Around the Helix: Cell and Gene Therapy Company Updates – April 5, 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. Vertex, CRISPR Complete Rolling BLAs for Exa-Cel in Sickle Cell Disease and Transfusion-dependent Beta Thalassemia

Vertex Pharmaceuticals and CRISPR Therapeutics today announced that they had completed the rolling submission of their biologics license applications (BLAs) for the investigational therapy exagamglogene autotemcel (exa-cel; previously known as CTX001) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

2. Lovo-cel BLA Submission Likely Delayed for Sickle Cell

bluebird bio’s biologics license application (BLA) submission for lovotibeglogene autotemcel (lovo-cel) to treat sickle cell disease (SCD) will likely be delayed as the company awaits FDA feedback on newly submitted manufacturing compatibility data.

3. Caribou Biosciences Provides Updates on Allogeneic CAR-T Clinical Trials

Caribou Biosciences has announced that the first patient has been dosed in its phase 1 CaMMouflage clinical trial (NCT05722418) evaluating CB-011 for the treatment of relapsed/refractory (r/r) multiple myeloma (MM) and that it has initiated the dose expansion portion of its phase ANTLER clinical trial (NCT04637763) evaluating CB-010 for the treatment of r/r B-cell non-Hodgkin lymphoma (B-NHL).

4. First Patient Dosed in Phase 1/1b Trial of PRGN-3007 CAR-T Cell Therapy in Solid Tumors, Hematologic Malignancies

The first patient has been dosed in the phase 1/1b dose-escalation and -expansion study (NCT05694364) of PRGN-3007, Precigen’s investigational and first-in-class multigenic, autologous CAR-T cell therapy, according to a company announcement.

5. BlueRock Therapeutics Partners With Rune Labs and Emerald Innovations for Parkinson Disease Monitoring

The partnership is focused on the use of wearable technology and invisible contactless digital health technology for disease monitoring. BlueRock may use the technology for monitoring in future clinical trials of its Parkinson disease cell therapy BRT-DA01.

6. Sartorius Will Acquire Polyplus in ~2.4 Billion Euro Agreement

Polyplus specializes in the manufacture of DNA and RNA delivery reagents and plasmid DNA.

7. Ocular Gene Therapy Company Vedere Bio II Announces Discontinuation of Operations

The company cited the results of preclinical efficacy research as the key factor in its decision.

8. Alaunos Therapeutics Hands Precigen Exclusive Rights to UltraCAR-T Platform Targets

The targets include CD19 and B-cell maturation antigen. The new agreement also gives Precigen exclusive rights to its IL-12 gene therapy.

9. Forcefield Therapeutics Passes Gene Therapy Development and Commercialization Rights to Freeline Therapeutics

The agreement specifically regards AAV gene therapies for certain cardiac conditions targeted at genes identified by Forcefield Therapeutics.

10. BioCardia Obtains Patents Related to Cell Therapy Delivery Tech

The patents include a US patent for a “Multi-Directional Steerable Catheter” and an India patent for "Steerable Endoluminal Devices and Methods for Use”.

11. AGC Biologics to Evaluate Idiopathic Pulmonary Fibrosis Exosome-based Therapy in Feasibility Study

AGC obtained the investigational product in a technology transfer agreement from the Jikei University in Japan.

12. Freeline Therapeutics Pauses Development of its Fabry Disease Gene Therapy

The company stated that it is now focusing its resources on Gaucher disease gene therapy FLT201.