PHILADELPHIA, PA — Phenylketonuria (PKU) is a rare newborn genetic disease that impacts between 1 in 10,000 to 1 in 20,000 people, depending on the individuals’ genetic ancestry. PKU causes an amino acid—called phenylalanine (Phe)—to build up in the bloodstream. Uncontrolled PKU can lead to intellectual disability, psychiatric issues, and...
treatment News
BOSTON, Mass. — PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PGN-EDODM1, an investigational candidate...
IRVINE, Calif. — PeproMene Bio, Inc., a clinical-stage biotech company developing novel therapies to treat cancers and immune disorders, today announced that the cohort 1 first patient treated in its Phase 1 relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) clinical trial of PMB-CT01 (BAFFR-CAR T Cells) has reached...
New Delhi – Achalasia is a disease where the function of the oesophagus stops working overtime. It is thought to be neural degeneration of the selective nerve fibers in the body of the oesophagus and the valve at the top of the stomach. It is a rare disease with an...
TUSTIN, Calif. — Peregrine Pharmaceuticals, Inc. (Nasdaq: PPHM) today highlighted the progress that the company has achieved in its Phase II program assessing the combination of bavituximab and chemotherapy in three separate cancer trials. Bavituximab is a monoclonal antibody with a unique mechanism that allows the body’s own immune system...
Peregrine has presented the data for ongoing trail for brain cancer at Society of Nuclear Medicine 2009 Annual Meeting. The study supports the potential for Cotara to treat brain cancer. Cotara, a targeted monoclonal antibody linked to a radioisotope, is being developed as a potential new treatment for glioblastoma multiforme...
NEW YORK & MIAMI – Pfizer Inc. (NYSE: PFE) and OPKO Health, Inc. (NASDAQ: OPK) announced today that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) for somatrogon. Somatrogon is an investigational once-weekly long-acting recombinant human growth hormone for...
New York, NY – The novel hemophilia therapeutic marstacimab has been accepted for submission by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for regulatory approval, according to a news release. Pfizer announced in a news release that the Biologics License Application (BLA) for its...
New York, NY – Health Canada on Wednesday approved Pfizer’s gene therapy fidanacogene elaparvovec, now to carry the brand name Beqvez, for the treatment of adults with hemophilia B. Beqvez is an adeno-associated virus-based gene therapy that delivers a functioning and high-activity copy of the human coagulation FIX gene, which...
DAEJEON, South Korea — PharmAbcine Inc., a clinical-stage biotech company focusing on next-generation antibody therapeutics, announced today the initiation of patient dosing in the Phase 1a/b clinical trial of PMC-309 in patients with advanced or metastatic solid tumors. PMC-309 is an IgG1 monoclonal antibody with specific binding to VISTA in...