Around the Helix: Cell and Gene Therapy Company Updates – March 8, 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. FDA Extends Val-Rox PDUFA Date in Hemophilia A

Following the submission of a 3-year data analysis from the ongoing GENEr8-1 clinical trial (NCT03370913), the FDA has informed Biomarin that it has extended the review of valoctocogene roxaparvovec (val-rox; Roctavian) for the treatment of severe hemophilia A.

2. Sangamo Joins Companies Halting Sickle Cell Gene-edited Cell Therapy Programs

Sangamo Therapeutics has announced its decision to cease further material investments in the development of BIVV003, an investigational zinc finger nuclease gene-edited cell therapy intended to treat sickle cell disease (SCD), beyond those necessary to complete the ongoing phase 1/2 PRECIZN-1 clinical trial (NCT03653247).

3. Patient With Parkinson Disease to Receive Compassionate Use of NK Cell Therapy

The FDA has approved NKGen Biotech’s natural killer cell (NK) therapy SNK01 for compassionate use in treating a patient with Parkinson disease (PD).

4. Sarepta May Have a Smooth Road to DMD Gene Therapy Approval

Sarepta’s road to approval for treating Duchenne muscular dystrophy (DMD) with its gene therapy delandistrogene moxeparvovec (SRP-9001) may be smoothed out after the company revealed that the FDA would not be requiring an advisory committee meeting prior to the PDUFA date of May 29, 2023.

5. Two New Grants for Rosalind Franklin University Investigators Related to New Therapeutic Approaches for HIV-related Cancers and Brain Repair

One grant will fund research into treatments for Kaposi Sarcoma-Associated Herpes Virus, while the other will cover research focused on reprogramming non-neuronal glial cells to replace lost neurons.

6. Adptimmune Therapeutics, TCR2 Therapeutics Announce Merger

The newly combined company will pursue development of cell therapies for the treatment of solid tumors.

7. BioLineRx, Washington University School of Medicine Evaluating Motixafortide's Ability to Address Unmet Needs in Patients Receiving Sickle Cell Disease Gene Therapies

Motixafortide will be evaluated in a clinical trial, both alone and in combination with natalizumab, for its ability to mobilize CD34+ hematopoietic stem cells for SCD gene therapies.

8. Danaher, University of Pennsylvania's Center for Cellular Immunotherapies Announce Collaboration to Address Cell Therapy Manufacturing Bottlenecks

The partners will seek to address challenges in scaling up the manufacture of cell therapies, with areas of focus including yield and the consistency of the product.

9. Novartis Licenses Novel Capsids from Voyager Therapeutics

The AAV capsids will be utilized in Novartis gene therapy candidates intended to treat neurological diseases. The licenses specifically cover use in 2 undisclosed indications only.

10. Porton Advance to Provide CDMO Services to DanausGT for Its Investigational Cell and Gene Therapies

DanausGT's pipeline includes investigational treatments for blood diseases, skin diseases, and liver-related diseases.

11. Chroma Medicine Raises $135 Million for its Epigenetic Editing Platform

The successful round of Series B financing will also help fund the company's programs.