Around the Helix: Cell and Gene Therapy Company Updates – April 6, 2022

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. bluebird bio, Among Other Biotechs, Slash Workforce As They Look to Refocus Efforts and Extend Funding

bluebird bio will be cutting its workforce by 30% in order to extend its cash runway into 2023 as it awaits 2 key FDA decisions on its promising gene therapy candidates, betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel). Orchard Therapeutics is similarly cutting its workforce by 30% and focusing their hematopoietic stem cell gene therapy platform on severe neurometabolic diseases and early research programs and Taysha Gene Therapeutics is laying off 35% of their workforce and focusing on their Rett syndrome program.

2. UltraCAR T-Cell Therapy Gets Fast Track Designation for Acute Myeloid Leukemia

The FDA has granted fast track designation to Precigen’s investigational CAR T-cell therapy PRGN-3006 for the potential treatment of relapsed or refractory acute myeloid leukemia. The CD33-targeted therapy expresses interleukin-15 for stronger in-vivo expansion and persistence and contains a kill switch to improve the toxicity profile.

3. SwanBio’s Gene Therapy Shows Preclinical Efficacy in Adrenomyeloneuropathy

SwanBio Therapeutics has presented new preclinical data supporting their gene therapy candidate, SBT101, for the treatment of adrenomyeloneuropathy at the American Academy of Neurology (AAN) 2022 Annual Meeting in Seattle. A phase 1/2 trial is anticipated in the second half of 2022.

4. ASC’s Gene Therapy Gets Fast Track Designation for Hemophilia A

ASC Therapeutics’ ASC618 has been granted Fast Track Designation by the FDA and a positive opinion by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products as a potential treatment for patients with hemophilia A. The therapy is currently being evaluated in a phase 1/2 study (NCT04676048).

5. Axi-Cel Approved as Second-Line Large B-Cell Lymphoma Treatment

The FDA has approved axicabtagene ciloleucel (Axi-cel; Yescarta) as a treatment for adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy based on positive data from the phase 3 ZUMA-7 trial (NCT03391466).

6. Bone Therapeutics to Focus on Allogeneic Cell Therapy Platform

The company is scrapping its earlier programs to focus on their ALLOB platform and announced the departure of multiple executive board members. ALLOB is currently being evaluated in a phase 2b study (NCT04432389) in patients with high-risk tibial fractures and topline results are expected in the first quarter of 2023.

7. Abeona Discontinuing MPSIIIB Gene Therapy Program

Abeona Therapeutics announced that they would be discontinuing development of ABO-101, their gene therapy for the potential treatment of mucopolysaccharidosis (MPS) type 3B after no neurocognitive benefits were seen in its phase 1/2 trial (NCT04088734). The company is pursuing a partnership to take over development of ABO-102 for MPSIIIA and is focusing on their therapy for recessive dystrophic epidermolysis bullosa, EB-101.

8. Enrollment Open in Keytruda/Reqorsa Study

The phase 1/2 Acclaim-2 trial (NCT05062980) is now enrolling patients with late-stage non-small cell lung cancer (NSCLC) whose disease progressed after treatment with pembrolizumab (Keytruda) and will evalaute ozeplasmid (Reqorsa; Genprex) in combination with pembrolizumab as a potential therapeutic option.

9. First Patient Dosed in Kidney Mismatch Cell Therapy

Sangamo Therapeutics announced that the first patient has been dosed in the phase 1/2 STEADFAST clinical trial (NCT04817774) evaluating their chimeric antigen receptor (CAR) regulatory T-cell therapy TX200 for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation from a living donor.

10. SanBio’s Cell Therapy Shows Efficacy in Traumatic Brain Injury

SanBio’s phase 2 STEMTRA trial (NCT02416492) of their mesenchymal stem cell therapy SB623 met its primary end point of yielding durable improvements in function and activities of daily living compared to sham surgery in patients with chronic motor deficit from traumatic brain injury.

11. Atara Bio Sells Cell Therapy Manufacturing Facility to Fujifilm, Kicking Off Long-Term Partnership

Atara’s partner, Fujifilm, has finished acquisition of the facility in California and will provide manufacturing capabilities for Atara’s cell therapy programs.

12. Neurona’s Cell Therapy Shows Preclinical Efficacy in Focal Epilepsy

NRTX-1001 has demonstrated efficacy in chronic focal epilepsy, according to data from a preclinical trial. The cell therapy is being evaluated in a first in-human phase 1/2 clinical trial (NCT05135091) in mesial temporal lobe epilepsy.

13. Novartis Expands Manufacturing Capability With New Facility

The FDA has granted commercial licensure approval to Novartis’ North Carolina gene therapy manufacturing facility. The facility will develop and manufacture Zolgensma (onasemnogene abeparvovec) as well as future gene therapy products.

14. Iovance Receives Positive Feedback on Lifileucel

The FDA has given positive feedback on Iovance’s proposed potency assay and cell co-culture assays for its upcoming Biologics License Application (BLA) for lifileucel for the potential treatment of metastatic melanoma. The company expects to request a pre-BLA meeting in July 2022 and to complete BLA submission for lifileucel by August 2022.