Rugonersen, a potentially best-in-class antisense oligonucleotide (ASO) therapy in development for Angelman syndrome, advanced into the pivotal Phase 3 BEACON clinical trial Angelman syndrome affects approximately 30,000 diagnosed individuals in the US and EU5 and has no approved treatments Topline data expected in early 2029 July 28, 2026 -- OHB Pediatrics Ltd. d/b/a Oak Hill Bio, a clinical-stage rare disease therapeutics company, today announced that the first participant has been dosed in B

Bemnifosbuvir/Ruzasvir (BEM/RZR) Achieved its Primary Endpoint of Statistical Non-inferiority Compared to Sofosbuvir/Velpatasvir (SOF/VEL; Epclusa®), a Standard-of-Care, in First-Ever Successful Head-to-Head Trial in a Global Phase 3 Program of Hepatitis C Virus (HCV) Treatments Results Reinforce Potential Best-in-Class Pro BEM/RZR as a Short 8-Week Treatment for Patients Without Cirrhosis Up to 4 Million People in the US Living with HCV, Which Can Progress to Cirrhosis, End-Stage Liver Disease

July 28, 2026 -- Cullinan Therapeutics, Inc. (Nasdaq: CGEM; “Cullinan”), a clinical-stage biopharmaceutical company accelerating potential first- or best-in-class, disease-modifying T cell engagers in autoimmune diseases and cancer, today announced positive feedback from the U.S. Food and Drug Administration (FDA) following an End-of-Phase 1 (EOP1) meeting for CLN-049, a FLT3xCD3 T cell engager being evaluated in patients with acute myeloid leukemia (AML). The EOP1 meeting focused on the planne

Argenx is spending $2.2 billion to absorb Forte Biosciences for its phase 2-stage vitiligo and celiac disease drug. The prize for argenx is FB102, an anti-CD122 antibody that demonstrated success in a phase 1b study earlier this month. In that trial, Forte tied FB102 to a 29.6% improvement in a score of facial vitiligo. A readout for phase 2 celiac trial is expected later this year. Argenx, which has previously invested in Forte, said phase 1 studies of FB102 in both indications were “key driv

- Company’s third program to enter clinical development leveraging FORCE platform with the goal of delivering functional improvement in rare neuromuscular disease - July 28, 2026 -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for its IND application to initiate a Phase 1

The venture financing and AMED grant funding position CORE to accelerate development of novel lineage-based oncology therapies for patients around the world. July 28, 2026 -- CORE Biomedicine, a biotechnology company pioneering lineage-based precision oncology therapeutics, today announced the completion of a $21 million Series A financing. The round is co-led by UTokyo Innovation Platform Co., Ltd. and Elikon Venture, alongside participation from InnoPinnacle Fund, Mitsubishi UFJ Capital Co.,

July 27, 2026 -- Agency issues "study may proceed" letter; development pathway does not require a clinical endpoint efficacy study. DifGen Pharmaceuticals LLC today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's Investigational New Drug (IND) application and issued a "study may proceed" letter for DFGN-101, an intramuscular injection under development for the treatment of advanced or metastatic breast cancer in women. DFGN-101 is designed to be administer

July 27, 2026 -- AbelZeta Pharma, Inc. ("AbelZeta" or the "Company"), a global clinical-stage biopharmaceutical company focused on the discovery and development of innovative and proprietary cell-based therapeutic products, today announced that the Company received the U.S. Food and Drug Administration (FDA) clearance under its Regenerative Medicine Advanced Therapy (RMAT) designation regarding the multiple-center registrational Phase II clinical trial for C-CAR168, an anti-CD20/BCMA bispecific

July 27, 2026 -- Harbour BioMed (HKEX: 02142), a global biopharmaceutical company committed to the discovery and development of novel antibody therapeutics in immunology, oncology and other areas, today announced that it has entered into a strategic collaboration with China National Pharmaceutical Group Co., Ltd. ("Sinopharm"). The two parties will establish the Sinopharm - Harbour BioMed Innovation Consortium (the "Innovation Consortium") to advance collaborative research and development of inn

Proceeds to support ongoing late-stage global clinical development and commercialization preparation of dotinurad, a next-generation, once daily, oral URAT1 inhibitor with the potential to deliver best-in-class safety and efficacy for the treatment of gout July 27, 2026 -- Urica Therapeutics, Inc. (“Urica” or the “Company”), a Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) subsidiary, today announced that Crystalys Therapeutics, Inc. (“Crystalys”), in which Urica maintains an equity positio