Around the Helix: Cell and Gene Therapy Company Updates – June 26, 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 Sarepta’s DMD Gene Therapy Elevidys for Expanded Indication

The FDA has approved Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus (AAV) vector-based gene therapy for patients with Duchenne muscular dystrophy (DMD), for an expanded indication in the disease. The therapy is now approved for ambulatory patients (via traditional approval) and nonambulatory patients (via accelerated approval) with a confirmed mutation in the DMD gene who are 4 years of age or older and who do not have any deletion in exon 8 or exon 9 in the gene.

2. Aurion’s Cell Therapy Wins RMAT, Breakthrough Therapy Designation for Corneal Edema

The FDA has granted Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy (RMAT) Designation to Aurion Biotech’s AURN001 cell therapy for the potential treatment of corneal edema secondary to corneal endothelial disease.

3. Kyverna’s CAR-T KYV-101 Shows Some Durable Responses in Autoimmune Disease, With One Relapse Reported

Fierce Biotech noted that Kyverna’s stock value dropped by approximately 34% in premarket trading on the Monday after the company’s presentation on the data was given, attributing the drop to the announcement of the new disease relapse. CGTLive has reached out to Kyverna for comment on the relapse and is currently awaiting a reply.

4. Huntington Disease Gene Therapy Nets RMAT Designation

The FDA has granted RMAT Designation to uniQure’s AMT-130 gene therapy for the potential treatment of Huntington disease.

5. CSL Behring’s Hemophilia B Gene Therapy Obtains Recommendation for Reimbursement in Denmark

CSL Behring’s etranacogene dezaparvovec (EtranaDez; Hemgenix), a marketed gene therapy for the treatment of hemophilia B, has received a recommendation from the Danish Medicines Council for reimbursement, making Denmark the first nordic country to reimburse the gene therapy product. “The Danish Medicines Council’s recommendation of Hemgenix means that this groundbreaking gene therapy can now be provided to eligible patients, and we are hopeful that its value will continue to be recognized in other Nordic countries,” Lise Grove, MBA, general manager, Denmark and Iceland, at CSL Behring, said in a statement.

6. Saudi Arabian Hospital Produces the Country’s First Nonimported CAR T-cells

The King Faisal Specialist Hospital & Research Centre in Saudi Arabia has manufactured the first chimeric antigen receptor (CAR)T-cells for internal use. Previously, the country had needed to import CAR T-cells from abroad. The internally produced CAR T-cells are expected to substantially cut costs and wait times for treatment of patients in Saudi Arabia.

7. NMDP BioTherapies Will Shoulder Provision of Donor Stem Cells for Partnership With BlueSphere

In a newly announced partnership, NMDP BioTherapies will handle activities related to the identification of donors for hematopoietic stem cells, and subsequent provision and delivery of donated cells, for use in BlueSphere Bio’s phase 1/2a clinical trial evaluating BSB-1001, an investigational T-cell receptor T-cell therapy that targets minor histocompatibility antigen-1, in several hematological malignancies.