Around the Helix: Cell and Gene Therapy Company Updates – October 16, 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. Nanoscope Announces BLA Submission Plans for Retinitis Pigmentosa Gene Therapy MCO-010 Following Meeting With FDA

Nanoscope Therapeutics has stated that it plans to file a biologics license application (BLA) for MCO-010 (sonpiretigene isteparvovec), its investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy intended to treat retinitis pigmentosa, following a meeting with the FDA that it characterized as “productive”.

2. Hematological Malignancies Detected in Some Patients Treated With bluebird bio’s Active Cerebral Adrenoleukodystrophy Gene Therapy Eli-Cel

Seven of 67 patients treated with bluebird bio's elivaldogene autotemcel (eli-cel; marketed as Skysona), a gene therapy product comprised of engineered autologous CD34+ hematopoietic stem cells, for early, active cerebral adrenoleukodystrophy across multiple clinical trials have developed hematologic malignancies, according to a study published in The New England Journal of Medicine.

3. Poseida Therapeutics’ Allogeneic CAR-T P-BCMA-ALLO1 Demonstrates 91% ORR in Enhanced Lymphodepletion Arm of R/R MM Study

Poseida Therapeutics’ P-BCMA-ALLO1, an allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, has generated positive data, including a 91% overall response rate (ORR) in an enhanced lymphodepletion arm, in interim data from a phase 1/1b clinical trial (NCT04960579) in relapsed/refractory (r/r) multiple myeloma (MM).

4. Kiromic BioPharma’s NSCLC T-cell Therapy Deltacel Continues to Provide Disease Stabilization in Updated Data

Kiromic BioPharma’s KB-GDT-01 (Deltacel), an investigational allogeneic gamma delta T-cell therapy being evaluated in the phase 1/2 Deltacel-01 clinical trial (NCT06069570) for the treatment of non-small cell lung cancer (NSCLC), has continued to show evidence of providing disease stabilization in updated data from the study.

5. Adicet's ADI-100 Snags Clearance for Evaluation in 2 Additional Autoimmune Disease Indications

Adicet Bio’s ADI-100, an investigational allogeneic CAR-engineered gamma delta T-cell therapy currently being evaluated in a phase 1 clinical trial (NCT06375993) for lupus nephritis, has received clearance from the FDA of an amendment to its investigational new drug (IND) application, allowing for the recruitment of patients with idiopathic inflammatory myopathy and stiff person syndrome into the trial.

6. ElevateBio Demonstrates Targeted Delivery of its LNP Platform in Mouse Model

ElevateBio has conducted nuclease editing in the liver of mouse models with the use of its lipid nanoparticle (LNP) platform that delivers its Life Edit gene editing system. The company noted that the platform has a low immunogenicity and is capable of targeting the liver or other tissues as needed. “This is an important milestone for Life Edit as we advance our proprietary delivery platforms alongside our base and reverse transcriptase editing capabilities to develop and deliver safe and efficacious gene editing therapeutics for a broad range of genetic diseases,” Tedd Elich, PhD, the chief scientific officer of Elevate Bio subsidiary Life Edit.

7. New Company Tolerance Bio Launches With Intent to Tackle Immune Disease With Cell Therapy

Tolerance Bio, which has launched with $17.2 million garnered in a round of seed financing, is developing an allogeneic thymus induced induced pluripotent stem cell (iPSC)-based cell therapy platform that it intends to use to delay or prevent the loss of function of the thymus, or restore its function if lost. The company expects that this technology could have applications in a range of immune-mediated diseases including cancers, transplant rejections, infections, allergies, autoimmune diseases, allergies, and immune deficiency.

8. GenPrex Given Go-Ahead to Move to Highest Dosing Group in ES-SCLC Gene Therapy Trial

GenPrex has received clearance from the safety review committee for the phase 1/2 Acclaim-3 clinical trial (NCT05703971), which is evaluating its investigational immunogene therapy quaratusugene ozeplasmid (Reqorsa) for the treatment of extensive stage small cell lung cancer (ES-SCLC), to move on to dosing patients in its highest dose group (0.12 mg/kg) in the phase 1 portion.