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.

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.

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).

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

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.

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.

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

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.

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.

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.

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

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