Around the Helix: Cell and Gene Therapy Company Updates – April 19, 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. CARTITUDE-4 Data Leak Reveals Promising Results for Cilta-Cel in Multiple Myeloma

Among individuals with relapsed and lenalidomide-refractory multiple myeloma (MM), ciltacabtagene autoleucel (Carvykti; Johnson & Johnson/Legend Biotech) has outperformed expectations compared with standard chemotherapy regimens such pomalidomide, bortezomib and dexamethasone, or daratumumab, pomalidomide and dexamethasone, according to leaked data from the phase 3 CARTITUDE-4 trial (NCT04181827) obtained by STAT News.

2. New Clinical Data Presented at AACR 2023

Catch up on CGTLive’s coverage of the American Association for Cancer Research (AACR) Annual Meeting 2023, held April 14-19, 2023, in Orlando, Florida. Our coverage will continue throughout the week, beyond the meeting’s end.

3. First Patient in Europe Dosed With B-cell Acute Lymphoblastic Leukemia CAR-T UCART22

The first patient in Europe has been dosed with Cellectis’ UCART22, an investigational allogeneic CD22-directed chimeric antigen receptor T-cell (CAR-T) therapy being evaluated for the treatment of relapsed/refractory (r/r) B-cell Acute Lymphoblastic Leukemia (B-ALL) in the phase 1/2a BALLI-01 clinical trial (NCT04150497).

4. News and Expert Insights in Pompe Disease Gene Therapy for International Pompe Day

Saturday, April 15, 2023, was International Pompe Day. The CGTLive team gathered some of our past news coverage and interviews on Pompe disease gene therapy to offer an overview of the therapeutic progress being made for the rare disease.

5. Gene-Agnostic Gene Therapy Shows Efficacy in NR2E3- and RHO- Associated Retinitis Pigmentosa

Ocugen’s OCU400 gene therapy has demonstrated positive preliminary data in patients with retinitis pigmentosa (RP) associated with NR2E3 and rhodopsin (RHO) mutations treated in a phase 1/2 trial (NCT05203939).

6. Janssen and Legend Biotech Enlist the Aid of Novartis in Manufacturing Cilta-cel

The Master Technology Transfer, Manufacturing and Clinical Supply Services Agreement went into effect on April 12, 2023, according to a securities filing. The agreement will continue for a 3-year period, according to the document.

7. Lysogene’s Search for Buyers Does Not Yield Fruit

The deadline for takeover offers passed on April 12, 2023, with no filings submitted. The Commercial Court of Nanterre is expected to rule on the appropriate steps for the reorganization proceedings and on the possibility of judicial liquidation proceedings in the coming weeks.

8. Takeda Drops Preclinical AAV Gene Therapy Programs

The company cited a desire to direct resources towards later-stage programs, specifically its oral TYK2 inhibitor TAK-279 that is under development for autoimmune disorders. Several of its collaborative R&D programs—including with Codexis for AAV-based gene therapy for Fabry disease and with Poseida for the development of nonviral in vivo gene therapy for hemophilia A—could be affected.

9. Ginkgo Partners With Wisconsin Alumni Research Foundation Investigators on Development of Novel CAR-T Therapies

The collaboration is specifically focused on targeting GD2-expressing solid tumors. Additionally, though, the pair of companies will collaborate on building a pooled in vivo screening platform to expand novel CAR discovery.

10. SonoThera Adds Bracco Imaging’s MicrobubbleTechnology Platform to Its Repertoire

The agreement, which includes a non-exclusive license, is intended to contribute to SonoThera’s ongoing development of its nonviral gene therapy approach. Thierry Bettinger, PhD, the director of Bracco Research Center in Geneva, stated that the combination of these technologies “will undoubtedly advance development in this novel approach to gene therapy."

11. ClearPoint Neuro, NE Scientific to Collaborate on Development of Modeling Software for Drug Infusions

The simulation software, which is intended to optimize delivery of treatments to patients’ brains, could be used to model the delivery of gene therapies and make adjustments before they are actually administered.

12. Bio-Techne Completes Its Part in 24-Partner Collaboration

The Cell and Gene Therapy Catapult Process Analytical Technology (Catapult PAT) consortium is intended to bring together various organizations for the purpose of innovation in process analytical technologies specific to the cell and gene therapy space.