Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Wugen’s WU-CART-007 has been granted fast track, rare pediatric disease, and orphan drug designations.
The European Commission has granted marketing authorization to PTC Therapeutics’ gene therapy Upstaza (eladocagene exuparvovec, PTC-AADC) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
Capricor Therapeutics has dosed the first patient with Duchenne muscular dystrophy (DMD) in its phase 3 HOPE-3 study (NCT05126758) of CAP-1002, an allogeneic cardiosphere-derived cell therapy.
The FDA has cleared the investigational new drug application for a phase 1 clinical study of OBX-115, an investigational tumor infiltrating lymphocyte (TIL) therapy developed by Obsidian Therapeutics and the University of Texas MD Anderson Cancer Center for the treatment of relapsed/refractory metastatic melanoma.
Mustang Bio has dosed the first participant with recombinase-activating gene-1 (RAG1) severe combined immunodeficiency (RAG1-SCID) with MB-110 in a phase 1/2 clinical trial (NCT04797260) in Europe.
Sangamo Therapeutics' TX200 is intended to reduce the risk of rejection in solid organ transplant. The STEADFAST phase 1/2 clinical trial dosed its first patient in March 2022.
The new company Replay will advance platform technologies that enable the writing and delivery of big DNA. These technologies could help enable polygenic therapy.
Cellularity's allogeneic off-the-shelf human placental CD34+ stem cell derived NK cell therapeutic candidate is genetically modified to express high-affinity and cleavage-resistant CD16 variant to enhance antibody-dependent cell-mediated cytotoxicity.
Kite's Tecartus, if approved, will be the only CAR T-cell therapy for patients 26 years of age and older with r/r B-cell precursor acute lymphoblastic leukemia.
The Beacon Platform, which Otsuka Pharmaceuticals acquired in October 2021, is also being used for cell line development research and antibody discovery.
The undisclosed private biotechnology company will gain the right to use the LentiVector platform in their lead CAR-T program, and in return Oxford Biomedica will receive an undisclosed upfront payment and various other payments and royalties related to development, manufacturing for clinical trials, and/or sales.
A-Gene, a collaboration between The Alliance for Regenerative Medicine and the National Institute for Innovation in Manufacturing Biopharmaceuticals, will attempt to address the issues that come from a lack of standardized methodologies and training for Chemistry, Manufacturing, and Controls in the field of gene therapy.