Around the Helix: Cell and Gene Therapy Company Updates – August 28, 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.

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1. Legend Biotech’s CAR-T Carvykti Approved for R/R MM Indication in China

Legend Biotech's ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti in the United States and Europe), a BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy, has been approved by China’s National Medical Products Administration (NMPA) for the treatment of adults with relapsed or refractory (r/r) multiple myeloma (MM) who previously received treatment with 3 or more lines of therapy, including 1 or more proteasome inhibitor and 1 immunomodulatory agent.

2. JW Therapeutics’ Relma-Cell Approved in China for R/R Mantle Cell Lymphoma

JW Therapeutics’ relmacabtagene autoleucel injection (relma-cel; marketed as Carteyva), a marketed CAR-T therapy, has received approval of its supplemental biologics license application for the treatment of adults with r/r mantle cell lymphoma (MCL) from China’s NMPA.

3. Galapagos Cleared by FDA to Bring Trial for CAR-T Therapy GLPG5101 in R/R NHL to US

Galapagos has received clearance from the FDA of an investigational new drug (IND) application for GLPG5101, an investigational CD19-directed CAR-T therapy, allowing the company to evaluate the CAR-T for the treatment of r/r nonHodgkin lymphoma (NHL) in a phase 1/2 clinical trial.

4. CARsgen Finishes Enrolling Patients in Pivotal Trial in China for Gastric/Gastroesophageal CAR-T Satri-Cel

CARsgen has completed the enrollment of patients in its pivotal phase 2 clinical trial (NCT04581473) evaluating satricabtagene autoleucel (satri-cel; CT041), an investigational chimeric antigen receptor T-cell (CAR-T) therapy that targets CLDN18.2, for the treatment of CLDN18.2-positive advanced gastric/gastroesophageal (GC/GEJ) cancer.

5. Tome Letting Go of 131 Employees

According to a Worker Adjustment and Retraining Act report on the Massachusetts government website, Tome Biosciences, which is developing technologies that fall under the umbrella of programmable genomic integration with the intent of treating a range of diseases, will be laying off 131 of its employees in November 2024.

6. New Company Tern Therapeutics Seeks to Tackle Batten Disease

Tern Therapeutics, a new biotech company focused on the development of one-time gene therapies for rare diseases, has announced its launch with $15 million from a round of financing. The company additionally noted that it has acquired 2 gene therapy candidates, RGX-381 and RGX-181, through a licensing agreement with REGENXBIO. It intends to continue the development of these candidates, which are intended to treat the ocular and central nervous system manifestations of the CLN2 subtype of Batten disease, respectively, and which it has renamed to TTX-381 and TTX-181, respectively.

7. Navigator Medicines In-Licenses Autoimmune Disease Bispecific Antibody Therapeutic

Navigator Medicines, which simultaneously garnered $100 million in a series A round of financing, has in-licensed NAV-240 (previously referred to as IMB101), a bispecific antibody against OX40L and TNFα, from IMBiologics Corp. “NAV-240 has the potential to make an impact on patients living with autoimmune diseases, and our Series A funding will be pivotal in accelerating its development alongside other exciting programs within our pipeline," Dana McClintock, MD, the chief medical officer of Navigator Medicines, said in a statement. "We look forward to initiating additional clinical studies with NAV-240 in the coming months and delivering on our commitment to innovation that enhances patient care.”

8. New Company Borealis Biosciences Will Seek to Treat Kidney Disease With RNA Therapeutics

Borealis Biosciences has come out of stealth, having raked in $150 million from Series A financing from Versant Ventures and Novartis AG and strategic research collaboration funding from Novartis, and announced its intent to address unmet needs in kidney disease through the development of RNA therapies. “We’ve recognized over the last 6 years that some of the most validated targets for kidney disease have been out of reach with traditional modalities..." Jerel Davis, PhD, the managing director at Versant and a chairperson of Borealis’ board of directors, said in a statement. “We are excited to launch this startup in close collaboration with Novartis, as this represents another example of Versant’s creative company-building together with an industry leader.”