Around the Helix: Cell and Gene Therapy Company Updates – May 10, 2023

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. ICER Reports Sickle Cell Gene Therapy Cost Effective at $1.9 Million

The Institute for Clinical and Economic Review (ICER) has drafted a report that has determined that gene therapy for sickle cell disease is cost-effective up to $1.93 million. The report focused on 2 gene therapies currently being reviewed by the FDA, Vertex and CRISPR’s exagamglogene autotemcel (exa-cel) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel).

2. Liso-cel Approved as Second-Line Treatment in LBCL by European Commission

Bristol Myers Squibb’s lisocabtagene maraleucel (liso-cel; Breyanzi), a marketed CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy, has been approved by the European Commission in all EU member states for the treatment of adult patients with diffuse large B-cell lymphoma (LBCL), high grade B-cell lymphoma, primary mediastinal LBCL, and follicular lymphoma grade 3B whose disease relapsed within 12 months from the completion of first-line chemoimmunotherapy or those whose disease is refractory to first-line chemoimmunotherapy.

3. Systemic Lupus Erythematosus CAR-T Gets FDA Fast Track Designation

Cabaletta Bio’s CABA-201, an investigational CD19-directed CAR-T therapy intended to treat systemic lupus erythematosus and lupus nephritis, has received fast track designation from the FDA. CABA-201 contains a 4-1BB costimulatory domain and is intended to deplete B-cells to “reset” the immune systems of patients with the disorders.

4. First Patient With ALS Dosed With Treg Cell Therapy

The phase 1 REGALS study (NCT05695521) evaluating the safety and tolerability of the regulatory T-cell (Treg) therapy CK0803 (Cellenkos) has dosed its first patient with amyotrophic lateral sclerosis (ALS). “The initiation of this study of CK0803 is an important achievement that brings us closer to delivering a potential new treatment which may have a life-changing impact for ALS," said Tara Sadeghi, Chief Operating Officer of Cellenkos Inc.

5. Janssen to Develop and Commercialize 2 of Cellular Biomedicine Group’s Non-Hodgkin Lymphoma CAR-Ts

As part of a new agreement, Janssen has gained exclusive rights for further development and commercialization of C-CAR039, which is targeted at both CD19 and CD20, and C-CAR066, which is CD20-targeted, in regions outside of China. Both CAR-T products have received clearance of their respective IND applications from the FDA.

6. WuXi Advanced Therapies to Manufacture Interius BioTherapeutics’ Investigational Lentivector

Interius BioTherapeutics’ INT-2104 is a lentivector product intended to generate autologous CAR-T cells in vivo via cell-specific gene delivery. WuXi Advanced Therapies’ GMP manufacturing platform will be utilized to produce INT-2104 product for use in a phase 1 clinical trial.

7. New Manufacturer Ascend Gene & Cell Therapies Launches Following Round of Series A Funding

Ascend Gene & Cell Therapies, which has received $132.5 million in total funding, will seek to provide biotechs with process development, clinical manufacturing services, and analytical/regulatory support. The company, which has assembled a team of 120 experts, noted that it has acquired CMC capabilities and technology from Freeline Therapeutics.

8. Life Biosciences and Forge Biologics Join Forces on Development of Aging-related Disease Gene Therapies

Forge Biologics will provide manufacturing services for Life Biosciences’ partial epigenetic reprogramming platform, along with AAV process development, analytical services, and toxicology. Development and manufacturing will take place at Forge Biologics’ facility in Columbus, Ohio. Life Biosciences noted that ophthalmic indications are among those that it intends to target with its platform.

9. Enlivex Receives Patent Related to Macrophage-Reprogramming Cell Therapy

Enlivex has received a European patent related to the use of Allocetra, its allogeneic cell therapy intended to reprogram macrophages, for purposes of preventing or managing cytokine release syndrome in patients being treated with CAR-T for cancer. The patent, which carries the title “Combination Immune Therapy and Cytokine Control Therapy for Cancer Treatment”, is numbered 3865189 and provides protection until at least 2037.

10. Bayer Launches New Life Science Incubator Focused on Cell Therapy and Gene Therapy in Cambridge

The new 26,000 square-foot facility, referred to as Co.Lab Cambridge, consists of laboratories and offices and is strategically located near the Bayer Research and Innovation Center. “[Co.Lab Cambridge’s] residents will benefit from the direct vicinity of industry-leading cell and gene therapy pioneers, global research, development, manufacturing, and investment know-how,” Friedemann Janus, the acting head of Business Development and Open Innovation at Bayer Pharmaceuticals, said.

11. Medigene Acquires Costimulatory Switch Receptor for TCR-T TherapiesFrom Helmholtz Munich

The CD40L-CD28 costimulatory switch receptor is intended to activate non-tumor cells in the tumor microenvironment and contribute to the breakdown of tumor stroma components, which in turn is expected to improve the ability of T-cell receptor engineered T-cell (TCR-T) therapies to infiltrate solid tumors.