Preclinical proof-of-concept data in GM2 gangliosidosis demonstrated survival benefit, functional improvement and target engagement Results are published in the 7 January issue of the Journal of Inherited Metabolic Disease Nizubaglustat is currently in Phase 3 registrational studies in GM1/GM2 gangliosidoses and Niemann-Pick type C disease (NPC) Azafaros, a company aiming to become a leader in lysosomal storage disorders (LSDs), focused on addressing especially neurological symptoms, today ann

Feb. 25, 2026 -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its Biologics License Application (BLA) for imsidolimab for the treatment of Generalized Pustular Psoriasis (GPP), with a target action date of December 12, 2026. GPP is a rare, chronic, life-threatening autoinflammatory skin disorder characterized by sudden flares of widespread pustules, erythema, and systemic symptoms such as fever and

- Blinded evaluation of safety and tolerability data by the Safety Monitoring Committee from Cohort 1 and advancement to Cohort 2 is expected in 3Q 2026 – - Advancement reflects continued execution of capital-efficient development strategy leveraging POZ Platform™- Feb. 24, 2026 -- Serina Therapeutics, Inc. ("Serina" or the "Company") (NYSE American: SER), a clinical-stage biotechnology company advancing drug candidates enabled by its proprietary POZ Platfor drug optimization technology, today

Feb. 25, 2026 -- Melodia Therapeutics AG today announced that the United States Patent and Trademark Office (USPTO) has granted a key U.S. patent covering a structurally novel DPP1 (Cathepsin C) inhibitor, currently developed as MLD-151 by Melodia Therapeutics and discovered by its partner, Alivexis, Inc., strengthening the intellectual property foundation supporting Melodia's lead development program. The newly issued patent (U.S. Patent No. 12,522,593; International Application No. PCT/JP2023

Approval in adults and children aged 6 years and older supported by Phase 3 trial demonstrating Dupixent significantly reduced nasal signs and symptoms, and systemic corticosteroid use or surgery compared to placebo AFRS is a chronic type 2 inflammatory disease of the sinuses characterized by an allergic hypersensitivity to fungi, often requiring surgery with high rates of post-operative recurrence Dupixent is now approved in the U.S. to treat nine distinct diseases driven in part by type 2 i

GSK said it will acquire a 100% equity stake in Canadian clinical-stage biotech 35Pharma for $950 million in cash, securing rights to the latter's lead asset — an activin signalling inhibitor (ASI) being developed for pulmonary hypertension (PH). "We're delighted to add HS235 to our pipeline, a potential best-in-class medicine with a differentiated profile to reduce risk of bleeding and provide potential metabolic benefits clinically relevant to PH patients," said Tony Wood, chief scientific of

February 24, 2026 – Jnana Therapeutics,a clinical-stage biotechnology company leveraging its next-generation RAPID chemoproteomics platform, announces the achievement of a second pre-clinical milestone from its first collaboration agreement with Roche (SIX:RO, ROG; OTCQX: RHHBY).Jnana has advanced a Roche-partnered immune program to an additional pre-clinical milestone, triggering a milestone payment to Jnana and handover to Roche. This is a first-in-class small molecule that uniquely inhibits a

February 25, 2026 Basilea Pharmaceutica Ltd, Allschwil (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, announced today that the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services, awarded USD 6 million to Basilea to continue the development of Basilea’s novel

Ontario's FAST program aims to speed up access to breakthrough cancer drugs for patients by almost a full year sooner.1 Itovebi offers a targeted treatment for advanced HR-positive, HER2-negative breast cancer with a PIK3CA mutation, following recurrence on or after completing adjuvant endocrine treatment. This mutation is found in approximately 40 per cent of HR-positive breast cancers and is generally associated with poor outcomes.2,3,4 Feb. 24, 2026 - Hoffmann-La Roche Limited (Roche Canada

Feb. 23, 2026 -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a clinical stage company pioneering immuno-oncology and RNA therapeutics for the treatment of high risk and advanced cancers, today announced the publication of a manuscript titled Template-Directed RIG-I Agonist Assembly for Image-guided Targeted Cancer Immunotherapy in the journal Molecular Imaging and Biology. The paper, published February 19, 2026, reports on a novel tumor-selective immunotherapy approach that activates innate imm