Around the Helix: Cell and Gene Therapy Company Updates – September 14, 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. Data From Various Clinical Studies Presented at ESMO 2022

Catch up on CGTLive's coverage of the European Society for Medical Oncology (ESMO) 2022 Congress, September 9-13, 2022 in Paris, France.

2. Ornithine Transcarbamylase Gene Editing Program Nabs Another FDA Designation

The FDA has granted orphan drug designation to iECURE’s gene editing therapy GTP-506 for the potential treatment of ornithine transcarbamylase (OTC) deficiency.

3. Polycythemia Vera mRNA Therapy Receives FDA Fast Track Designation

Silence Therapeutics’ SLN124, an investigational siRNA therapy intended for the treatment of rare hematological diseases, has been granted fast track designation by the FDA for the treatment of polycythemia vera (PV).

4. Ide-Cel to Be Evaluated in Newly Diagnosed Multiple Myeloma

2seventy bio’s idecabtagene vicleucel (ide-cel; Abecma), a B-cell maturation antigen-directed chimeric antigen receptor T-cell therapy which was previously approved by the FDA for adults with relapsed or refractory multiple myeloma, will be tested in a new clinical trial (KarMMa-9) for patients with newly diagnosed MM who have a suboptimal response to transplant.

5. Potential Gene Therapy for Leber Congenital Amaurosis Identified in NIH Study

Retinal organoid disease models derived from patients with Leber congenital amaurosis (LCA) caused by a mutation in the NPHP5 gene (NPHP5-LCA) had cilia defects rescued by an adeno-associated virus (AAV)-mediated NPHP5 gene augmentation therapy, indicating a potential treatment for NPHP5-LCA, according to preclinical research published in Stem Cell Reports.

6. BioMarin to Investigate B-ALL Case in Val-Rox Gene Therapy Clinical Trial

BioMarin revealed in an SEC filing that it is investigating a case of b-cell acute lymphoblastic leukemia (B-ALL) in a patient who received its investigational gene therapy for hemophilia, valoctocogene roxaparvovec (val-rox; BMN-270).

7. Gene Therapy for OTOF-Mediated Hearing Loss Gets Green Light to Enter the Clinic

The FDA has cleared an investigational new drug (IND) application for Akouos’ AK-OTOF, an investigational dual AAV vector-based gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss, clearing the way for a clinical trial to begin.

8. EMA Grants Advanced Therapy Medicinal Product Classification to Myrtelle’s Gene Therapy for Canavan Disease

rAAV-Olig001-ASPA previously received orphan drug designation, rare pediatric disease designation, and fast track designation from the FDA.

9. AnGes' HGF Gene Therapy Clinical Trial in Rest Pain Discontinued

AnGes cited failure to meet its primary end points for rest pain in the phase 3 clinical trial as a reason for the cancellation.

10. Sensorion's Gene Therapy for OTOF-Mediated Hearing Loss Gets Positive Opinion for EMA Orphan Drug Designation

The European Commission intends to announce its decision within 30 days.

11. CSL's Haemophilia B Gene Therapy Receives Orphan Drug Designation From the TGA

CSL aditionally announced intention to submit a market authorization application to the Therapeutics Goods Administration (TGA) for etranacogene dezaparvovec.

12. GenSight Biologics Adds 2 New Senior Executives

Philippe Motté and Michiel Hemels will help support the company's launch of LUMEVOQ in Europe.

13. Forge Biologics Raises $90 Million in Series C Financing

The gene therapy-focused contract development and manufacturing organization intends to use the funding for the expansion of client offerings and the addition of services to its end-to-end manufacturing platform.

14. Stargardt Disease Gene Therapy Clinical Trial Completes Enrollment

Nanoscope Therapeutics expects 6-month data from the phase 2 clinical trial of MCO-010 in the first half of next year.

15. Pre-clinical Studies for Regen BioPharma's CAR-T Cell De-differentiation Approach Initiated

Regen BioPharma is working with ProMab Biotechnologies to validate the therapy using shRNA NR2F6-inhibiting technology.

16. Draft Evidence Report Covering Etranacogene dezaparvovec and Valoctocogene roxaparvovec Released By ICER

The Institute for Clinical and Economic Review (ICER)'s draft assesses the clinical efficacy of CSL's and BioMarin's gene therapies.

17. Coave Therapetuics, Institute of Neurodegenerative Diseases of Bordeaux to Collaborate on Gene Therapies for Neurodegenerative Disorders

The collaboration will focus on protein degradation and will aim to develop therapies for the treatment of alpha-synucleinopathies.

18. Oxford Biomedica Solutions and Undisclosed US-based Biotechnology Company Sign Agreement for AAV Platform

Oxford Biomedica Solutions will provide the partner with access to its process and manufacturing platform.

19. New Cell Engineering-focused Biotechnology Company Capstan Therapeutics Launches

The company launched with $165 million in financing and intends to initially focus efforts on developing CAR therapies for diseases with no effective treatments.