U.S. Food and Drug Administration– tag –
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Fatal Adverse Event Halts HuidaGene’s First-in-Human CRISPR Gene Therapy Trial for DMD
HuidaGene Therapeutics China Overview HuidaGene Therapeutics has reported a fatal serious adverse event (SAE) in the HG302-01 first-in-human trial of its investigational CRISPR-based gene-editing therapy, HG302, for Duchenne muscular dys... -
Peptide Therapeutics: Charting a Course to Oral Delivery Amidst a Surge in FDA Approvals
Stanford Medicine USA Overview The FDA has approved nearly 100 peptide therapeutics for conditions ranging from diabetes to multiple sclerosis, yet most require injection due to their susceptibility to gastrointestinal degradation. A rec... -
FDA Grants Dual Breakthrough and Orphan Designations to Volixibat for PSC-Related Cholestatic Pruritus
HCPLive USA Overview The U.S. FDA has granted both Breakthrough Therapy Designation (BTD) and Orphan Drug Designation (ODD) to volixibat, an investigational ileal bile acid transporter (IBAT) inhibitor, for the treatment of cholestatic p... -
ReCode Therapeutics Secures Funding, Partners with Gene Editing Firm to Fast-Track Cystic Fibrosis Gene Therapy with SORT LNP Platform
BioSpace (via Business Wire) USA Overview ReCode Therapeutics has announced new funding from the Cystic Fibrosis Foundation and a strategic partnership with a leading gene editing company. These initiatives will accelerate the developmen... -
FDA Designates Washington University’s WU-CART-007 as Breakthrough Therapy for T-Cell Malignancies: Allogeneic Anti-CD7 CAR T-Cell Therapy Achieves 73% Complete Remission
Siteman Cancer Center at Barnes-Jewish Hospital and WashU Medicine USA Overview The FDA has granted Breakthrough Therapy Designation (BTD) to WU-CART-007, an allogeneic anti-CD7 CAR T-cell therapy developed by Washington University Schoo... -
Entera Bio’s Oral Peptide Platform Advances to Phase 3 for Osteoporosis, Heralding a New Era for Injectable Therapeutics
Entera Bio, Inc. USA Overview Clinical-stage biopharmaceutical company Entera Bio has secured an agreement with the FDA to initiate a Phase 3 clinical trial for EB613, an oral parathyroid hormone (PTH) analog for postmenopausal osteoporo... -
Merck’s sBLA for ENFLONSIA™ Accepted by FDA for Expanded RSV Prophylaxis in High-Risk Infants Under Two for Second Season
Merck.com (News releases) USA Overview Merck announced that the U.S. FDA has accepted its supplemental Biologics License Application (sBLA) for ENFLONSIA™ (clesrovimab-cfor) to expand its Respiratory Syncytial Virus (RSV) lower respirato... -
Merck Achieves Robust Q2 2026 Performance, Upgrades Full-Year Guidance, and Empatran Secures FDA Breakthrough Therapy Designation for Lupus
Merck USA Overview Merck reported strong second-quarter 2026 financial results and consequently upgraded its full-year guidance, bolstered by the U.S. FDA's Breakthrough Therapy Designation for Empatran in July. Empatran, developed for l... -
Researchers Uncover Key Cellular Uptake Pathway for Antisense Therapies, Paving Way for Enhanced Efficacy
ecancer Global Overview Researchers have made a significant discovery, identifying the crucial intracellular uptake pathway for antisense oligonucleotides (ASOs), a therapeutic approach to halt aberrant protein production in various dise... -
Exosome Therapeutics Advancing in Neurodegenerative and Oncology Fields, Eyeing Blood-Brain Barrier Penetration
BioInformant Global Overview Exosome therapeutics are showing significant promise in 2026, particularly for neurodegenerative diseases due to their unique ability to cross the blood-brain barrier, with clinical development also progressi...
