Biotechnology

FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease

The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal […]

Gene Therapy Pharmaceutical Business News

Abeona Receives FDA Regenerative Medicine Advanced Therapy Designation for Gene Therapy in Epidermolysis Bullosa

Gene therapy developer, Abeona Therapeutics, announced that the FDA has granted Regenerative Medicine Advanced Therapy designation to EB-101, a gene-corrected autologous cell therapy product for patients with recessive dystrophic epidermolysis bullosa (RDEB). Abeona`s EB-101 product is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient`s own skin cells (keratinocytes) for […]

Cancer Discovery Immunotherapies

FDA Approves the Roll-Over Combination Study with Checkpoint Inhibitor Immunotherapies to Allow Continued Access to BriaVax™ in Patients with Advanced Breast Cancer

The FDA has approved the roll-over combination study of the investigational breast cancer vaccine, BriaVax™ with pembrolizumab {Keytruda; manufactured by Merck & Co., Inc. or ipilimumab {Yervoy; manufactured by Bristol-Myers Squibb Company for patients previously treated with BriaVax™ from the ongoing Phase I/IIa Clinical Trial in Advanced Breast Cancer. BriaVax™ is a whole-cell breast cancer […]

cancer

FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma

This week, the U.S. Food and Drug Administration approved Yescarta (axicabtagene ciloleucel), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment. Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the […]

rare diseases

FDA awards 15 grants for clinical trials to stimulate product development for rare diseases

The U.S. Food and Drug Administration today announced that it has awarded 15 new clinical trial research grants totaling more than $22 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry across the country. “Given […]

Dermatology Gene Therapy

Autologous Cell Therapy for Epidermolysis Bullosa Gains FDA Breakthrough Therapy Designation

Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced today that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation status to the Company’s EB-101 gene therapy program for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB).  The designation from the FDA enables collaborative […]

Biotechnology cancer Clinical Trials Immunotherapies

FDA Halts Three Multiple Myeloma Studies Evaluating Merck’s KEYTRUDA®

Merck known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on KEYNOTE-183, KEYNOTE-185 and KEYNOTE-023, three combination studies of KEYTRUDA® (pembrolizumab), the company’s anti-PD-1 therapy, in the blood cancer multiple myeloma. This decision follows a review of data by the […]

cancer Clinical Trials Immunotherapies

Experimental CAR-T Treatment Halted as Two More Patients Die During Clinical Trials

Juno Therapeutics said Wednesday it has suspended a Phase II clinical trial of a cancer drug after two patients suffered cerebral edema earlier this week, leaving one dead and the other not expected to recover. The company’s ‘Rocket’ trial for B cell acute lymphoblastic leukemia is testing a drug it calls JCAR015. These drugs work […]