Around the Helix: Gene and Cell Therapy Company Updates - January 19, 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. GeneTherapyLive’s Around the Helix is your chance to catch up with the latest news in gene and cell therapies, including partnerships, pipeline updates, and more.

1. FDA Extends BLA Review for bluebird bio’s β-Thalassemia and CALD Gene Therapies

The FDA has extended the review dates for bluebird bio’s biologics license applications for its investigative gene therapies: betibeglogene autotemcelfor β-thalassemia and elivaldogene autotemcel for cerebral adrenoleukodystrophy to August 19, 2022, and September 16, 2022, respectively.

2. Celularity’s CYNK-101 Wins Fast Track Designation for HER2+ Gastric/GEJ Cancers

The FDA has granted fast track designation to CYNK-101, a genetically modified natural killer cell therapy being developed by Celularity, in combination with standard frontline chemotherapy, trastuzumab (Herceptin), and pembrolizumab (Keytruda) for patients with advanced HER2-positive gastric or gastroesophageal junction adenocarcinoma. 

3. Gamida Cell on Track to Submit Omidubicel BLA in First Half of 2022

Gamida Cell is planning to complete their biologics license application for omidubicel, a cell therapy for bone marrow transplant in patients with hematologic malignancies, in the first half of 2022 following positive Type B meeting correspondence from the FDA. The FDA had previously requested additional manufacturing data from a phase 3 study.

4. REGENXBIO Initiates ASCENT Trial of RGX-314 Wet AMD Gene Therapy

ASCENT is the second of 3 pivotal trials of their subretinal gene therapy RGX-314 for the potential treatment of wet age-related macular degeneration. ATMOSPHERE (NCT04704921), the first trial to be initiated by REGENXBIO in collaboration with AbbVie, is currently active and screening patients.

5. Rubius Therapeutics Doses First Patient in Cell Therapy Trial for Solid Tumors

The phase 1/2 trial is assessing RTX-224, an allogeneic, cell therapy candidate expressing 4-1BBL and IL-12, for the potential treatment of relapsed/refractory or locally advanced solid tumors, including non-small cell lung cancer, cutaneous melanoma, head and neck squamous cell carcinoma, urothelial carcinoma and triple-negative breast cancer.

6. Atara and Imeka Collaborating to Use Advanced Imaging in MS Cell Therapy Trial

Atara Biotherapeutics will use Imeka’s proprietary biomarker imaging technology in the phase 2 EMBOLD trial (NCT03283826) assessing ATA188 as a monotherapy in multiple sclerosis. Atara hopes to use the imaging technology to further analyze the effects of ATA188 on neuroinflammation and remyelination.

7. Mesoblast’s Cell Therapy Reduces Chronic Low Back Pain

Mesoblast’s allogeneic cell therapy rexlemestrocel-L (MPC-06-ID) reduced chronic low back pain associated with degenerative disc disease, according to 36-month data from their 404-patient phase 3 trial (NCT02412735). The cell therapy will be assessed in an additional phase 3 trial.

8. BlueRock Therapeutics Doses First Patient in Phase 1 Cell Therapy Trial in Parkinson Disease

The first patient in Canada has been dosed in a phase 1, open-label trial (NCT04802733) of MSK-DAO1, BlueRock’s pluripotent stem cell-derived dopaminergic neuron cell therapy in patients with advanced Parkinson disease.

9. Longeveron Publishes Trial Design for Frailty in Aging Cell Therapy

The phase 2b trial will assess Lomecel-B, a living cell therapy product, as a regenerative medicine for the potential treatment of aging frailty. The design and rationale of the study was published in the Journal of Frailty and Aging.

10. Tevogen Granted Patent for SARS-CoV-2 T-Cell Therapy Preparation

Tevogen Bio has been granted a patent for their method of preparing and manufacturing TVGN-489, a COVID-19 peptide specific cytotoxic T cell therapy. The cell therapy is designed to target the entire SARS-CoV-2 viral genome rather than only spike proteins.

11. Intellia Doses First Patient With ATTR Amyloidosis and Cardiomyopathy in New Gene Therapy Trial Cohort

The phase 1 trial (NCT04601051) of NTLA-2001, an investigational CRISPR gene editing therapy delivered with Intellia’s lipid nanoparticle technology for the treatment of ATTR amyloidosis, recently added a cardiomyopathy arm which has dosed its first patient. Intellia expects the trial, which continues to evaluate NTLA-2001 in patients with hereditary ATTR amyloidosis with polyneuropathy, to complete enrollment in 2022.

