Dual Designations Affirm Significant Unmet Need; Multi-Year Phase 1 Data Published in the New England Journal of Medicine Underpin Advancement to Phase 2 April 29, 2026 -- Expression Therapeutics, Inc., a clinical-stage biopharmaceutical company developing a curative autologous stem cell therapy for hemophilia A, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation and Rare Pediatric Disease Designation to its investigational therapy. The dual desi

ERAPs (ERAP1 and ERAP2) are emerging therapeutic targets associated with multiple autoimmune diseases. Genomics will assemble the largest available genetic resource to evaluate how ERAP variants influence an individual's risk of developing multiple autoimmune diseases. Insights will be used to strengthen Greywolf Therapeutics' autoimmune disease pipeline. April 29, 2026 -- Genomics, a science-led techbio company using large-scale genetic information to accelerate drug discovery and development

Following positive Phase IIa results for oral Rentosertib in GENESIS-IPF with good tolerability, a favorable PK profile, and dose-dependent efficacy improvements, the inhalation solution has received IND clearance from the CDE. The inhalation solution is designed for targeted lung delivery to achieve rapid onset and high local bioavailability at lower doses, while reducing systemic exposure and related side effects. Rentosertib inhalation solution is the 13th program from Insilico's AI-driven pi

Population modelling study published in Kidney International Reports Analysis of over 1.2 million genomes from five international databases predicts a substantial, previously underdiagnosed adult NPHS2-FSGS population Approximately 77% of cases in the US are predicted to involve two different NPHS2 variants (compound heterozygosity), most commonly R229Q paired with A284V. A284V is particularly prevalent in mixed-ancestry populations This modelling is supported by real world cohorts taken from cl

Funds will support one of the largest interventional clinical trials of a full-spectrum microbiome product, and the largest ever to be conducted in IBS Dosing of first patient of the Phase 2b ‘RISE’ trial anticipated in Q2 2026, and topline efficacy data anticipated in H2 2027 29 April 2026. EnteroBiotix Limited (‘EnteroBiotix’), a biopharmaceutical company developing best-in-class microbiome therapies for gut health, today announces the completion of a £19m ($25m) financing to advance its lead

After breaking through the blockbuster sales threshold in 2025, AstraZeneca’s three-in-one inhaler Breztri Aerosphere has passed another crucial milestone as it chips in on the British drugmaker’s goal to reach $80 billion in revenues by 2030. On Tuesday, the FDA approved Breztri in its second indication, clearing the triple combination therapy of budesonide, glycopyrrolate and formoterol fumarate as a maintenance treatment for asthma in adults and kids ages 12 and older. Breztri was first gree

April 28, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call today, April 28 at 4:30 p.m. ET to share results from the 24-month interim analysis of the PIVOT-HD long-term extension study of votoplam. PTC's partner Novartis stated on its quarterly earnings call today that long-term extension data are supportive of the now-initiated Novartis Phase 3 INVEST-HD study and Novartis and PTC will assess potential best next steps for the program including further actions wi

MENLO PARK, Calif.---Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy for the treatment of high-risk hematologic malignancies. "The FDA’s RMAT designation for Orca-Q recognizes the significant un

Onco3R Therapeutics unveiled a new name and a fresh financing on Tuesday. Now operating as Coultreon Biopharma, the biotech bagged a $125-million A round backed by big pharma to begin Phase II studies of its lead candidate, COL-5671 (formerly O3R-5671). The company is developing therapeutics that modulate salt-inducible kinases (SIKs), a protein family linked to inflammatory pathway regulation, to treat autoimmune diseases. COL-5671 is an oral, once-daily inhibitor of SIK3, a target that Coultr

Transaction establishes global ownership rights of lead program and includes initial capital funding with additional optional milestone-based capital support aligned with key clinical milestones. April 24, 2026 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) ("Telomir" or the "Company"), a biotechnology company developing small-molecule therapeutics targeting epigenetic and metabolic drivers of cancer and age-related diseases, today announced the successful closing of its previously announced acq