Around the Helix: Cell and Gene Therapy Company Updates – April 13, 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. CAR T Therapy/RNA Vaccine Combo Shows Efficacy in Solid Tumors

BioNTech’s BNT211, a novel combination approach of autologous CAR T-cell therapy and a CAR T-cell amplifying RNA vaccine, demonstrated encouraging results in patients with testicular or ovarian cancer in an ongoing phase 1 /2a trial.

2. Gracell’s Allogeneic CAR T-Cell Therapy Shows Promise in R/R B-Cell Acute Lymphoblastic Leukemia

The CD19/CD7 dual directed chimeric antigen receptor (CAR) T-cell TruUCARtherapy GC502 has demonstrated promising efficacy in patients with relapsed/refractory B-cell acute lymphoblastic leukemia.

3. European Commission Approves Liso-Cel for R/R B-Cell Lymphoma

Lisocabtagene maraleucel (liso-cel; Breyanzi; Bristol Myers Squibb) was approved for the treatment of relapsed/refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), and follicular lymphoma grade 3B (FL3B) in patients who have received 2 or more lines of systemic treatment.

4. Cord Blood Combined Natural Killer Cell Engager Therapy Shows Efficacy in Lymphoma

Affimed’s innate cell engager therapy, AFM13, has demonstrated promising efficacy in patients with CD30-positive, relapsed/refractory (R/R) lymphoma when precomplexed with cord blood (CB)-derived natural killer (NK) cells, according to positive data from a phase 1 trial (NCT04074746) being conducted at MD Anderson Cancer Center.

5. Trial Evaluating CAR-NK Cell Therapy in Ovarian Clear Cell Carcinoma

The chimeric antigen receptor (CAR)-expressing natural killer (NK) cell therapy iCAR-ILC-N101 seems to be well-tolerated in 1 patient with ovarian clear cell carcinoma (OCCC) dosed so far in a first in-human phase 1 clinical trial being conducted in Japan.

6. Good COVID Outcomes Seen in Patients With Living Donor Kidney Transplant Cell Therapy

Most patients that received a living donor kidney transplant treated with Talaris Therapeutics’ cell therapy FRC001 remained off all immunosuppression and had good outcomes with COVID-19, with no cases of severe disease or hospitalizations.

7. Rubius’ Red Blood Cell Therapy Shows Efficacy in Advanced Solid Tumors

Data from the phase 1 arm of a phase 1/2 trial (NCT04372706) demonstrated partial responses in metastatic anal cancer, non-small cell lung cancer, and metastatic uveal melanoma with no treatment-related grade 3/4 adverse events or dose-limiting toxicities.

8. Genocea’s Neoantigen Peripheral T Cell Therapy Shows Anti-Tumor Activity

The Phase 1/2a TiTAN trial has demonstrated positive data of GEN-011 in patients with refractory solid tumors, with patients experiencing stable disease and positive biologic changes associated with their tumors. No dose-limiting toxicities have been observed.

9. First Patient With Leber Congenital Amaurosis 10 Dosed in CRISPR Gene Editing Trial

Editas Medicine has dosed the first pediatric patient in their phase 1/2 BRILLIANCE (NCT03872479) trial of EDIT-101, a CRISPR gene-editing therapy, marking the first in-vivo dosing of a pediatric patient with a CRISPR therapy.

10. AdaptImmune Identifies MAGE-A4 as CAR T-Cell Therapy Target

The company confirmed that MAGE-A4 is broadly expressed in cancers and can serve as a good target for their SPEAR CAR T-cell therapies. They presented data from a multinational, multicenter, screening protocol (NCT02636855).

11. Trial Cleared for Mesenchymal Stem Cell Therapy for ARDS in COVID-19

BioCardia’s Neurokinin-1 receptor positive therapy BCDA-04 will be evaluated in adult patients recovering from acute respiratory distress syndrome (ARDS) due to COVID-19 with a first-in-human phase 1/2 trial expected to initiate in the third quarter of 2022.