Around the Helix: Cell and Gene Therapy Company Updates – September 6, 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. Abeona is Sending Off RDEB Cell Therapy For BLA Submission

Abeona Therapeutics has shared that it has had a positive pre-biologics license application (BLA) meeting with the FDA regarding its cell therapy EB-101 for patients with recessive dystrophic epidermolysis bullosa (RDEB) and is preparing for a BLA submission for the therapy.

2. Kyverna Makes Progress Evaluating Lupus Nephritis Cell Therapy in US, Germany

Kyverna Therapeutics has received regulatory clearance in Germany to initiate a phase 1/2 clinical trial of the KYV-101 chimeric antigen receptor (CAR) T-cell therapy in patients with lupus nephritis. The announcement follows Kyverna’s June update that its United States (US) phase 1/2 trial (NCT05938725) of KYV-101 for the same indication dosed its first patient with lupus nephritis.

3. Wave Life Sciences Submits CTA for Alpha-1 Antitrypsin Deficiency RNA Editing Therapy WVE-006

Wave Life Sciences has submitted a clinical trial application (CTA) for WVE-006, an investigational RNA editing oligonucleotide intended to treat alpha-1 antitrypsin deficiency (AATD). Although Wave Life Sciences did not indicate in which country the CTA was submitted, in response to a request for comment a representative for the company told CGTLive that the first locations for a potential clinical trial are expected to be in Australia and Europe.

4. Design Therapeutics' DT-216 Displays Positive Phase 1 Results in Friedreich Ataxia

Data from a phase 1, multiple-ascending dose study (NCT05285540) suggest DT-216, an investigational agent developed by Design Therapeutics, was generally well tolerated among patients with Friedreich ataxia, with statistically significant and dose-related increases in frataxin levels in skeletal muscle biopsies.

5. Epigenic Therapeutics Reels in $32 Million

The funds, resulting from a Series A round of financing that was led by Qiming Venture Partners and OrbiMed, will be put towards further development of existing programs and technology platforms, discovery activities, and growth of the company’s leadership. EPIREG, Epigenic Therapeutics’ proprietary technology platform, utilizes an epigenetic approach to modulating the expression of genes, and has undergone preclinical evaluations of delivery capabilities for indications including viral hepatitis, rare disease, and ocular disease.

6. Beam Therapeutics Doses First Patient in Base-Edited Allogeneic CAR-T Trial

BEAM-201, Beam Therapeutics’ antiCD7 CAR-T, is being evaluated in a phase 1/2 clinical trial (NCT05885464) for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma. BEAM-201 features base-edits that knockout expression of 4 different genes with the intention of improving efficacy and safety. “As the first patient dosed with a Beam therapeutic candidate and the first patient in the US to receive a base editing therapeutic, this represents a major milestone for the company, the scientists that made this possible, and the patients we hope to serve,” John Evans, MA, MBA, the chief executive officer of Beam, said in a statement.

7. First Patient in Europe Receives BioMarin’s Roctavian in Commercial Setting

The first patient was treated with the Hemophilia A gene therapy, recently approved for use in both the US and European Union, in Germany. “This one-time infusion represents an important milestone, offering new hope and potential, for eligible individuals in Germany," Robert Klamroth, MD, PhD, a treating physician and the chief physician of the Center for Hemophilia and Hemostaseology at the Vivantes Klinikum in Berlin, Germany, said in a statement.

8. University of Nottingham Spin-out TherageniX Snags £995,000 Grant for Development of Dry Powder Gene Therapy

The grant, from the UK’s innovation agency Innovate UK, will support development of TherageniX's gene therapy, which will first be evaluated for application during orthopedic surgery to enhance the repair of tissue; though, other indications may be evaluated later on. “We hope that our system will generate a platform of transformative, and economically viable approaches to clinical problems that remain poorly addressed in modern medicine,” James Dixon, PhD, an associate professor at the School of Pharmacy and National Institute for Health and Care Research Nottingham Biomedical Research Centre at University of Nottingham, who developed the TherageniX platform, said in a statement.

9. Eterna Therapeutics Tackling Gene Edits in iPSC Cell Line for Collaboration With Lineage

As part of a previously established exclusive option and license agreement with Lineage Cell Therapeutics, Eterna Therapeutics has begun development activities on a hypoimmune induced pluripotent stem cell (iPSC) line that the companies hope will ultimately be able to be applied in the treatment of central nervous system diseases. At the current stage, Eterna will be applying a set of specific gene edits that have been selected by Lineage, before passing off the cell line to Lineage for evaluation as a starting material for cell transplant.