Around the Helix: Cell and Gene Therapy Company Updates – January 11, 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. Intellia to Advance Development of ATTR Amyloidosis, Angioedema, AATD Gene Therapies

Intellia Therapeutics has announced its intention to initiate global pivotal trials of its CRISPR-based therapies NTLA-2001 for the treatment of transthyretin amyloidosis and NTLA-2002 for hereditary angioedema and to submit an investigational new drug application for NTLA-3001 for alpha-1 antitrypsin deficiency-associated liver disease.

2. Sickle Cell Disease Gene-Editing Therapy Trial Voluntarily Paused Following Serious Adverse Event

Graphite Bio has voluntarily paused the phase 1/2 CEDAR clinical trial (NCT04819841) of nulabeglogene autogedtemcel (nula-cel; formerly known as GPH101), an autologous CD34+ hematopoietic stem cell gene-editing therapy intended to correct the mutation that causes sickle cell disease, following an unexpected serious adverse event deemed likely to be related to the treatment.

3. Val-rox Meets End Points in Hemophilia A at 3 Years

A 3-year analysis of the phase 3 GENEr8-1 study (NCT03370913) has revealed that the study’s primary and secondary endpoints have continued to be met and valoctocogene roxaparvovec (val-rox; Roctavian; BioMarin) has a durable effect on annualized bleeding rate and factor VIII usage in patients with hemophilia A.

4. Engineered TIL Therapy Trial Reopens Enrollment After Voluntary Pause Related to Manufacturing Analysis

The phase 1 clinical trial (NCT05397093) for Instil Bio’s ITIL-306, an investigational autologous tumor infiltrating lymphocyte therapy intended to treat various types of solid tumors, has been resumed following a voluntary pause of enrollment related to a manufacturing analysis.

5. Catalent to Provide Manufacturing Services for Sarepta's SRP-9001

Sarepta Therapeutics' delandistrogene moxeparvovec (SRP-9001) is an investigational gene therapy intended to treat Duchenne muscular dystrophy.

6. Genprex Acquires Rights Related to Diabetes Gene Therapy From the University of Pittsburgh

The signed agreement includes provisions for a worldwide, exclusive license to a patent application and related technology for GPX-003, a preclinical gene therapy candidate intended to treat type 2 diabetes.

7. Fate Therapeutics, Janssen to Discontinue Collaboration

Fate Therapeutics additionally announced a prioritization of its pipeline and initiatives for 2023.

8. 2seventy bio, Regeneron Expand Collaboration

The expansion of the pre-existing collaboration is intended to accelerate the companies' development of cell therapy-based combinations intended to treat solid tumors.

9. Viralgen Granted cGMP Clearance for Production of Commercial-grade rAAV Product

The new facility, located in San Sebastian, Spain, recently underwent a successful inspection.

10. Synthekine Announces Intention to Advance STK-009 + SYNCAR-001 Following Round of Series C Financing

The $100 million raised will help support the development of the clinical-stage IL-2 + CD19 CAR-T combination therapy, in addition to the company's preclinical programs.

11. PsiOxus Therapeutics, Now Renamed "Akamis Bio," Receives $30 Million in Financing

The company also provided an update on its clinical-stage tumor gene therapies.

12. Neurocrine Biosciences, Voyager Therapeutics to Collaborate on Development of Gene Therapies Targeting CNS Diseases

Among the programs included in the collaboration is a preclinical GBA1 gene replacement therapy intended to treat Parkinson disease and other GBA1-mediated diseases.

13. ReCode Therapeutics, Asklepios BioPharmaceutical to Pursue Delivery of Gene Editing by Single Vector Approach

The 2 companies announced a new collaboration intended to develop a novel platform to be used for liver and lung diseases.

14. Cabaletta Bio to Use Autolus Therapeutics' RQR8 Safety Switch in Autoimmune Disease Cell Therapy

The non-exclusive license agreement also includes provisions for Cabaletta to use the technology in as many as 4 other programs.

15. Myrtelle, rAAVen Therapeutics to Collaborate on recombinant AAV Vectors for CNS Disease Gene Therapies

The 2 companies will focus their efforts on CNS diseases that affect myelin.

16. PTC Therapeutics Announces Pipeline Updates and 2022 Highlights

The company also reported on its financial progress and its expected development milestones for the coming year.

17. bluebird bio Provides Update on Patient Dosing With Newly Approved Gene Therapies

The company announced that the preparations have begun for the first patients to be treated with beti-cel and eli-cel.

18. Orchard Therapeutics On Track With FDA Actions for 2023

The company will launch a global registration trial for OTL-203 in mucopolysaccharidosis type I in the second half of the year.