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Rocket Pharmaceuticals Receives FDA RMAT Designation for Gene Therapy Targeting PKP2-Arrhythmogenic Cardiomyopathy

Friday, July 18, 2025

Rocket Pharmaceuticals has announced that the US Food and Drug Administration FDA has granted Regenerative Medicine Advanced Therapy RMAT designation to RPA its investigational gene therapy for plakophilin related arrhythmogenic cardiomyopathy PKPACM

Endeavor BioMedicines Secures Orphan Drug Designation for Taladegib in Both U.S. and Europe for Idiopathic Pulmonary Fibrosis

Thursday, July 17, 2025

Endeavor BioMedicines has received Orphan Drug Designation for its investigational treatment taladegib ENV from both the US Food and Drug Administration FDA and the European Commission EC

KalVista Pharmaceuticals Receives Approval for EKTERLY® (sebetralstat), the First Oral On-Demand Treatment for Hereditary Angioedema

Wednesday, July 16, 2025

KalVista Pharmaceuticals has announced that the UK Medicines and Healthcare products Regulatory Agency MHRA has granted marketing authorisation for EKTERLY sebetralstat

Repare Therapeutics Enters into Agreement with Debiopharm for Global Rights to Lunresertib

Wednesday, July 16, 2025

Repare Therapeutics has entered an exclusive global licensing agreement with Debiopharm for its investigational cancer therapy lunresertibLunresertib is a firstinclass PKMYT inhibitor designed to target hardtotreat solid tumours

Acumen and JCR Partner to Develop Advanced Brain-Targeted Therapy for Alzheimer’s Disease

Wednesday, July 16, 2025

Acumen Pharmaceuticals has announced a strategic collaboration and licence agreement with JCR PharmaceuticalsThe partnership will support the development of an Enhanced Brain Delivery EBD therapy aimed at improving the treatment of AD

Bayer Receives U.S. FDA Approval for Finerenone in Heart Failure with Preserved Ejection Fraction

Tuesday, July 15, 2025

Bayer has announced that the US Food and Drug Administration FDA has approved a new indication for finerenone Kerendia a nonsteroidal selective mineralocorticoid receptor antagonist for the treatment of adult patients with heart failure

Vertex Enters into Agreement with NHS England for New Cystic Fibrosis Treatment ALYFTREK®

Monday, July 14, 2025

Vertex Pharmaceuticals has reached a reimbursement agreement with NHS England for ALYFTREK deutivacaftortezacaftorvanzacaftor a oncedaily triple combination therapy for eligible patients with cystic fibrosis

FDA Grants Breakthrough Therapy Designation to Transcend Therapeutics’ TSND-201 for PTSD

Friday, July 11, 2025

Transcend Therapeutics has announced that the US Food and Drug Administration FDA has granted Breakthrough Therapy designation to TSND methylone an investigational treatment for PostTraumatic Stress Disorder

Merck to Acquire Verona Pharma in $10 Billion Deal, Adding COPD Treatment Ohtuvayre® to Its Pipeline

Thursday, July 10, 2025

Merck has announced plans to acquire Verona Pharma in a deal valued at approximately billionUnder the agreement Merck will purchase Verona Pharma for per American Depositary Share with each ADS representing eight ordinary shares

Sartorius Stedim Biotech and Sensible Biotechnologies to Scale Up Cell-Based mRNA Manufacturing Platform

Thursday, July 10, 2025

Sartorius Stedim Biotech has entered into a Memorandum of Understanding MoU with Sensible Biotechnologies to support the scaleup and industrialisation of Sensibles cellbased mRNA design and manufacturing platform