Around the Helix: Cell and Gene Therapy Company Updates – March 16, 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. Adrenomyeloneuropathy Gene Therapy Gets Orphan Drug Designation

The FDA has granted orphan drug designation to SwanBio Therapeutics’ investigational adeno-associated virus vector-based gene therapy SBT101 for the potential treatment of adrenomyeloneuropathy.

2. First Patient Dosed in T-Cell Malignancy CAR T-Cell Therapy Trial

The first patient has been dosed in the phase 1/2 trial (NCT04984356) of Wugen’s WU-CART-007, which is being explored for the potential treatment of relapsed/refractory T-cell acute lymphoblastic leukemia or lymphoblastic lymphoma.

3. FDA Recognizes Canavan Disease Gene Therapy With Multiple Designations

The FDA has granted fast track, rare pediatric disease, and orphan drug designations to Myrtelle’s gene therapy rAAV-Olig001-ASPA for the treatment of Canavan disease. The gene therapy is currently being evaluated in a phase 1/2 first in-human clinical trial (NCT04833907) for the rare disease.

4. Presymptomatic SMA Treatment With Zolgensma Associated With Normal Development

Pediatric patients with spinal muscular atrophy treated presymptomatically with onasemnogene abeparvovec (Zolgensma; Novartis) continue to demonstrate age-appropriate development, according to new data from the phase 3 SPR1NT study (NCT03505099).

5. Selecta’s Methylmalonic Acidemia Gene Therapy Program Resumes

The FDA has lifted their clinical hold on the gene therapy candidate SEL-302, which consists of the gene therapy MMA-01 in combination with Selecta’s ImmTOR nanoparticles. The FDA had placed the program on clinical hold in November 2021 due to questions on chemistry, manufacturing, and controls.

6. Intellia’s CRISPR-Engineered Cell Therapy Receives Orphan Drug Designation for Acute Myeloid Leukemia

NTLA-5001 is a T-cell receptor (TCR) T-cell therapy as well as Intellia’s first ex vivo CRISPR therapy. It is currently being evaluated in a phase 1/2a study (NCT05066165) in adults with persistent or recurrent AML. Intellia announced earlier in March that the first patient had been dosed in the study.

7. Novartis to Manufacture Carisma CAR-M Therapy for Solid Tumors

Novartis will manufacture Carisma’s chimeric antigen receptor macrophage therapy, CT-0508, for the potential treatment of solid tumors, beginning in 2023.

8. DMD Cell Therapy Continues to Show Efficacy

Capricor Therapeutics reported updated, 1-year data from the phase 2 HOPE-2 trial (NCT03406780) that demonstrated continued improvements in skeletal and cardiac measurements as evaluated on Performance of the Upper Limb scores after 4 doses of CAP-1002 over the course of 1 year.

9. Mesenchymal Stem Cell Therapy IND Cleared for Crohn Disease

Ossium Health’s investigational new drug application has been cleared for OSSM-001 for the potential treatment of refractory perianal fistulas in patients with Crohn disease. Ossium plans to initiate a multi-center phase 1 clinical trial by the end of 2022.

10. LLU Cancer Center Launches Division of Transplant and Cellular Therapy

Loma Linda University Cancer Center launched the new division to enhance treatment options and improve accessibility to blood and marrow transplant and novel cell and gene therapies.

11. Helixmith Plasmid DNA Therapy Moves Forward With New Formulation

VM202 (Engensis) will now be developed with a new formulation and freeze-drying process for future clinical trials in diabetic peripheral neuropathy. Helixmith has demonstrated equivalence to the previous therapy formulation.

12. Phase 3 OVAL Trial Completes Enrollment

VBL Therapeutics’ trial (NCT03398655) of the gene therapy ofranergene obadevovec (ofra-vec, VB-111) has completed enrollment for patients with platinum-resistant ovarian cancer. The trial has enrolled 409 patients throughout the United States, Europe, Israel, and Japan.

13. Phase 1/2 B-LIEVE Trial Doses First Patient With Hemophilia B

The first patient with hemophilia B has been dosed in Freeline Therapeutics’ trial (NCT05164471) of the gene therapy FLT180a. Initial safety and biomarker data is expected in the first half of 2022.