Around the Helix: Cell and Gene Therapy Company Updates – September 7, 2022

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.

1. Tecartus Approved in Europe for R/R Acute Lymphoblastic Leukemia

Kite’s brexucabtagene autoleucel (KTE-X19; Tecartus), a CD19-directed autologous chimeric antigen receptor T-cell (CAR-T) therapy, has been granted a European Marketing Authorization (EMA) by the European Commission (EC) for the treatment of patients aged 26 years and older with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL).

2. IND Halted for T-Cell Malignancy Multiplex-Edited Cell Therapy

Beam Therapeutics has revealaed the FDA's request for additional preclinical data on BEAM-201, a CAR T-cell therapy for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T cell lymphoblastic lymphoma (T-LL).

3. Interim Trial Data Demonstrate MPS II Gene Therapy Safety and Efficacy

REGENXBIO’s RGX-121, an investigational gene therapy intended for the treatment of mucopolysaccharidosis Type 2 (MPS II; Hunter syndrome), has demonstrated promising safety and efficacy in interim data from the phase 1/2/3 CAMPSIITE trial (NCT03566043).

4. Cardiovascular Mesenchymal Cell Therapy Fast Tracked, Soon to Be Patented

Longeveron has received an intent-to-grant notice from the European Patent Office for its methods of using vascular biomarkers to monitor the efficacy of its Lomecel-B cell therapy. Lomecel-B is being investigated in several indications, including hypoplastic left heart syndrome (HLHS), for which the FDA has also just granted fast track designation.

5. Phase 3 Heart Failure Cell Therapy Trial Receives Go-Ahead from DSMB

Following a prespecified data review of the ongoing phase 3 clinical trial (NCT02438306) of BioCardia’s CardiAMP cell therapy, an investigational autologous cell therapy for the treatment of heart failure, an independent Data Safety Monitoring Board (DSMB) has given a positive review and recommendation to continue.

6. Regen Developing Novel CAR Approach for Solid Tumors

Regen Biopharma is developing a novel immunotherapy approach using CAR natural killer (NK), macrophage (M), and T cells (T) for treating solid tumors.

7. FDA Recognizes CRISPR Therapy for Treatment of Hereditary Angioedema

The FDA has granted orphan drug designation to Intellia Therapeutics’ investigational CRISPR therapy for treating hereditary angioedema (HAE), NTLA-2002.

8. First Patient Treated in Dry AMD iPSC Therapy Clinical Trial

The first patient has been treated in the National Institutes of Health’s (NIH) phase 1/2a clinical trial (NCT04339764) of an autologous induced pluripotent stem cell (iPSC) therapy for the treatment of dry age-related macular degeneration (AMD).

9. FDA Greenlights IND for MPS Type 1 Cell Therapy

The FDA has cleared an investigational new drug application (IND) for Immusoft’s ISP-001, an investigational engineered autologous B-cell therapy intended for the treatment of mucopolysaccharidosis type I (MPS I), allowing the company to initiate a phase 1 clinical trial.

10. Astrocyte Cell Therapy Well-Tolerated in Amyotrophic Lateral Sclerosis

An allogeneic human neural progenitor cell therapy transduced with glial cell line-derived neurotrophic factor (GDNF) and differentiated to astrocyte cells, termed CNS10-NPC-GDNF, was found to be well-tolerated and produced GDNF in participants with amyotrophic lateral sclerosis (ALS) in a phase 1/2a study (NCT02943850).

11. Charles River Laboratories, Cure AP-4 to Collaborate on Gene Therapy Manufacturing

Charles River Laboratories will manufacture High Quality plasmid DNA to be used in Cure AP-4’s phase 1/2 clinical trial of a gene therapy intended for the treatment of Adapter-Protein 4 Hereditary Spastic Paraplegia (AP-4 HSP).

12. Aresenal Biosciences to Take its Programmable Cell Therapy Programs to Clinical Development With $220 Million Provided in Series B Financing

The company expects that the financing will help expand its research activities and grow its pipeline, which currently includes lead program AB-1015, a candidate for the treatment of ovarian cancer, and candidates for kidney cancer and prostate cancer, among other indications.

13. US Patent and Trademark Office Issues Notice of Allowance for Enlivex's Application to Cover the use of Allocetra to Prevent CRS in Patients Receiving CAR-T

The patent is expected to be issued by the end of the first quarter of next year and it will provide protection though at least the end of 2036.

14. First Batch of CAR-T Cells for its Clinical Trials Released by CARsgen's CGMP Manufacturing Facility

The facility, located in Research Triangle Park, North Carolina, has a gross floor area of approximately 3,300 square meters is expected to be capable of producing CAR-T cell therapy products for approximately 700 patients each year.