GAINESVILLE, Fla. — University of Florida scientists have discovered why a paralyzing brain disorder speeds along more rapidly in some patients than others — a finding that may finally give researchers an entry point toward an effective treatment for amyotrophic lateral sclerosis, often referred to as ALS or Lou Gehrig’s...
Latest News
SAN DIEGO – Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer, today announced encouraging interim Phase 1 data from the Company’s off-the-shelf, iPSC-derived natural killer (NK) cell programs in relapsed / refractory acute myeloid leukemia (AML). The ongoing Phase...
5 of 6 Patients Achieve Objective Response, including 4 Patients with Complete Response, with Single Dose of FT596 at 900 Million Cells in Combination with Rituximab 13 of 19 Patients Achieve Objective Response with Single Dose of FT596 at 90 Million and 300 Million Cell Dose; 10 of 11 Patients...
When a doctor asks for your family medical history, he or she is not just being nosy – it can mean the difference between life and death. Just ask the Smith family of the Cotton Grove community. Tony Smith, 42, tested positive for colon cancer in February 2008. He had...
How far would you go to save your child’s life? Haoyang was born in Kunming in southwestern China two years ago with a very rare disease: Menkes Syndrome. The disease is a genetic disorder that impacts how copper – which is crucial for brain and nervous system development – is...
Nashville, Tennessee – Among adult patients with severe sickle cell disease (SCD), favorable outcomes were reported with use of a modified transplantation procedure, according to results from a multicenter study presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition in December 2023. Although the use of...
EMERYVILLE, Calif. — XOMA Corporation the biotech royalty aggregator, announced today that based upon the U.S. Food and Drug Administration’s (FDA) acceptance of Zevra Therapeutics’ New Drug Application (NDA) for arimoclomol, an orally-delivered, first-in-class therapy for Niemann-Pick disease type C (NPC), XOMA will make a $1 million milestone payment to...
On March 22, an FDA Advisory Committee (AdCom) unanimously voted in favor of accelerated approval for Biogen’s tofersen for a rare form of amyotrophic lateral sclerosis (ALS). Panelists voted 9-0 that tofersen’s demonstrated reduction in plasma neurofilament—a biomarker of neurodegeneration—was “reasonably likely” to predict clinical benefit in ALS. The AdCom...
Washington DC – The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee agreed that Vertex Pharmaceuticals had sufficiently demonstrated the safety of its investigational sickle cell disease gene-edited therapy exagamglogene autotemcel (exa-cel). Still, the panel of external experts did indicate that Vertex and its partner CRISPR Therapeutics could follow treated...
Most parents wouldn’t be thrilled with the idea of their kids getting hooked up to an IV bag filled with trillions of viruses. But for Melanie Hennick, whose son, Connor, has Duchenne muscular dystrophy, it was an opportunity she hoped would change his life. “We knew this wasn’t a cure,”...