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.
The FDA has approved UniQure and CSL Behring’s etranacogene dezaparvovec (EtranaDez), now marketed as Hemgenix, for treating adults with hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
PTC Therapeutics’ Upstaza (eladocagene exuparvovec), a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy intended to treat aromatic L–amino acid decarboxylase (AADC) deficiency with a severe phenotype, has received marketing authorization from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of children aged 18 months or older.
New FDA feedback has pushed back Iovance Biotherapeutics’ expected 2022 lifileucel biologics license application for treating melanoma to the first quarter of 2023. The FDA has requested supplemental validation information and comparability data for lifileucel, which the company will address “promptly”.
The Prescription Drug User Fee Act (PDUFA) date for Gamida Cell’s omidubicel, an investigational allogeneic hematopoietic stem cell transplant (HSCT) candidate intended for the treatment of patients with hematological cancers, has been extended from its original January 30, 2023, date to May 1, 2023.
The system is intended to provide cost-effective current good manufacturing practice production of adeno-associated virus (AAV) vectors for gene therapies at large scales.
Telaria will pursue new gene therapies for rare skin diseases that utilize Replay's herpes simplex virus (HSV) delivery vector.
The €7M Seed Plus funding round will enable Sarcura to advance development of its platform, which uses silicon chip technology and microfluidic processing.
The 7 Adeno-associated Virus Vectors (AAV) Detection Kits utilize the company's AlphaLISA technology and are intended expand assay range and speed up the research for new gene therapies.
The collaboration will combine Umoja's iCIL platform with IASO's chimeric antigen receptors for the development of new off-the-shelf therapies, and will start with a focus on acute myeloid leukemia.
IQVIA will help determine ideal targets for Coeptis Therapeutics' SNAP-CAR platform, which was licensed the University of Pittsburgh.
The company has invested $188 million in the planned facility, which will be more than 250,000 square feet in area and located in Research Triangle Park, North Carolina.
NecstGen will provide process and analytical development services for Pan Cancer T's investigational TCR-T therapies, which the company intends to progress towards clinical evaluation for the treatment of solid tumors.