Around the Helix: Cell and Gene Therapy Company Updates – May 31, 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. FDA Accepts Lifileucel's BLA for Advanced Melanoma With Priority Review

The FDA has accepted for filing a biologics license application (BLA) for Iovance Biotherapeutics’ lifileucel (LN-144), an investigational autologous tumor infiltrating lymphocyte (TIL) therapy intended to treat advanced melanoma. The BLA was accepted with priority review, with a PDUFA action date set for November 25, 2023.

2. European Application Submitted for Cilta-cel in Relapsed and Lenalidomide-Refractory Myeloma

The European Medicines Agency (EMA) has announced that it received a Type II variation application for the approval of ciltacabtagene autoleucel (Carvykti; cilta-cel; Johnson & Johnson/Legend Biotech) in adult patients with relapsed and lenalidomide (Revlimid; Bristol Myers Squibb)-refractory multiple myeloma (MM). Sen Zhuang, MD, PhD, vice president of clinical research and development at Janssen Research & Development, said in a press release that “this submission is a testament to our relentless commitment to advance science, transform outcomes, challenge what a [diagnosis of] MM means for patients, and ultimately, work toward our goal of one day curing this complex disease."

3. Sarepta’s Delandistrogene Moxeparvovec Shows Functional Improvements and Acceptable Safety in 4-Year Trial Update

Sarepta Therapeutics’ delandistrogene moxeparvovec (SRP-9001), an investigational gene therapy intended to treat ambulant individuals with Duchenne muscular dystrophy, has demonstrated functional improvements and safety in 4-year data from 4 patients treated in a phase 1/2a clinical trial (SRP-9001-101; NCT03375164). A BLA for SRP-9001 is currently under review by the FDA, with its review deadline recently delayed to June 22, 2023.

4. ACGT Awards Luncheon Honors Crystal Mackall, MD, With Edward Netter Leadership Award

Alliance for Cancer Gene Therapy (ACGT) awarded The Edward Netter Leadership Award to Crystal Mackall, MD, of Stanford University, at the ACGT Awards Luncheon on March 30, 2023, at Riverpark restaurant at the Alexandria Center for Life Science, in New York City. The ACGT Awards Luncheon celebrated the nonprofit organization’s 20th anniversary and ushered in a new decade.

5. Beam Therapeutics Recruits Genome Medical to Provide Genetic Counseling for Patients Enrolling in BEACON Clinical Trial

The phase 1/2 BEACON study (NCT05456880) is evaluating BEAM-101, an investigational CD34-positive base-edited autologous hematopoietic stem cell therapy intended to treat sickle cell disease. “We consider our work with biopharmaceutical companies critical to the advancement of genomic medicine and enabling access to life-saving therapies and treatments for patients living with rare disease,” Jill Davies, the CEO of Genome Medical, said in a statement.

6. NIH-NIAMS Grant Provides Funding for Amplo Biotechnology’s Congenital Myasthenic Syndrome Gene Therapy

The Fast Track Phase 1/2 STTR grant provides funding to push forward development of AMP-201, an adeno-associated virus vector-based gene therapy intended to treat the severe congenital myasthenic syndrome caused by Collagen Q Deficiency. Amplo Therapeutics is developing the therapy based on data licensed from the laboratory of Kinji Ohno, MD, PhD, at Nagoya University and is working with Ricardo Maselli, MD, a professor at the University of California, Davis, on the therapy’s development.

7. UCB Enlists ClearPoint Neuro for Delivery of its Investigational Gene Therapy Products 

UCB will utilize ClearPoint Neuro’s delivery technology in the development of its gene therapy products as part of a multiyear license agreement, which includes success-based milestone payments for ClearPoint. “We believe that ClearPoint Neuro are a strong strategic fit for UCB as they are a company dedicated to innovation in the neurology space with a unique portfolio of navigation and drug delivery tools,” Dhaval Patel, MD, PhD, the executive vice president and chief scientific officer of UCB, said in a statement.

8. Terumo Blood and Cell Technologies Launches Cell Collection Training Program

The newly inaugurated training program is focused on training cell therapy manufacturers in apheresis. The company stated that the training is intended to help improve time-to-treatment for patients with critical disease. “Each patient's or donor's characteristics may impact the quality or quantity of cells collected during the procedure, so we help participants use data analytics services to show how they may reduce collection days and advance treatments more quickly for patients in need,” Veerle d'Haenens, the general manager of global therapeutic systems and cell therapy technologies at Terumo, said in a statement.

9. Catalent Installs Cryogenic Freezers in Japan Facility

The addition to the facility located in Shiba, Japan, which was originally opened in October 2021, enables storage, kitting, and distribution of clinical supply for investigational cell therapy products and was made with the intention of meeting the demands of the growing market for advanced therapies in Japan. 

10. Nexcella Begins Producing Engineering Batches for AL Amyloidosis CAR-T in United States

The company stated that its commencement of engineering batches for NXC-201 (HBI0101), an investigational chimeric antigen receptor T-cell (CAR-T) therapy intended to treat light chain amyloidosis, is a key step in bringing the phase 1b/2a NEXICART-1 (NCT04720313) clinical trial to the United States. NEXICART-1 is currently treating patients at Hadassah Medical Center in Israel.

11. ElevateBio to Support Continued Platform Development With $401 Million in New Funding

The company announced that funds from the recently closed round of series D financing will support projects including the ongoing development of its subsidiary Life Edit’s gene editing technology platform. ElevateBio noted that the funds will also support activities related to BaseCamp, its subsidiary that provides cGMP manufacturing services for cell and gene therapy products.