Around the Helix: Cell and Gene Therapy Company Updates - February 9, 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. GeneTherapyLive’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. Study to Evaluate Gene-Edited Cell Therapy for MPSII

A phase 1/2 study will evaluate a gene-edited cell therapy (LV-HSCGT) for the potential treatment of neuronopathic mucopolysaccharidosis type 2 (nMPSII, Hunter syndrome). The therapy consists of cryopreserved autologous CD34+ hematopoietic stem cells, transduced ex vivo with CD11b lentiviral vector, in order to produce functional human iduronate-2-sulphatase (IDS) ApoEII lacking in MPSII.

2. Gene Therapies for Mucopolysaccharidosis Show Safety, Efficacy

REGENXBIO’s investigational gene therapy candidates RGX-121 and RGX-111 have demonstrated interim efficacy and safety in their first in-human studies in severe mucopolysaccharidosis type 2 (MPSII) and type 1 (MPSI), respectively, in data presented at the 18th Annual WORLDSymposium.

3. Beti-Cel Yields Durable Transfusion Independence in β-Thalassemia

Betibeglogeneautotemcel (beti-cel; LentiGlobin; bluebird bio) gene therapy yielded transfusion independence in most treated adult and pediatric patients with transfusion-dependent β-thalassemia and a non–β0/β0 genotype, according to results from a phase 3 study (HGB-207; NCT02906202) published in the New England Journal of Medicine.

4. Etranacogene Dezaparvovec Superior to FIX Prophylaxis in Hemophilia B

Etranacogenedezaparvovec (EtranaDez, AMT-061; CSL Behring) is superior to Factor IX (FIX) prophylactic therapy in reducing annualized bleeding rate (ABR) in patients with hemophilia B, meeting the primary endpoint of the phase 3 HOPE-B study (NCT03569891).

5. COVID-19 Cell Therapy Trial Continues Dosing

The second, higher-dose cohort has finished dosing in the phase 1 study (NCT04765449) of TVGN-489, Tevogen Bio’s investigational allogeneic SARS-CoV-2 specific CD8+ T-cell therapy. No dose-limiting toxicities or treatment-emergent adverse events have been reported so far.

6. Stargardt Disease Gene Therapy IND Cleared to Proceed

Nanoscope Therapeutics has received FDA approval for its investigational new drug (IND) application for MCO-010, a gene therapy for the potential treatment of Stargardt disease, a type of macular degeneration. The company plans to initiate a phase 2 trial in the first half of 2022.

7. NexImmune and Rutgers Collaborating in Neuroendocrine Tumors

NexImmune and Rutgers, The State University of New Jersey, are collaborating to discover immune checkpoint proteins in neuroendocrine tumors and other cancers and develop novel, targeted therapies for them.Steven K. Libutti, MD, FACS, director, Rutgers Cancer Institute of New Jersey will serve as principal investigator.

8. Orchard UK Agreement Opens Access to MLD Cell Therapy

Orchard Therapeutics has reached an agreement with England’s National Health Service to allow access to atidarsagene autotemcel (Libmeldy) to children with metachromatic leukodystrophy in England and Wales.

9. Medigene’s Cell Therapy Shows Preliminary Efficacy in Hematologic Malignancies

Nine patients have been dosed with the TCR-T cell therapy MDG1011 in a phase 1/2 study (NCT03503968). Most patients had detectable TCR T-cells and decreased PRAME levelsin follow-up. Promising disease stabilization was observed.

10. Immune Cell Engineering Shows Preclinical Efficacy

SQZ Biotechnologies is researching engineering multiple immune cell types to drive major histocompatibility class I antigen presentation for the potential treatment of multiple diseases. A Journal of Immunology publication revealed preliminary success.

11. MCO-010 Trial Completes Enrollment in Retinitis Pigmentosa

Nanoscope Therapeutics has completed enrollment in the phase 2b RESTORE trial (NCT04945772). The trial has enrolled 27 participants with severe vision loss due to RP. Topline results are expected in the first quarter of 2023.

12. Fabry Disease Gene Therapy Demonstrates Preliminary Efficacy

Sangamo Therapeutics’ isaralgagenecivaparvovec (ST-920) has been well-tolerated in 5 treated patients across 5 cohorts. α-Gal A levels have increased from baseline and elevated activity has been maintained for up to 1 year of follow-up.

13. GM1 Gangliosidosis Gene Therapy Trial Doses Third Patient

The third patient has been dosed in the safety cohort of Lysogene’s phase 1/2 trial (NCT04273269) of their gene therapy LYS-GM101 for the potential treatment of GM1 gangliosidosis. The fourth patient is currently being enrolled, after which enrollment in the efficacy cohort will commence.

14. Gene Therapy Well Tolerated in Pompe Disease

Astellas Pharma announced positive safety data from the phase 1/2 FORTIS study (NCT04174105) of their gene therapy AT845 in late-onset Pompe disease. The therapy has been well-tolerated so far, with no serious adverse events.

15. T-Cell Therapy Gets Orphan Drug Designation for Hepatocellular Carcinoma

Eureka Therapeutics’ ARTEMIS T-cell therapies, ET140203 and ECT204, have received orphan drug designation for the treatment of hepatocellular carcinoma. ET140203 is being evaluated in the ARYA-1 (NCT04502082) and ARYA-2 (NCT04634357)studies while ECT204 is being evaluated in the ARYA-3 study (NCT04864054).

16. First Patients Dosed in European Trial of Huntington Disease Gene Therapy

uniQure has dosed the first 2 patients in a phase 1/2 trial of AMT-130, their investigational gene therapy for Huntington disease. A similar trial is ongoing in the US (NCT04120493) and topline data from that trial is expected in the second quarter of 2022.

17. 4D Announces Positive Interim Gene Therapy Data for Fabry Disease

The first 3 patients with Fabry disease dosed with 4D Molecular Therapeutics’ 4D-310 have exhibited mean serum AGA enzyme activity above or within the normal range. Promising trends have been observed in cardiac endpoints and quality of life. The therapy has been well-tolerated in all 3 patients.