Around the Helix: Cell and Gene Therapy Company Updates – July 26, 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.

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1. BioCardia's CardiAMP Heart Failure Cell Therapy Faces Uncertain Future

BioCardia is pausing enrollment in its phase 3 trial (NCT02438306) assessing CardiAMP cell therapy in patients with heart failure with reduced ejection fraction while additional blinded data are collected, in accordance with a data safety monitoring board recommendation. The recommendation concluded that the trial was unlikely to meet its primary composite endpoint comprising deaths, adverse events, and functional outcomes.

2. Excision's CRISPR Therapy Fast Tracked for HIV

The FDA has granted Fast Track designation to Excision Biotherapeutics’ CRISPR-based, multiplexed in vivo gene-editing therapy EBT-101 for the potential treatment of human immunodeficiency virus type 1 (HIV-1). The therapy is being evaluated in an open-label, multicenter, single ascending dose phase 1/2 trial to evaluate safety, tolerability, and preliminary efficacy.

3. MD Anderson and Syena’s TCR NK Cell Therapy Gets Another IND Clearance for Multiple Myeloma

The University of Texas MD Anderson Cancer Center and Syena, a subsidiary of Replay, have received clearance of a new investigational new drug (IND) application for their T-cell receptor natural killer cell therapy NY-ESO-1 TCR/IL-15 NK. The new IND is the second the FDA has cleared for NY-ESO-1 TCR/IL-15 NK after a previous IND for an advanced synovial sarcoma and myxoid/round cell liposarcoma indication.

4. T-Cell Therapy Racks Fast Track, Orphan Designations for EBV-Nasopharyngeal Carcinoma

The FDA has granted both fast track and orphan drug designations to Biosyngen’s T-cell therapy BRG01 for the treatment of Epstein-Barr virus (EBV)-positive relapsed/metastatic nasopharyngeal carcinoma. BRG01 is an engineered, allogeneic, EBV-targeting T-cell therapy that has previously demonstrated preliminary safety and efficacy in exploratory clinical trials.

5. KromaTiD Launches 300 New Probes for Directional Genomic Hybridization in-Site Assays

The new directional Genomic Hybridization (dGH) in-Site probes, which are intended allow for customers to perform in-house assays, are expected to enable evaluations of genomic structural variation and gene-editing outcomes beyond those possible with next generation sequencing or fluorescence in-situ hybridization. “The key difference between dGH and other in-situ hybridization (ISH) methods is the outcome: dGH provides a much deeper data set than any other genomic ISH technique,” Christopher Tompkins, PhD, the chief technology officer of KromaTiD, said in a statement.

6. Roche Joins Forces With Alnylam on Hypertension RNAi Therapeutic

Zilebesiran is currently being evaluated by Alnylam in 2 phase 2 clinical trials, KARDIA-1 (NCT04936035) and KARDIA-2 (NCT05103332), for the treatment of patients with hypertension. “We are thrilled to announce this collaboration, as it combines Alnylam’s proven track record in RNAi therapeutics with Roche’s global commercial reach, commitment to innovation and desire to transform the landscape for patients with severe cardiovascular diseases,” Yvonne Greenstreet, MBChB, the CEO of Alnylam, said in a statement.

7. DTx Pharma Acquired by Novartis

DTx Pharma is developing DTX-125, a preclinical stage siRNA therapy intended to treat Charcot-Marie-Tooth disease type 1A by reducing expression of the PMP22 protein in Schwann cells. It is based on the company’s proprietary FALCON platform and has received orphan drug designation from the FDA. DTx Pharma also has 2 other siRNA therapies for neurological indications in preclinical development.

8. Turnstone Biologics Prices IPO at $12 per Share

Turnstone Biologics, which is focused on the development of tumor-infiltrating lymphocyte therapies directed at solid tumor indications including breast cancer and melanoma, made an initial public offering (IPO) of 6,666,667 shares of common stock priced at $12.00 per share, with the expectation of accumulating $80.0 million in gross proceeds. 

9. Greenleaf Health Adds Wilson Bryan, MD, to its Team

Wilson Bryan, MD, the first director of the Office of Tissues and Advanced Therapies in the FDA’s Center for Biologics Evaluation and Research, who served in that role until February 2023, is now the executive vice president, drug and biological products, at Greenleaf Health, an FDA regulatory consulting firm. In total, Bryan worked at the FDA for 19 years.

10. Editas Medicine Recruits Linda C. Burkly, PhD

Editas Medicine has added Linda C. Burkly, PhD, who has previously served as a vice president and senior distinguished investigator on neuroscience-focused research teams at Biogen, as its new executive vice president and chief scientific officer. “Linda has an outstanding track record of inventing or contributing to the foundations of multiple approved medicines and late-stage clinical candidates,” Gilmore O’Neill, MB, MMSc, the president and CEO of Editas Medicine, said in statement.

11. Sanofi, Scribe Therapeutics Add Terms for In Vivo Therapies to Their Collaboration

Sanofi and Scribe Therapeutics, which previously formed an agreement to collaborate on the development of ex vivo NK cell therapies for cancer indications, have expanded their collaboration to include in vivo genome editing approaches to treating diseases like sickle cell disease. “We are continually impressed by the expertise, rigor, and urgency that Sanofi has brought to our existing ex vivo collaboration, as well as their rapid advancements and innovation in non-viral delivery technologies,” Benjamin Oakes, PhD, the co-founder and CEO of Scribe, said in a statement. “Now, we are thrilled to expand our work together into new areas of high unmet need.”

12. Neurogene, Neoleukin Therapeutics To Merge

The combined company, which is anticipated to operate under the Neurogene name, will direct its efforts towards further development of Neurogene’s existing portfolio of investigational genetic medicine products. “This transaction is expected to bolster our ability to progress our differentiated pipeline, including our clinical-stage program in Rett syndrome which contains our novel, proprietary EXACT technology,” Rachel McMinn, PhD, the founder and CEO of Neurogene, said in a statement.