Around the Helix: Cell and Gene Therapy Company Updates – October 12, 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. New, Multicenter Trial of MB-106 Doses First Patient

A phase 1/2 clinical trial (NCT05360238) has dosed its first patient with MB-106 (Mustang Bio), a chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas and chronic lymphocytic leukemia.

2. Fabry Disease Gene Therapy Clinical Trial Moves Onto Dosing Second Cohort

The phase 1/2 MARVEL-1 clinical trial (NCT04040049) of Freeline Therapeutics’ FLT190, an investigational adeno-associated virus vector-based gene therapy intended to treat Fabry disease, has dosed the first patient in its second cohort.

3. TIL Therapy to Be Assessed in Relapsed/Refractory Melanoma

The FDA has cleared Lyell Immunopharma’s investigational new drug application for LYL845, a tumor-infiltrating lymphocyte therapy to treat relapsed/refractory melanoma.

4. Allogeneic CAR-T Begins Phase 2 Clinical Trial

Allogene Therapeutics has initiated a phase 2 clinical trial (ALPHA2) for ALLO-501A, an investigational allogeneic anti-CD19 chimeric antigen T-cell (CAR-T) therapy, which will treat patients with relapsed/refractory large B-cell lymphoma.

5. Retinitis Pigmentosa Optogenetic Therapy Gets FDA Fast Track Designation

Nanoscope Therapeutics’ MCO-010, an investigational ambient-light activatable multi-characteristic opsin optogenetic monotherapy, has received fast track designation from the FDA for the treatment of retinitis pigmentosaB via intravitreal injection.

6. CardiAMP Cell Therapy Yields 100% Response Rate in Heart Failure

The CardiAMP cell therapy showed clinical improvements in patients with heart failure, according to 2-year data from a phase 3 trial (NCT02438306).

7. TCR-T Therapy Yields Clinical Responses Across Tumor Types

IMA203, Immatics’ PRAME-targeted ACTenginecell therapy, has demonstrated clinical responses as a monotherapy in multiple forms of solid tumors, according to new data from a phase 1 dose-escalation trial (NCT03686124).

8. Multiple Sclerosis Cell Therapy Demonstrates Promising Safety and Efficacy in Phase 2 Trial

BrainStorm Cell Therapeutics’ NurOwn, an investigational autologous mesenchymal stem cell neurotrophic factor–secreting cell therapy product, demonstrated encouraging preliminary safety and efficacy data in an open-label phase 2 clinical trial (NCT03799718) in patients with primary or secondary progressive multiple sclerosis, according to a report published in Multiple Sclerosis Journal.

9. Gene Therapy Shows Promise in Leber Congenital Amaurosis

Atsena Therapeutics’ ATSN-101 (previously referred to as SAR439483), an investigational adeno-associated virus vector-based gene therapy intended to treat Leber Congenital Amaurosis caused by biallelic mutations in GUCY2D (LCA1), has demonstrated promising safety and efficacy in a phase 1/2 clinical trial (NCT03920007), according to data presented at the American Academy of Ophthalmology Annual Meeting which took place from September 30 – October 1, 2022, in Chicago, Illinois.

10. Lineage Cell Therapeutics' Manufacturing Processes for Investigational Oligodendrocyte Progenitor Cell Therapy to Be Covered by US Patent

Lineage will also be receiving a patent from the European Patent Office for its retinal pigment epithelium cell manufacturing process.

11. Plasmid DNA Manufacturing Being Added to Ray Therapeutics and Forge Biologics AAV Manufacturing Partnership

The addition will support Ray Therapeutics' RTx-015, an investigational gene therapy for retinitis pigmentosa.

12. ONI, University of Pennslyvania to Collaborate on Biomarker Research Related to CAR-T Safety and Efficacy

The collaboration will utilize ONI's imaging technology and will proceed with the aims of better understanding of CAR-T treatments and gathering information potentially useful for the development of future CAR-T therapies.

13. CD19 Binder the Subject of New License Agreement Between IASO Biotherapeutics, Cabaletta Bio

Cabaletta Bio intends to use IASO's clinically validated CD19 binder in its investigational CD-19 targeted CAR-T therapy CABA-201.

14. Patent Assignment Signed Between CLS Therapeutics, Xenetic Biosciences

The patent assignment from CLS to Xenetic is related to a collaboration between the 2 companies and VolitionRx Limited focused on using Volition's Nu.Q technology and Xenetic's DNase-Armored CAR-T platform to develop new cell therapy treatments for solid tumors.

15. Ochre Bio's $30 Million in Series A Financing to be put Toward Development of RNA Therapies for Liver Disease

Ochre intends to use the funds to build off previous research with the goal of developing new investigational RNA-based therapies, which they intend to carry out preclinical studies with in their recently launched "Liver ICUs".