE-3 CSR
FDA has requested the full HOPE-3 clinical study report (CSR) and supporting data to address Capricor’s Complete Response Letter for the Deramiocel BLA; Capricor will submit the CSR and updates in February 2026 to support continued FDA review and a new PDUFA date, following HOPE-3 topline data showing significant skeletal muscle and cardiac improvements in Duchenne muscular dystrophy.
Corvus' eczema pill shows early promise with strong skin-improvement signals
Corvus Pharmaceuticals reports encouraging early-stage results for its oral eczema treatment soquelitinib: after eight weeks, 75% of treated patients reached EASI-75 and 33% achieved clear or almost clear skin versus 20% and none on placebo, suggesting potential superiority to Dupixent, though data are preliminary and from a small trial.
Metsera's Monthly Weight Loss Drug Shows Promising Early Results
Metsera's experimental drug targeting the amylin hormone showed promising results in early clinical trials, indicating potential for longer-lasting weight loss effects compared to existing weekly injections, highlighting a significant development in obesity treatment.
"Insitro's AI Biotech Breakthroughs at JPM 2024"
Insitro, a well-funded AI biotech company founded by Daphne Koller, has unveiled its efforts in ALS, liver disease, and cancer at JPM. The South San Francisco firm, which has raised over $643 million in venture capital, is making strides in the biotech industry with its AI technologies and has more than 260 employees working on its projects.
Vertex CSO David Altshuler discusses non-opioid pain pills, AI, and the potential for a groundbreaking drug
David Altshuler, Chief Scientific Officer of Vertex Pharmaceuticals, implemented a unique R&D strategy that focused on targeting specific diseases such as sickle cell and type 1 diabetes and utilizing various tools and platforms, including CRISPR-Cas9 and cell therapy, to develop treatments. The company's CRISPR therapy for sickle cell is awaiting approval and could potentially be the first of its kind on the market.
Nimbus Therapeutics Secures $210M in Funding for Tech-Enabled Medicines
Biotech company Nimbus Therapeutics has raised $210 million in private financing, including investments from GV (formerly Google Ventures) and returning investors such as SR One, Atlas Venture, and Gates Frontier. This comes after the company sold an experimental pill to Takeda Pharmaceutical earlier this year in a deal that could be worth up to $6 billion. Nimbus has not yet opened a lab, commercialized a drug, or gone public with an IPO.