Around the Helix: Cell and Gene Therapy Company Updates – September 13, 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.

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1. Novartis Drops Development of Geographic Atrophy Gene Therapy GT005

Novartis has discontinued development of GT005 (PPY988), an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat geographic atrophy secondary to dry age-related macular degeneration, that was being evaluated in several clinical trials including the phase 2 EXPLORE study (NCT04437368).

2. First Patient Receives Gene Therapy for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

The first patient with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer has been treated with nadofaragene firadenovec(Adstiladrin; Ferring Pharmaceuticals) as part of an Early Experience Program.

3. BioCardia Likely to Miss the Mark in Phase 3 CardiAMP Heart Failure Cell Therapy Trial

BioCardia’s phase 3 clinical trial (NCT02438306) of its CardiAMP cell therapy, an investigational autologous cell therapy for the treatment of heart failure, is unlikely to meet its primary end point based on the results of an interim analysis, according to an announcement made by the company.

4. Axi-Cel Expands LBCL Indication in China

Axicabtagene ciloleucel (axi-cel), marketed under the name Yikaida (Fosun Kite Biotechnology, Kite Pharma) in China, has been approved in the country under an expanded indication, for the treatment of adult patients with large B-cell lymphoma whose disease did not respond to first-line immunochemotherapy or relapsed within 12 months.

5. Kriya Picks Up Nonalcoholic Steatohepatitis Gene Therapy Program Through Acquisition of Tramontane Therapeutics

Tramontane’s lead program, an AAV vector-based gene therapy, is intended to treat nonalcoholic steatohepatitis by enabling expression of fibroblast growth factor 21. “The addition of Tramontane’s FGF21 program strategically aligns with our Metabolic Disease portfolio which also includes a one-time gene therapy candidate for insulin-dependent diabetes,” Shankar Ramaswamy, MD, the co-founder and CEO of Kriya, said in a statement.

6. Verily to Provides Support to Kyverna With Immune Profiler Platform

As part of a new collaboration, Verily is lending support in the form of its immune mapping platform, Immune Profiler, to Kyverna Therapeutics’ efforts in developing KYV-101, a CAR-T therapy currently under clinical evaluation for lupus nephritis. Immune Profiler will be used to help identify biomarkers for treatment response that may help improve the design and carrying out of clinical trials.

7. Moderna and Immatics Link Up for 3-Pronged Collaboration

The 2 companies will put their efforts towards implementing mRNA technology for in-vivo expression of T-cell receptor bispecifics; mRNA-based cancer vaccine development activities; and assessing the potential of a combination treatment consisting of Moderna’s PRAME mRNA-based cancer vaccine used inconjunction with Immattics’ TCR-T therapy IMA203.

8. Otsuka and ShapeTX Join Forces

With the use of ShapeTX’s capsid discovery platform, the 2 companies will put their combined efforts towards development of AAV vector-based gene therapies for ocular disease indications that will be delivered intravitreally. “We’ve built our AAVid platform on generative AI approaches akin to those behind Midjourney and DALL-E 2 to tackle industry challenges with gene therapy delivery,” Francois Vigneault, PhD, the co-founder and chief executive officer of ShapeTX, said in a statement.

9. 2seventy bio Lets Go of 40% of Its Employees

The company cited the current economic climate as a reason behind the layoffs, which included the removal of 176 roles. 2seventy bio also noted that it will narrow its efforts on a subset of its pipeline programs and prioritize Abecma. In addition to these changes, the company also recently announced an expansion of its collaboration with JW Therapeutics that includes the addition of another solid tumor program.

10. MDA and Charcot-Marie-Tooth Association Team Up 

The Muscular Dystrophy Association (MDA) and Charcot-Marie-Tooth Association (CMTA) have engaged in a $299,992 collaborative research grant that will focus on assessing a nonviral gene delivery approach to treating CMT disease type X. “This project applies the latest techniques in nanoparticle technology to tackle a major challenge in the development of CMT treatments, getting them to the part of the body where they are needed,” Katherine Forsey, PhD, the chief research officer of CMTA said in a statement.

11. Sanguine Biosciences Launches Its New Apheresis Donor Center 

The center, located in Los Angeles, California, will collect materials consisting of concentrated white blood cell biospecimens, referred to as leukopaks, for the purpose of use in research and development activities for advanced therapeutics including cell therapies. "We looked at the market and saw the unmet supply for scientists' high demand for healthy and disease-state leukopak products," Cyrus K. Mirsaidi, chairman of BioSelective Capital Investments and Sanguine board member, said in a statement.

12. CancerVAX and UCLA Working on Universal CAR-T Platform Alternative to Ex-Vivo Production of CAR T-cells

UCLA reported to CancerVAX findings from the Universal Cancer Vaccine project that indicate potential for using cancer vaccine technology to create CAR T-cells, reprogrammed from normal T-cells, within patients’ bodies. “The extreme low cost of the COVID vaccine, which uses similar technology and concepts, leads us to believe that this major cost reduction is possible with our Universal CAR-T Cell platform,” Ryan Davies, the CEO of CancerVAX, said in a statement.