Around the Helix: Cell and Gene Therapy Company Updates – November 8, 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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social and use #AroundTheHelix!

1. New Clinical Data Presented at SITC’s 38th Annual Meeting

Catch up on CGTLive’s coverage of the Society for Immunotherapy of Cancer’s (SITC) 38th Annual Meeting, held November 1-5, 2022, both virtually and in San Diego, California. Our coverage will continue throughout the week.

2. Clinical Trial Updates Presented at ESGCT’s 30th Annual Congress

Catch up on CGTLive’s coverage of the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), held October 24-27 in Brussels, Belgium.

3. University of Manchester Launches MPS II Gene Therapy Trial, Picking Up Where AVROBIO Left Off

University of Manchester was previously collaborating on the development of AVR-RD-05 with AVROBIO but regained the license to the hematopoietic stem cell gene therapy following the company’s cessation of development on all programs.

4. Tenaya's Phase 1b MyPeak-1 Trial is Testing the Waters in Gene Therapy for Hypertrophic Cardiomyopathy

CGTLive took a closer look at Tenaya Therapeutics’ recently initiated clinical trial evaluating TN-201, an investigational adeno-associated virus vector-based gene therapy intended to treat hypertrophic cardiomyopathy caused by mutations in the MYBPC3 gene

5. The Future of Dermatology? B-VEC's Impact in DEB 6 Months Post Approval

As we reach 6 months out from the therapy’s approval for the treatment of dystrophic epidermolysis bullosa (DEB), CGTLive spoke to experts in the field about their experiences and impressions of Krystal Biotech’s beremagene geperpavec (B-VEC) as well as future questions and research needs that remain.

6. Newcomer Rampart Biosciences Garners $85 Million in Series A Round of Financing

Rampart Biosciences, a company newly launched with $40 million in seed funding from OrbiMed, is focused on developing nonviral DNA-based medicines. The company stated that its initial efforts will be directed at rare genetic diseases, with its first program being intended to treat patients with hypophosphatasia. 

7. CDMO AGC Biologics to Take On Manufacturing of Medigene’s TCR-T Therapy 

Under a new service agreement, AGC Biologics, a contract development and manufacturing organization (CDMO), will provide support to Medigine by undertaking the manufacturing of its investigational product MDG1015 for the purpose of investigational new drug application filing and clinical trial activities. MDG1015, currently in preclinical development, is an autologous T-cell receptor T-cell (TCR-T) therapy intended to treat solid tumor indications.

8. Kyverna and Charité Join Forces to Investigate the Impact of B-Cell-Targeting Therapies in Autoimmune Disease

Charité – Universitätsmedizin Berlin, Department of Rheumatology and Clinical Immunology has signed an agreement with Kyverna Therapeutics to fund research on outcomes for patients with autoimmune diseases treated with approved or investigational therapies that target B-cells and plasma cells. The research, which will cover the impact of chimeric antigen receptor T-cell (CAR-T) therapies like Kyverna’s own KYV-101, will evaluate patients treated at Charité.

9. Cabaletta Bio to Try Out Manufacturing of CAR-T CABA-201 On Cellarres’ Platform

Under a new agreement, Cabaletta Bio and Cellarres will test manufacturing of CABA-201, Cabaletta’s investigational CAR-T that is intended to treat autoimmune diseases, with the use of Cellarre’s Cell Shuttle, an automated manufacturing platform. “Through our Technology Adoption Partnership program, we aim to fully automate all cell therapy manufacturing processes, thereby bringing us a step closer to potentially delivering CAR T-cell therapies for patients with a broad range of autoimmune disease,” Fabian Gerlinghaus, MS, the CEO of Cellares, said in a statement.

10. Lexeo Therapeutics Prices IPO at $11 Per Share

Lexeo Therapeutics, which is developing gene therapies for indications including arrhythmogenic cardiomyopathy and APOE4-associated Alzheimer disease, made an initial public offering of 9,090,910 common stock shares on November 3, 2023. This share pricing was slightly lower than previous expectations that the shares would be offered at $13 to $15.