Around the Helix: Cell and Gene Therapy Company Updates – May 25, 2022

The cell and gene therapy sectors are growing exponentially, with new playersemerging 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. Gene Therapy for Knee Cartilage Lesions Receives RMAT Designation

Ocugen has added the phase 3 cell therapy platform NeoCart to its pipeline following its Regenerative Medicine Advanced Therapy (RMAT) recognition for the repair of full-thickness lesions of the knee cartilage in adults.

2. EtranaDez Accepted for Priority Review in Hemophilia B

uniQure's biologics license application (BLA) for etranacogene dezaparvovec (EtranaDez) has been accepted by the FDA for priority review for the treatment of hemophilia B. A final analysis of the pivotal HOPE-B study was presented at ASGCT 2022.

3. Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion

The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has granted a positive opinion to PTC Therapeutics’ gene therapy eladocagene exuparvovec (PTC-AADC; Upstaza) for treating aromatic L-amino acid decarboxylase (AADC) deficiency based on findings from studies conducted in Taiwan.

4. International Partnership Aims to Improve CAR T-Cell Therapy Accessibility

University of Pennsylvania Medicine and Children’s Hospital of Philadelphia are teaming up with Costa Rica’s Caja Costarricense de Seguro Social to facilitate research with chimeric antigen receptor (CAR) T-cell therapies in the country.

5. Bayer Ends Collaboration with Atara

Bayer has decided to end their exclusive worldwide licensing agreement with Atara Biotherapeutics for next-generation mesothelin-directed CAR T-cell therapies, which includes cell therapy programs indicated for malignant pleural mesothelioma and non-small-cell lung cancer.

6. Consortium to Accelerate Rare Disease Therapy Development

ASC Therapeutics has joined Joins the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium US Consortium of Gene Therapy Partners, which includes the NIH and FDA. The consortium aims to accelerate development of therapies for rare diseases by tackling challenges in research, production, and regulations.

7. Kelonia Gets Funding for Safer CAR T-Cell Therapies

Kelonia Therapeutics, a new start-up, has designed off-the-shelf CAR T-cell therapies that are safer than traditional CAR T therapies but still personalized to patients, created with detargeted and redirected lentivirus-like particles.

8. Castle Creek Biosciences Funding Advances Gene Therapy Programs

The company has raised $112.8 million to advance the development of their phase 3 gene therapy program for recessive dystrophic epidermolysis bullosa (RDEB) as well as to submit an investigational new drug application for their in vivo gene therapy program for hereditary tyrosinemia type 1.

9. Myrtelle and Rescue Hearing to Collaborate on Hearing Loss Gene Therapy

Myrtelle has entered into a Worldwide Exclusive License Agreement with Rescue Hearing to develop a novel gene therapy for DFNB8 genetic hearing loss.

10. Ultragenyx Acquires Rights to MPSIIIA Gene Therapy

The company has acquired rights to Abeona Therapeutics' adenovirus-associated vector (AAV) gene therapy ABO-102 for the potential treatment of Sanfilippo Syndrome Type A, which is currently being evaluated in the phase 1/2 Transpher A trial (NCT02716246).

11. Catalent Launches New AAV Platform

Its new platform, titled UpTempo Virtuoso, which includes optimized standard protocols for cell culture, transfection, and downstream purification, streamlines AAV manufacturing with the goal of reducing the timeline from development to clinic and to enable rapid first-in-human clinical evaluation.

12. Roche, Spark, and bluebird Settle Trademark Suit

The lawsuit by Roche, on behalf of their subsidiary Spark Therapeutics, was filed in response to bluebird’s “Be the Spark” campaign. The lawsuit has been dismissed and the campaign remains live.