Around the Helix: Cell and Gene Therapy Company Updates – October 5, 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. Allogeneic CAR T Recognized for Cutaneous T-Cell Lymphomas

The FDA has granted regenerative medicine advanced therapy (RMAT) designation to CRISPR Therapeutics' allogeneic chimeric antigen receptor (CAR) T-cell therapy for the treatment of CTX130 cutaneous T-cell lymphomas (CTCL), specifically for the treatment of mycosis fungoides and Sézary syndrome (MF/SS).

2. Cellenkos to Try T Regulatory Cell Approach in ALS

The FDA has cleared Cellenkos’ investigational new drug application (IND) for CK0803, a T regulatory (Treg) cell therapy for the potential treatment of amyotrophic lateral sclerosis (ALS).

3. Sarepta’s DMD Gene Therapy Goes In Front of FDA With BLA Submission

Sarepta Therapeutics has submitted its biologics license application (BLA) for SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy developed in collaboration with Roche, for the potential treatment of ambulant patients with Duchenne muscular dystrophy (DMD).

4. KIR-CAR T-Cell Therapy Recognized for Mesothelioma Treatment

The FDA has granted orphan drug designation to Verismo Therapeutics’ SynKIR-110, a killer immunoglobulin-like receptor - chimeric antigen receptor (KIR-CAR) T-cell therapy, for the treatment of mesothelin-expressing mesothelioma.

5. Lineage Cell Therapeutics Expands Manufacturing, R&D Facilities

With positive data continuing to roll in from its RG6501 (OpRegen) platform, Lineage Cell Therapeutics is investing in an expansion of its GMP manufacturing facility in Israel and opening a new R&D facility in California to support new cell therapy ventures in blindness and hearing loss.

6. GPRC5D-Targeted CAR-T Shows Promise in Heavily Pre-treated Multiple Myeloma

MCARH109, a chimeric antigen receptor (CAR) T-cell therapy targeted at G protein–coupled receptor, class C, group 5, member D (GPRC5D), yielded responses and demonstrated that GPRC5D is an active immunotherapeutic target in a phase 1 clinical trial (NCT04555551) for patients with heavily pretreated multiple myeloma (MM) carried out at Memorial Sloan Kettering Cancer Center (MSKCC), according to results published in The New England Journal of Medicine.

7. Modified Immune Cell Therapy Induces Kidney-Donor-Specific Immunosuppression

MIC-Lx cell therapy (TolerogenixX) induced long-term donor-specific immunosuppression and yielded a significant increase in regulatory B lymphocytes in kidney transplant recipients, according to 3-year follow-up data from a phase 1 study (NCT02560220).

8. BioMarin Resubmits BLA for Hemophilia A Gene Therapy Val-Rox

BioMarin has resubmitted the biologics license application (BLA) to the FDA for valoctocogene roxaparvovec (val-rox; BMN-270), an adeno-associated virus (AAV)-based gene therapy intended to treat hemophilia A.

9. Omidubicel Improves Survival in Hematologic Malignancies With HSCT

The umbilical cord blood (UCB) cell therapy omidubicel (NiCord; Gamida Cell) improved survival in patients with hematological malignancies receiving allogeneic hematopoietic stem cell transplantation (HSCT).

10. Allogeneic Anti-CD7 CAR-T Demonstrates Promising Results in Hematological Malignancies

Bioheng Biotech’s RD13-01, an investigational allogeneic CD7-targeted chimeric antigen receptor T-cell (CAR-T) product with genetic modifications to resist fratricide, graft versus host disease (GvHD), and rejection, demonstrated promising safety and efficacy data in a phase 1 clinical trial (NCT04538599) for patients with CD7+ hematological malignancies, according to results published in Cell Research.

11. Wet AMD Gene Therapy Reduces Anti-VEGF Treatment Burden

RGX-314, REGENXBIO’s investigational gene therapy for wet age-related macular degeneration (AMD), was well-tolerated and reduced treatment burden in participants treated in the phase 2 AAVIATE trial (NCT04514653).

12. Solid Bio Adds Friedreich Ataxia to Pipeline With Aavanti Acquisition

Solid Biosciences has acquired AavantiBio, a company whose lead program, AVB-202, is a gene therapy candidate in preclinical studies for Friedreich’s ataxia.

13. Gene Therapy Yields Improvements in Danon Disease

The gene therapy RP-A501 (Rocket Pharmaceuticals) was well-tolerated and yielded functional improvements in adult and pediatric patients with Danon disease, according to data from a phase 1 trial (NCT03882437) presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022.

14. Gene Therapy Trial Makes Headway in Canavan Disease

A phase 1/2 clinical trial (NCT04833907) of Myrtelle’s rAAV-Olig001-ASPA (MYR-101), an investigational recombinant adeno-associated virus (rAAV) vector-based gene therapy intended to treat Canavan disease (CD), has completed dosing of the first 8 patients.

15. B-NHL Cell Therapy Cleared for Trials

The FDA has granted IND clearance for a phase 1/2 dose escalation study of Oncternal Therapeutics’ chimeric antigen receptor (CAR) T therapy ONCT-808 for treating aggressive B cell non-Hodgkin’s lymphoma (B NHL).

16. Frontera Therapeutics' FT-001 IND Cleared by China's Center for Drug Evaluation

An IND for FT-001, an investigational gene therapy intended to treat inherited retinal degenerations, was previously cleared by the US FDA earlier this year.

17. Krystal Biotech's Dystrophic Epidermolysis Bullosa Gene Therapy Gets Positive Opinion on Pediatric Investigation Plan from the EMA's PDCO

The European Medicines Agency (EMA) Pediatric Committee (PDCO)'s positive opinion clears the way for a European Marketing Authorization Application (MAA) for beremagene geperpavec (B-VEC), which is expected in late 2022.

18. New Bill Could Expand Access to Blood and Blood-derived Biomaterial Storage Services for Americans With Flexible Spending and Healthcare Savings Accounts

The "Access to Future Cures Act" (H.R.8912) bill, which was introduced to Congress in September 2022, intends to give Americans with Health Savings Accounts (HSA), Health Reimbursement Arrangements (HRA), and Flexible Spending Accounts (FSA) additional options in light of the rapidly expanding field of approved cell and gene therapies.

19. Circularis Acquired by Ginkgo Bioworks

Ginkgo Bioworks' plans include the use of Circularis' promoter screening platform in an attempt to identify promoters that modulate expression in tumor micro-environments for the purpose of cell therapy development.

20. BCMA-targeted Cell Therapy CARVYKTI Approved in Japan for R/R Multiple Myeloma

The CARVYKTI (ciltacabtagene autoleucel) approval is restricted to patients who have no history of treatment with CAR-positive T-cell infusion therapy targeting BCMA, who have peviously received therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody, and who are relapsed/refractory (r/r) to their most recent therapy.

21. Real Endpoints Marketplace, Bluebird Bio Partner to Enable Additional Patient Access to Zynteglo

The collaboration will provide options for an outcomes-based agreement for ZYNTEGLO to plans that cover almost 16 million Americans.

22. Pfizer Licenses Next-Generation AAV Capsid from Voyager Therapeutics

Pfizer intends to utilize the capsid in its development of an investigational gene therapy for an undisclosed rare neurologic disease. The licensing is part of an agreement announced late last year.