Around the Helix: Cell and Gene Therapy Company Updates – June 28, 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 BLA Filing for Hemophilia B Gene Therapy Fidanacogene Elaparvovec

Pfizer’s biologics license application (BLA) for fidanacogene elaparvovec, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat adult patients with hemophilia B, has been accepted by the FDA for filing with a Prescription Drug User Fee Act date set for Q2 2024. In addition to the BLA acceptance, Pfizer noted that a European marketing authorization application for the gene therapy has been accepted by the European Medicines Agency.

2. MD Anderson and Syena’s Sarcoma TCR NK Cell Therapy Gets IND Clearance

The University of Texas MD Anderson Cancer Center and Syena, a subsidiary of Replay, have received clearance of an investigational new drug (IND) application for their T-cell receptor natural killer (TCR NK) cell therapy NY-ESO-1 TCR/IL-15 NK, which is intended to treat advanced synovial sarcoma and myxoid/round cell liposarcoma. NY-ESO-1 TCR/IL-15 NK consists of cord blood-derived NK cells that have been engineered to express a TCR directed at the NY-ESO-1 cancer-associated antigen.

3. Sirnaomics Gets FDA Guidance to Push Forward Development of RNA Therapeutic STP705 for Squamous Cell Carcinoma

Sirnaomics has shared phase 2a/2b clinical data regarding STP705, an RNA therapeutic being evaluated for the treatment of squamous cell carcinoma in situ, with the FDA in an End of Phase-2 meeting and is now making moves towards a phase 3 clinical trial based on the agency’s guidance. The company stated that this year it plans to initiate a single dosage study that will serve as a subgroup of a larger phase 3 clinical trial.

4. Liso-cel Achieves Durable Responses in Patients With R/R Follicular Lymphoma and R/R Mantle Cell Lymphoma

Lisocabtagene maraleucel (liso-cel; Breyanzi; Bristol Myers Squibb), a CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy currently marketed in the United States and European Union for large B-cell lymphoma indications, has produced durable responses among patients with relapses/refractory (r/r) follicular lymphoma treated in the phase 2 TRANSCEND FL clinical trial (NCT04245839) and patients with r/r mantle cell lymphoma treated in the phase 1 TRANSCEND NHL 001 clinical trial (NCT02631044).

5. Kadimastem Receives US Patent for Technology Used in Development of Its IsletRx Cell Therapy

The patent protects a technology used for selecting and enriching islet cells in the production of IsletRx, an investigational cell therapy product intended to treat diabetes, until July 2038. Kadimastem indicated that the technology may also be used in a regenerative technology project it is collaborating on with iTolerance and the BIRD Foundation.

6. Bespoke Gene Therapy Consortium to Pursue Further Development of Gene Therapies for Rare Diseases

The Accelerating Medicines Partnership Bespoke Gene Therapy Consortium, a public-private partnership, announced that it has selected 8 gene therapies for rare disease indications for acceleration of clinical development. Among the 8 indications are Charcot-Marie-Tooth disease type 4J, congenital hereditary endothelial dystrophy, and multiple sulfatase deficiency.

7. SK pharmteco Subsidiary Yposkesi Expands Its Cell Therapy and Gene Therapy Manufacturing Capacity

Yposkesi, a French subsidiary of SK pharmteco, has completed the construction of a new manufacturing facility for adeno-associated virus and lentiviral vectors for cell therapy and gene therapy products. Together with a previously existing facility, the company now has facilities covering 10,000 square meters in Europe.

8. Orchard Therapeutics Gains $34 Million in New Funding in Relation to Initiation of BLA Submission

The new capital is the outcome of the second closing of a securities purchase agreement that previously completed its first closing in March 2023; the second closing was related in part to the initiation of a BLA submission for OTL-200. “The initiation of our BLA submission marks a significant de-risking event for Orchard and our OTL-200 program ahead of a potential US approval in metachromatic leukodystrophy,” Frank Thomas, the president and chief operating officer of Orchard Therapeutics, said in a statement.

9. Andelyn Biosciences Opens New Cell Therapy and Gene Therapy Facility in Columbus, Ohio

The Andelyn Corporate Center, the company’s new manufacturing headquarters, is 180,000 square feet and includes offices and labs in addition to the space for manufacturing. “The new facility includes 16 current good manufacturing practice modular manufacturing suites with upstream, downstream, solution prep, and filling capabilities, along with quality control labs and warehouse and corporate headquarters space,” Wade Macedone, the CEO of Andelyn Biosciences, said in a statement.

10. Kite Regains Marketing Authorization for Axi-cel in Japan

The Marketing Authorization in Japan for the CAR-T axicabtagene ciloleucel (axi-cel; Yescarta) has been transferred from Daiichi Sankyo to Kite Pharma’s Japan subsidiary Gilead Sciences K.K. Daiichi Sankyo originally gained the marketing authorizationas a result of a 2017 agreement prior to the acquisition of Kite by Gilead Sciences.

11. Ultragenyx Launches 110,000-square-foot Gene Therapy Manufacturing Facility in Bedford, Massachusetts

The facility, which is located on a 10.7-acre site and held an official grand opening on June 21, will manufacture AAV gene therapy products at scale using Ultragenyx’s Pinnacle Producer Cell Line manufacturing platform. “Through this facility, Ultragenyx will develop and produce gene therapy treatments at greater scale than previously possible, which we believe will make these rare disease medicines more accessible to the people who need them,” Dennis Huang, the chief technical operations officer and executive vice president for gene therapy research and development at Ultragenyx, said in a statement.