Around the Helix at ASGCT: Cell and Gene Therapy Company Updates – May 18, 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. This week’s Around the Helix focuses on the latest updates presented at this week’s 2022 American Society of Gene and Cell Therapy Annual Meeting.

1. Company Collaboration Identifies At-Risk Genotypes for DMD Gene Therapy

A working group established by sponsors Pfizer, Sarepta, Genethon, and Solid Biosciences developing gene therapies for the potential treatment of Duchenne muscular dystrophy has elucidated at-risk genotypes for anti-transgene serious adverse events shared across programs.

2. Editas’ Gene-Edited Cell Therapy for Beta Thalassemia Wins Orphan Status

EDIT-301 is currently being evaluated in severe sickle cell disease, with a phase 1/2 trial in transfusion-dependent beta thalassemia upcoming later in the year.

3. Arcellx Doses First Patient in ARC-SparX CAR-T Program for Multiple Myeloma

ACLX-001 is being evaluated in a phase 1, open-label clinical trialand is the first to come out of the company’s proprietary ARC-SparX platform.

4. Mustang Bio’s Third Generation CAR T Product Shows Efficacy in Follicular Lymphoma

Further data from Mustang Bio’s phase 1/2 trial will be presented at the EHA 2022 Congress.

5. Caribou’s CRISPR-Edited CAR T-Cell Therapy CB-010 Elicits Complete Response in R/R B-NHL

CB-010 produced a 100% overall response rate (ORR) in all 5 patients with relapsed or refractory B cell non-Hodgkin lymphoma who received the therapy in the ANTLER phase 1 trial (NCT04637763).

6. Improved Function Observed Following Mini-Dystrophin Gene Therapy in DMD

Study sponsor Pfizer recently announced the launch of a phase 3 trial in patients with Duchenne muscular dystrophy.

7. Analysis Elucidates Lentiviral Vector-Mediated Myelodysplastic Syndrome Cases

More study is necessary of bluebird bio’s eli-cel and LVV-mediated oncogenesis but the gene therapy offers significant clinical benefit to patients with cerebral adrenoleukodystrophy.

8. Autoantigen CAR T Cell Therapy Well-Tolerated in Mucosal-Dominant Pemphigus Vulgaris

Cabaletta Bio’s DSG3-CAART cell thearpy was found to be well-tolerated with dose-dependent persistence in participants with mucosal-dominant pemphigus vulgaris (mPV).

9. CAR Macrophage Therapy for HER2 Solid Tumors Continues Through First in Human Trial

Investigators are evaluating the safety, tolerability and preliminary efficacy of Carisma Therapeutics' CT-0508 in patients with solid tumors and HER2 overexpression.

10. AVROBIO’s Cystinosis Gene Therapy Reduces Cystine Crystals

Data from the ongoing Phase 1/2 gene therapy clinical trial of AVR-RD-04 demonstrated the therapy’s efficacy in reducing the disease impact of cystinosis, with no treatment-related AEs reported.