Belief BioMed Group Initiates Clinical Study for Hemophilia A Gene Therapy BBM-H803 in China
Belief BioMed Group, a Shanghai-based gene therapy specialist, has announced the first subject dosing in...
Rare / orphan disease drugs
BMS’ Augtyro Receives EMA Review for Advanced Lung Cancer and Solid Tumors Treatments
The European Medicines Agency (EMA) has accepted for review a filing submitted by Bristol Myers...
Boan Biotech Secures FDA Orphan Drug Designations for Pancreatic Cancer Treatments
China-based Boan Biotech (HKG: 6955) has announced that its novel antibody BA1105 and antibody-drug conjugate...
Janssen Acquires Rights to Gene Therapy Botaretigene Sparoparvovec from MeiraGTx for XLRP Treatment
Janssen Pharmaceuticals, a subsidiary of Johnson & Johnson (J&J, NYSE: JNJ), announced last week its...
AstraZeneca Secures FDA Approval for Self-Administered hATTR-PN Therapy Wainua
AstraZeneca (AZ, NASDAQ: AZN), headquartered in the UK, announced last week that the U.S. Food...
Simcere Affiliate Shanghai Simnova Biotech Gets China Nod for CAR-T Therapy SNC109
Shanghai Simnova Biotech Co., Ltd, known as Simnova outside of China and an affiliate of...
HuidaGene Secures Rare Pediatric Disease Designation for CRISPR Therapy HG302 Targeting DMD
HuidaGene Therapeutics, based in China, has announced that it has received Rare Pediatric Disease Designation...
EMA’s CHMP Gives Green Light to Sanofi’s Fexinidazole for Acute Sleeping Sickness Treatment
Last week, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines...
Sino Biopharmaceutical Submits Market Approval Application for Recombinant Coagulation Factor VIIa
Sino Biopharmaceutical Ltd (HKG: 1177), a leading biopharmaceutical company based in China, has announced the...
Lepu Biotechnology’s Pancreatic Cancer Drug Earns Orphan Drug Status from FDA
Lepu Biotechnology Co., Ltd (HKG: 2157), a Chinese pharmaceutical company, has announced that it has...
AstraZeneca Acquires Icosavax to Boost Infectious Disease Pipeline with VLP Vaccine Technology
UK-based pharmaceutical giant AstraZeneca (AZ; NASDAQ: AZN) has announced the acquisition of US vaccines specialist...
Sanofi Abandons MZE001 Licensing Deal Following FTC Intervention Over Pompe Disease Monopoly Concerns
Sanofi (NASDAQ: SNY) has withdrawn from an agreement announced earlier this year to in-license the...
Skyline Therapeutics’ Gene Therapy SKG0201 for SMA Gets Green Light from China’s NMPA
Skyline Therapeutics has received clinical trial approval from China’s National Medical Products Administration (NMPA) for...
Novartis Reports Kisqali Success in Reducing Recurrence Risk in Early Breast Cancer
Novartis (NYSE: NVS) has announced an update on the late-stage trial for its CDK4/6 inhibitor...
Novartis’ Fabhalta Wins FDA Nod as First-in-Class Treatment for Paroxysmal Nocturnal Hemoglobinuria
Swiss pharmaceutical giant Novartis (NYSE: NVS) has secured regulatory approval from the US Food and...
Stem-Cell Startup Xellsmart Earns FDA Orphan Drug Designation for ALS Therapy
Xellsmart, a stem-cell startup headquartered in Suzhou, has announced that it has received orphan drug...
China’s State Council Unveils Comprehensive Plan to Boost Beijing’s Healthcare Sector
The State Council has unveiled a comprehensive “Work Plan” aimed at enhancing Beijing’s service industry...
BMS and Avidity Biosciences Join Forces on $2.2 Billion Cardiology Antibody Oligonucleotide Conjugate Deal
Bristol Myers Squibb (BMS, NYSE: BMY) has announced a strategic partnership with Avidity Biosciences (NASDAQ:...
Sichuan Kelun Pharmaceutical’s RET Inhibitor A400 Earns FDA Orphan Drug Designation
Sichuan Kelun Pharmaceutical Co., Ltd (SHE: 002422), a Chinese pharmaceutical company, through its innovative drug...
China’s CDE Releases Draft Guidelines for Decentralized Trials in Rare Disease Drug Development
China’s Center for Drug Evaluation (CDE) has released draft “Technical Guidelines for the Application of...