Around the Helix: Cell and Gene Therapy Company Updates – March 20, 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. FDA Approves Liso-Cel (Breyanzi) as First CAR-T for CLL, SLL

The FDA has approved Bristol Myers Squibb’s chimeric antigen receptor (CAR) T-cell therapy lisocabtagene maraleucel (liso-cel; Breyanzi) for treating adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least 2 prior lines of therapy, including a Bruton tyrosine kinase inhibitor and a B-cell lymphoma 2 inhibitor, under the accelerated approval pathway.

2. ODAC Meeting: Cilta-Cel Gets Unanimous Vote, Ide-Cel Wins Majority Vote for Benefit-Risk Profile

The FDA’s Oncologic Drugs Advisory Committee (ODAC) Meeting has unanimously voted (11 yes, 0 no, 0 abstain) in favor of ciltacabtagene autoleucel's (cilta-cel; Janssen, Legend Biotech) benefit-risk profile for patients with relapsed and lenalidomide-refractory multiple myeloma (MM) after at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent.

3. FDA Approves Orchard Therapeutics’ Gene-Edited Cell Therapy Arsa-Cel for Metachromatic Leukodystrophy

The FDA has approved Orchard Therapeutics’ gene-edited cell therapy atidarsagene autotemcel (arsa-cel, previously referred to as OTL-200 and now marketed in the US under the name Lenmeldy), for the treatment of children with presymptomatic late infantile, PS early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Several days after the approval Orchard announced that the therapy will be priced at $4.25 million in the US.

4. Patients With Hemophilia Biding Their Time for Gene Therapy

Many patients with hemophilia A/B are content with their current treatment regimens and only a minority are currently eager to undergo gene therapy, presenting a challenge in attempting to establish best practices for informed consent with the treatment mode.

5. ImmPACT Bio Secures Grant to Bring CD19/CD20 Bispecific CAR-T to Lupus

ImmPACT Bio has received an $8 million grant from the California Institute for Regenerative Medicine that will help support clinical development of IMPT-514, an investigational CAR-T that is currently being evaluated in a phase 1b/2 clinical trial for the treatment of refractory lupus nephritis and systemic lupus erythematosus.

6. Cartherics to Try CAR-NK Cell Therapy Targeted at Tissue Factor Via Agreement With Ohio State University

Cartherics has executed an option agreement for research using intellectual property from The Ohio State University that covers the use of CAR natural killer (NK) cell therapy to target tissue factor (TF), a coagulation factor expressed in some tumor tissues and tumor blood vessels.

7. Hemogenyx Garners $4.2 Million Via Placing Shares

The company will put the net proceeds from this move towards HEMO-CAR-T, its CAR-T therapy that has been cleared by the FDA for a phase 1 clinical trial in patients with acute myeloid leukemia. "This is a key development that represents the progression of Hemogenyx Pharmaceuticals to become a clinical stage company and the fundraise was crucial to enable us to progress toward clinical trials, the essential next step for the development of HEMO-CAR-T," Vladislav Sandler, PhD, the CEO and cofounder of Hemogenyx, said in a statement.