Around the Helix: Cell and Gene Therapy Company Updates – November 23, 2022

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.

1. Hemgenix First Gene Therapy Approved for Hemophilia B

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.

2. Upstaza Approved for AADC Deficiency in UK

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.

3. Lifileucel BLA Completion Pushed Back to Q1 2023

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”.

4. Omidubicel PDUFA Date Extended to May 2023

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.

5. Thermo Fisher Scientific Unveils Gibco CTS AAV-MAX Helper-Free AAV Production System

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.

6. Replay Launches Second HSV Gene Therapy Company

Telaria will pursue new gene therapies for rare skin diseases that utilize Replay's herpes simplex virus (HSV) delivery vector.

7. Sarcura Successfully Closes Round of Financing for Cell Therapy Manufacturing System Prototype

The €7M Seed Plus funding round will enable Sarcura to advance development of its platform, which uses silicon chip technology and microfluidic processing.

8. PerkinElmer Launches New Viral Vector Assays

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.

9. Umoja Biopharma, IASO Biotherapeutics to Collaborate on New Cell Therapies for Hematological Cancers

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.

10. Coeptis Therapeutics Partners With IQVIA To Bring New CAR-T Technology to the Clinic

IQVIA will help determine ideal targets for Coeptis Therapeutics' SNAP-CAR platform, which was licensed the University of Pittsburgh.

11. Fujifilm Announces Details of New Cell Culture Media Manufacturing Facility Plans

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.

12. Pan Cancer T, NecstGen Partner to Bring New TCR-T Therapies to the Clinic

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.