Around the Helix: Cell and Gene Therapy Company Updates – September 27, 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. Taysha Gene Therapies Drops Development of Giant Axonal Neuropathy Gene Therapy TSHA-120

Taysha Gene Therapies has discontinued development of its lead candidate TSHA-120, an investigational adeno-associated virus (AAV) vector-based gene therapy that was being evaluated for the treatment of giant axonal neuropathy.

2. Mesoblast Gears Up for Third Push for Remestemcel-L's Approval in GvHD

Mesoblast is continuing its push to try and get its mesenchymal stromal cell therapy remestemcel-L approved to treat pediatric and adult steroid-refractory acute graft versus host disease (GvHD) under the name Ryoncil, with plans including conducting an additional single-arm trial informed by a recent Type A meeting with the FDA.

3. Krystal Biotech’s Type 1 Alpha-1 Antitrypsin Deficiency Gene Therapy KB408 Cleared for US Trial

Krystal Biotech’s KB408, an investigational herpes simplex virus type 1-derived vector-based gene therapy, has been cleared by the FDA for a phase 1 clinical trial (NCT06049082; Serpentine-1) in patients with type 1 alpha-1 antitrypsin deficiency.

4. Precigen Trying Its Hand at CAR-T for Ovarian Cancer in Phase 1/1b Trial

Precigen’s PRGN-3005 UltraCAR-T, an investigational MUC16-directed chimeric antigen receptor T-cell (CAR-T) therapy intended for the treatment of patients with platinum-resistant ovarian cancer, is currently being assessed in a phase 1/1b clinical trial (NCT03907527).

5. Decibel Officially Becomes a Subsidiary of Regeneron

Regeneron has completed its acquisition of Decibel Therapeutics, which is developing DB-OTO, an investigational AAV vector-based gene therapy intended to treat otoferlin-related hearing loss. DB-OTO is currently being evaluated in the phase 1/2 CHORD clinical trial (NCT05788536). Decibel also has several gene therapies in preclinical development for other hearing loss indications.

6. ElevateBio BaseCamp to Incorporate Kyverna’s Manufacturing Platform

The Ingenui-T platform, Kyverna’s process for autologous manufacturing of CAR-T products specifically directed at autoimmune indications, will be incorporated into partner ElevateBio’s manufacturing and development operations, which are carried out under the name ElevateBio BaseCamp. “We are delighted to build a long-term relationship with Kyverna to optimize manufacturing processes and accelerate the development of their therapies,” Michael Paglia, the chief operating officer of ElevateBioBaseCamp, said in a statement. In related news, Kyverna also recently licensed Oxford Biomedica’s LentiVector platform.

7. Genprex’s Reqorsa Garners Orphan Drug Designation

Quaratusugene ozeplasmid (Reqorsa) is an investigational immunogene therapy being developed for the treatment of various indications, including non-small cell lung cancer and small cell lung cancer, as part of combination therapies. All 3 ongoing combination therapy clinical programs have received fast track designation from the FDA

8. Neogene Therapeutics Enlists Breakthrough Properties to Build Research Headquarters

The facility, dubbed Kavel F by Breakthrough, will be located on-site at the Amsterdam University Medical Center (UMC) and will aim to maintain several environmental sustainability standards. "As we continue our efforts to pioneer T-cell receptor therapies, we are excited to support our further growth in Amsterdam by developing a state-of-the-art R&D building together with Breakthrough Properties, Amsterdam UMC and the City of Amsterdam," Carsten Linnemann, PhD, the CEO and co-founder of Neogene Therapeutics, said in a statement.

9. Novo Nordisk to Install Cell Therapy Research Center in Technical University of Denmark

The company has put 127 million EUR towards the project, which will be referred to as The Novo Nordisk Foundation Cellerator upon its expected completion in 2027 and which may perform translational research for cell therapy products intended to address indications including Parkinson disease and chronic heart failure.

10. Seraxis Rakes in Additional Funding

Seraxis has closed out a second venture capital tranche, which included Frazier Life Sciences and Eli Lilly among its investors, bringing the cell therapy company’s total equity investment across the $50 million mark. Seraxis is developing SR-02, an islet organoid therapy intended to treat hypoglycemia.

11. Alsa Ventures Obtains Axovia Therapeutics With Intent to Bring Lead Gene Therapy Program to Clinical Trials

Axovia is developing AXV101, a preclinical AAV9 vector-based gene therapy intended to treat retinal dystrophy associated with Bardet-Biedl syndrome, which Alsa hopes to help bring into clinical trials by late 2024 or early 2025. “Our investment team was particularly attracted to Axovia Therapeutics as a valuable addition to our growing portfolio of companies because of its striking preclinical results, its Rare Pediatric Disease Designation, and the proven AAV delivery mechanism,” Alek Safarian, MBA, the CEO of Alsa Ventures, said in a statement.