Around the Helix: Cell and Gene Therapy Company Updates – December 15, 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. Data From Various Clinical Studies Presented at ASH 2022

Catch up on CGTLive's coverage of the 64th American Society of Hematology (ASH) Annual Meeting, held December 10-12, 2022, in New Orleans, Louisiana.

2. Genome-Edited HCT Clinical Trial Demonstrates Engraftment in First Patient With AML

Vor Bio’s tremtelectogene empogeditemcel (trem-cel; VOR33), an investigational allogeneic genome-edited hematopoietic stem and progenitor cell product, has demonstrated successful transplant and engraftment in the first patient with acute myeloid leukemia (AML) in the phase 1/2a VBP101 clinical trial (NCT04849910).

3. Gamma-Delta T-cell Therapy Gets Green Light From FDA for Phase 2 Trial

IN8bio’s INB-400, an investigational, genetically modified gamma-delta T-cell therapy intended to treat glioblastoma (GBM), has received clearance of its investigational new drug (IND) application by the FDA.

4. Phase 1/2 MPS I Gene Therapy Trial Finishes Dosing

The phase 1/2 clinical trial (NCT03580083) of REGENXBIO’s RGX-111, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat severe mucopolysaccharidosis Type I (MPS I), has completed dosing of all 8 enrolled patients.

5. Immix Biopharma In-Licenses NXC-201, a Next Generation BCMA-targeted CAR-T Therapy

Immix Biopharma has formed a wholly-owned subsidiary, Nexcella, Inc., to develop and potentially commercialize NXC-201.

6. Actinium Pharmaceuticals, Columbia University to Evaluate Actimab-A in the Treatment of Patients With AML Who Previously Received CD33-negative HSC

The gene-edited HSC technology was developed in the lab of Siddhartha Mukherjee, MD, DPhil, assistant professor of medicine at Columbia University Medical Center in the Department of Medicine and Division of Hematology/Oncology, and is the same technology also licensed to Vor Bio.

7. Japan Marketing Authorization for YESCARTA to be Transferred from Daiichi Sankyo to Gilead Sciences K.K.

Daiichi Sankyo originally acquired the marketing rights to the CAR-T in Japan from Kite Pharma as part of a 2017 agreement.

8. SonoThera to Use Lantheus's Microbubble Platform for Ultrasound-Guided Gene Therapy

The companies intend to provide a potentially safer and more cost-effective alternative to the viral vector approach of delivering gene therapies.

9. Neogene Therapeutics Announces its Acquisition by AstraZeneca

Neogene Therapeutics is focused on the development of T-cell receptor therapies (TCR-Ts) for solid tumors.

10. Therabest, Glycotope Announce Collaboration on Combination Therapy

The companies will evaluate Therabest's TB-100, an iPSC derived NK cell therapy, and Glycotope's immuno-cytokine GT-00AxIL15 for the potential treatment of triple-negative breast cancer.

11. Xpress Biologics Acquired by Polyplus

Polyplus intends to leverage the acquisition of Xpress Biologics, a Belgian CDMO focused on plasmid DNA and protein manufacturing, to provide GMP grade plasmids by early next year.

12. Emmes Announces the Creation of New Research Center

The clinical research organization's new Cell and Gene Therapy Center will employ a team of 100 researchers to aid the development of the company's client's therapies for indications including hematology, oncology, cardiology, ophthalmology, and rare diseases.