Around the Helix: Cell and Gene Therapy Company Updates – February 14, 2024

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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social and use #AroundTheHelix!

1. New Clinical Data Presented at WORLDSymposium 2024

Catch up on CGTLive's coverage of WORLDSymposium 2024, held February 4-9, in San Diego, California. Expert insights will continue to be posted throughout the week.

2. FDA Lifts Clinical Hold on Hemogenyx’s Planned Trial for Acute Myeloid Leukemia CAR-T HEMO-CAR-T

The FDA has lifted its clinical hold on Hemogenyx Pharmaceuticals’ investigational new drug application for HEMO-CAR-T, an autologous chimeric antigen receptor T-cell (CAR-T) therapy developed with the use of Hemogenyx’s proprietary humanized monoclonal antibody against an acute myeloid leukemia target.

3. Sylentis’ siRNA Therapy Fails to Improve Symptoms of Dry Eye Disease Due to Sjögren’s Syndrome

Sylentis’ phase 3 clinical trial (NCT04819269) of tivanisiran RNA therapy has failed the primary endpoint of improving dry eye symptoms associated with Sjögren’s Syndrome. Sylentis is a wholly owned subsidiary of PharmaMar.

4. FDA Pushes Rocket’s LAD-I Gene Therapy PDUFA for CMC Review

The FDA has extended its priority review for Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel; RP-L201) gene therapy for treating leukocyte adhesion deficiency-I (LAD-I), pushing back the therapy’s Prescription Drug User Fee Act (PDUFA) date from March 31, 2024, to June 30.

5. Sensorion Infused With €50.5 Million From Investors

Among the participants were existing investors like Redmile Group and Invus, as well as newcomers such as Aquilo Capital. “The capital increase will enable the Company to pursue its relentless efforts in moving forward its gene therapy programs developed in the framework of the renewed collaboration with the Institut Pasteur,” Nawal Ouzren, MSc, the CEO of Sensorion, said in a statement.

6. Kinea Bio Secures $500,000 for Gene Therapy Program from Parent Project Muscular Dystrophy (PPMD)’s PPMD Venture Pathways

Kinea Bio is developing a dual adeno-associated virus vector gene therapy that delivers a gene construct called midi-dystrophin, which contains a greater amount of key domains from the natural dystrophin protein than does the micro-dystrophin construct used in some other Duchenne Muscular Dystrophy gene therapies.

7. Invitae Files for Bankruptcy

Invitae, which provides genetic information via digital technology for the intended purpose of informing medical decisions, has filed for voluntary Chapter 11 Protection in New Jersey. The company noted that it is attempting to obtain approval to use cash on hand funding for the case and that it has the intention of carrying out business activities as normal during the Chapter 11 process.

8. AstraZeneca Wades Deeper Into the Cell Therapy Pool With Investment in New Manufacturing Facility

The company is putting $300 million into the facility, which is located in Rockville, Maryland and will serve as a manufacturing hub for oncology cell therapy clinical trials with a global reach. AstraZeneca noted that the facility’s manufacturing operations may eventually support production of therapies for nononcologic indications, as well.