At WORLDSymposium, Astellas Pharma presented interim safety data from a Phase I/II study assessing the company’s AAV gene therapy for the rare, autosomal metabolic disease Pompe disease.
Amicus Therapeutics is launching a next-generation genetic medicine company called Caritas Therapeutics through a merger deal with ARYA Sciences Acquisition Corp IV, a special purpose acquisition company sponsored by Perceptive Advisors.
The U.S. Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients 1 year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe.
Amicus Therapeutics announced topline results from the Phase III PROPEL pivotal trial of AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease.
Data with Sanofi’s investigational enzyme replacement therapy avalglucosidase alfa in late-onset Pompe disease and infantile-onset Pompe disease will be featured as platform and poster presentations at the 17th annual WORLDSymposiumTM, to be held February 8-12, 2021.
Fast Track, Breakthrough and Priority Reviews: Sanofi and ViiVAcid alpha-glucosidase (GAA) enzyme, Biologics License Application (BLA), Breakthrough Therapy Designation, Bruton's Tyrosine Kinase (BTK) Inhibitors, Chronic Immune Thrombocytopenia (ITP), Clinical Trials, Enzyme Replacement Therapy (ERT), Fast Track Designation, FDA, HIV pre-exposure prophylaxis (PrEP), HIV Prevention, Muscle Disease, Orphan Drug Designation, PDUFA, Pompe disease, Priority Review Status, R&D, Sanofi
The U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.
Bayer is making a big bet on gene therapy with the acquisition of North Carolina-based Asklepios BioPharmaceutical (AskBio).
Asklepios BioPharmaceutical (AskBio) secured a $235 million investment from Vida Ventures and TPG Capital.
Roche acquires Spark Therapeutics for $4.3 billionAcquisitions, Analysts, Blindness, Blockbusters, Business, Cancer, Cells, Choroideremia, Gene Therapy, Hemophilia A, Hemophilia B, Huntington's Disease, Immune System, Immuno-oncology, Inherited Diseases, Leber’s Congenital Amaurosis, M&A, Pompe disease, Rare Diseases, Rare Genetic Diseases, Rare Inherited Disorders, Shares, Spinal Muscular Atrophy (SMA), Steroids
Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.
The U.S. FDA issued a Complete Response Letter to Netherlands-based Pharming Group for the company’s supplemental Biologics License Application for Ruconest.