Around the Helix: Cell and Gene Therapy Company Updates – May 29, 2024

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. Anixa Biosciences Makes Progress in the Clinical Evaluation of CER-T Approach to Treating Ovarian Cancer

Anixa Biosciences has dosed the fifth patient in its phase 1 clinical trial (NCT05316129) evaluating its novel follicle stimulating hormone receptor-targeting chimeric endocrine receptor (CER) T-cell therapy for the treatment of recurrent ovarian cancer.

2. Wugen Garners New FDA and EMA Regulatory Designations for T-Cell Malignancy CAR-T WU-CART-007

Wugen’s WU-CART-007, an investigational allogeneic CD7-directed chimeric antigen receptor T-cell (CAR-T) therapy intended to treat relapsed/refractory T-cell acute lymphoblastic leukemia and r/r T-cell lymphoblastic lymphoma, has been granted regenerative medicine advanced therapy designation by the FDA and priority medicines designation by the European Medicines Agency.

3. Cabaletta Bio’s CAR-T CABA-201 Shows Safety for First Patients Dosed in Myositis and Lupus Trials

CABA-201 has also received clearance from the FDA for a phase 1/2 clinical trial (RESET-MG; NCT06359041) in generalized myasthenia gravis and phase 1/2 clinical trial (RESET-SSc; NCT06328777) in systemic sclerosis (SSc). Cabaletta Bio expects to report initial clinical data from these 2 trials in the second half of this year.

4. Aurion Biotech Evaluating Combination Cell Therapy Product AURN001 as Alternative to Corneal Transplant in Phase 1/2 ABA-1, CLARA Clinical Trial

In honor of Healthy Vision Month, observed annually in the month of May by the patient and clinician communities, CGTLive took a closer look at the clinical trial design for this novel treatment.

5. US District Judge Rejects Ultragenyx’s Motion to Dismiss Case Put Forth by Henrietta Lacks Estate

A lawsuit filed on behalf of the estate of Henrietta Lacks against gene therapy company Ultragenyx over the use of the HeLa cell line in the production of its adeno-associated virus vector-based gene therapies may now go forward, after United States District Judge Deborah Boardman rejected the company’s motion to dismiss the case. "We applaud Judge Boardman's historic ruling, which allows our unjust enrichment claims to proceed and acknowledges the deep injustices that Henrietta Lacks and her family have endured," Christopher Seeger and Christopher Ayers of Seeger Weiss, the estate's attorneys, said in a statement.

6. Orna Therapeutics Brings ReNAgade Therapeutics Into the Fold

Orna Therapeutics, a company focused on development of circular RNA therapies, has acquired ReNAgade Therapeutics, a company developing lipid nanoparticle-based technology for delivery of RNA to cells outside the liver. “The combination of technologies positions Orna to advance best-in-class panCAR in vivo CAR RNA therapies and expand existing gene editing delivery solutions with circular RNA to address the massive unmet need in multiple diseases,” Tom Barnes, PhD, founding CEO of Orna, said in a statement.

7. Merck KGaA to Purchase Mirus Bio

Merck KGaA and Mirus Bio, a company focused on the development of transfection reagents, have entered a definitive agreement for the former company to purchase the latter at a price of 600 million US dollars. Merck KGaA is making the purchase with the intent of eventually being able to provide goods and services necessary for every step of viral vector production.

8. Radar Therapeutics Secures $13.4 Million in Seed Financing

New mRNA technology focused company Radar Therapeutics has launched with an oversubscribed round of seed financing that included investment from Eli Lilly and Company, Biovision Ventures, and others. Radar’s platform incorporates the use of RNA sensors that gate mRNA expression,with the intent of achieving targeted and timed delivery based on other RNA in a given cell.