Around the Helix: Cell and Gene Therapy Company Updates – February 22, 2023

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 Clinical Data Presented at Tandem 2023

Catch up on CGTLive's coverage of the 2023 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in Orlando, Florida, February 15-19, 2023.

2. Hemgenix Approved in EU for Hemophilia B

Etranacogene dezaparvovec (Hemgenix; CSL Behring) has been approved by the European Commission (EC) for the treatment of severe and moderately severe hemophilia B in adults without a history of Factor IX inhibitors.

3. HHS, CMS Working to Improve Access to Cell and Gene Therapies, Decrease Costs

The Secretary of the Department of Health and Human Services (HHS) has selected 3 new models by the Centers for Medicaid & Medicare Services for testing, 2 of which directly affect cell and gene therapies, to help lower costs and improve access to novel therapies.

4. Cell Therapy Decreases Viral Load in Patients With BK Viremia After Kidney Transplant

Posoleucel (AlloVir) cell therapy has shown efficacy in treating patients with BK viremia who have received a kidney transplant in new data from a phase 2 trial (NCT04605484).

5. Athersys to Meet With the FDA in March Regarding Potential Changes to Ischemic Stroke Cell Therapy Trial

The Type B meeting will be focused on proposed changes to the ongoing phase 3 MASTERS-2 clinical trial (NCT03545607), which is evaluating MultiStem (invimestrocel).

6. GAIA BioMedicine to Utilize TreeFrog's C-Stem Technology for NK-like Immune Cell Expansion

The collaboration is focused on the development of allogeneic therapies for solid tumors.

7. Lineage Cell to Evaluate Eterna's iPSC Lines

The exclusive option and license agreement is focused on the potential use of novel beta 2 microglobulin (B2M)-deficient induced pluripotent stem cell (iPSC) lines, which will be developed by Eterna Therapeutics, in therapies directed at central nervous system indications.

8. Nexcella Secures Manufacturer for Multiple Myeloma CAR-T

Nexcella has entered into a GMP manufacturing agreement with an undisclosed US manufacturer for the production of NXC-201, in preparation for submission an investigational new drug application to the FDA.

9. Enlivex Receives Israeli Patent for its Macrophage Reprogramming Cell Therapy

The patent covers the use of Allocetra in the prevention or treatment of cytokine release syndrome caused by CAR-T therapy, infection, and other sources.

10. Aera Therapeutics Announces Focus on Novel Gene Therapy Delivery Platform

Aera Therapeutics launched in February 2023 with the support of $193 million raised across Series A and Series B financing rounds.

11. CMT Research Foundation, Nanite to Develop Method to Deliver Gene Therapy to Schwann Cells

Nanite will leverage its SAYER gene delivery platform with the goal of developing a delivery vehicle capable of efficiently passing through the blood-nerve barrier.