Around the Helix: Cell and Gene Therapy Company Updates – October 1, 2025

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.

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1. MavriX Bio’s Angelman Syndrome Gene Therapy MVX-220 Garners FDA Fast Track Designation

MavriX Bio’s MVX-220, an investigational adeno-associated virus (AAV) vector-based gene therapy, has received fast track designation from the FDA for the treatment of Angelman syndrome.

2. FDA Publishes 3 New Draft Guidance for Industry Documents Aimed at Cell and Gene Therapy Development

The FDA has published 3 new “Draft Guidance For Industry” documents intended to provide guidance for the development of cell and gene therapy products.

3. CDKL5 and Gene Therapy

CGTLive® interviewed Jainu Jogani, the cofounder of Child’s Cure Genetic Research, whose daughter Reyna has CDKL5 deficiency disorder (CDKL5). Jogani went over the currently available treatment options and potential future treatment options for the disorder, highlighting a preclinical gene therapy program he and his team are currently working on.

4. World Heart Day 2025: Looking Back at Recent Progress in Cardiology Cell and Gene Therapy

This year for World Heart Day, which is held annually on September 29, CGTLive® has collected some of key news stories from the past few months that highlight the ongoing progress in the field of gene and cell therapy for cardiovascular disease.

5. BrainChild Bio Enlists CDMO OmniaBio for Manufacture of Pediatric Brain Tumor CAR-T

Under a new collaboration agreement, the contract development and manufacturing organization (CDMO) OmniaBio will provide manufacturing services to BrainChild Bio for the latter's planned pivotal clinical trial for BCB-276, an investigational chimeric antigen receptor T-cell (CAR-T) therapy that targets B7-H3 and is being developed for the treatment of a type of pediatric brain tumor referred to as diffuse intrinsic pontine glioma.

6. Takeda Jettisons Cell Therapy Programs

Takeda has announced that it will be ceasing all work on its cell therapy programs, citing a "strategic portfolio prioritization process". The company noted that it will look for a partner to carry on research with its cell therapy platform technologies and stated its intent to focus on its own efforts on its programs in other modalities, such as small molecules, biologics, and antibody-drug conjugates. Although, Takeda noted that it expects its work to continue to benefit from "novel insights" obtained from its cell therapy research.

7. Umoja Biopharma's In Situ CAR-T Therapy Fast Tracked for Several R/R B-Cell Malignancy Indications

The FDA has granted fast track designation to Umoja Biopharma's UB-VV111, a gene therapy product intended to create CD19-directed CAR T-cells within the body, for the treatment of relapsed/refractory (r/r) large B-cell lymphoma that has previously been treated with at least 2 prior lines of therapy and for the treatment of r/r chronic lymphocytic leukemia that has previously been treated with at least 2 prior lines of therapy.