REFERENCES

1. bluebird provides update on FDA review timelines for betibeglogeneautotemcel (beti-cel) for beta-thalassemia and elivaldogeneautotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD). News release. bluebird bio. January 18, 2022. https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-provides-update-fda-review-timelines-betibeglogene
2. Celularity receives fast track designation from U.S. FDA for its NK cell therapy CYNK-101 in development for the first-line treatment of advanced HER2/neu positive gastric and gastroesophageal junction cancers. News release. Celularity. January 18, 2022. Accessed January 18, 2022. https://bit.ly/3tyy3Vv
3. Gamida Cell provides update on omidubicel BLA submission. News release. Gamida Cell. January 19, 2022. https://www.yahoo.com/now/gamida-cell-provides-omidubicel-bla-120000727.html
4. Gamida Cell Provides Update on Pre-BLA Meeting With FDA for Omidubicel. News release. Gamida Cell. News release. November 11, 2021. https://www.businesswire.com/news/home/20211111005505/en/Gamida-Cell-Provides-Update-on-Pre-BLA-Meeting-With-FDA-for-Omidubicel
5. REGENXBIO announces initiation of second pivotal trial in RGX-314 clinical program for the treatment of wet AMD using subretinal delivery. News release. REGENXBIO. January 10, 2022. https://regenxbio.gcs-web.com/news-releases/news-release-details/regenxbio-announces-initiation-second-pivotal-trial-rgx-314
6. Rubius Therapeutics announces dosing of first patient in phase 1/2 trial of RTX-224, a broad immune agonist, for the treatment of certain solid tumors. News release. Rubius Therapeutics. January 13, 2022. https://www.biospace.com/article/releases/rubius-therapeutics-announces-dosing-of-first-patient-in-phase-1-2-trial-of-rtx-224-a-broad-immune-agonist-for-the-treatment-of-certain-solid-tumors/?s=107
7. Imeka collaborates with Atara Biotherapeutics to provide neuroimaging for phase 2 study in multiple sclerosis. News release. Imeka. January 11, 2022. https://www.prnewswire.com/news-releases/imeka-collaborates-with-atara-biotherapeutics-to-provide-neuroimaging-for-phase-2-study-in-multiple-sclerosis-301457246.html
8. Single dose of Mesoblast’s allogeneic cell therapy provides durable pain reduction for at least three years in patients with degenerative disc disease 36-Month results of phase 3 trial in chronic low back pain presented at 2022 biotech showcase. News release. Mesoblast. January 12, 2022. https://www.biospace.com/article/releases/single-dose-of-mesoblast-s-allogeneic-cell-therapy-provides-durable-pain-reduction-for-at-least-three-years-in-patients-with-degenerative-disc-disease36-month-results-of-phase-3-trial-in-chronic-low-back-pain-presented-at-2022-biotech-showcase/?s=69
9. BlueRock Therapeutics announces first patient dosed in Canada in phase 1 trial in patients with advanced Parkinson's disease. News release. BlueRock Therapeutics. January 18, 2022. https://finance.yahoo.com/news/bluerock-therapeutics-announces-first-patient-130000024.html
10. Longeveron announces publication of Lomecel-B phase 2b trial design in Journal of Frailty and Aging. News release. Longeveron. January 12, 2022. https://www.biospace.com/article/releases/longeveron-announces-publication-of-lomecel-b-phase-2b-trial-design-in-journal-of-frailty-and-aging/
11. Tevogen Bio™ further strengthens IP portfolio with additional patent for method of preparing its investigational SARS-CoV-2 specific T cell therapy. News release. Tevogen Bio. January 12, 2022. https://www.businesswire.com/news/home/20220112006056/en/Tevogen-Bio%E2%84%A2-Further-Strengthens-IP-Portfolio-With-Additional-Patent-for-Method-of-Preparing-Its-Investigational-SARS-CoV-2-Specific-T-cell-Therapy
12. Intellia Therapeutics highlights strategic priorities and anticipated development milestones for 2022. News release. Intellia Therapeutics. January 6, 2022. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-highlights-strategic-priorities-and-0