Around the Helix: Cell and Gene Therapy Company Updates – August 2, 2023

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Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

CGTLive Around the Helix

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. PKP2 Gene Therapy Cleared for Cardiomyopathy Trial

The FDA has granted investigational new drug (IND) clearance to LEXEO Therapeutics’ LX2020 for the potential treatment of arrhythmogenic cardiomyopathy caused by variants in the PKP2 gene. LX2020 is an AAVrh10-based gene therapy candidate designed to intravenously deliver a functional PKP2 gene to cardiac muscle to increase PKP2 protein levels in the cardiac desmosome.

2. Exstrophy-Epispadias Complex Cell Therapy Gets Orphan Drug, Rare Pediatric Disease Designations

The FDA has granted orphan drug designation to MyoPax’s regenerative cell therapy Satori-01 for the treatment of exstrophy-epispadias Complex. Satori-01 is being evaluated in the phase 1/2a first-in-human MuST trial (NCT04729582) in Germany under the guidance of Simone Spuler, MD, cofounder, MyoPax, and professor, myology, Max Delbrück Center, and Charité – Universitätsmedizin Berlin, which is sponsoring the trial.

3. Allogeneic CAR-T AVC-201 Cleared by European Medicines Agency for Trial in R/R AML

AvenCell Therapeutics’ AVC-201 (Allo-RevCAR01-T-CD123), an allogeneic CD123-directed chimeric antigen receptor T-cell (CAR-T) intended to treat relapsed/refractory (r/r) acute myeloid leukemia (AML) and certain other CD123-expressing hematological malignancies, has received clearance of a clinical trial application for a phase 1 study (NCT05949125) from the European Medicines Agency. The planned phase 1 trial will seek to recruit approximately 35 patients at sites across Germany including Berlin, Ulm, Würzburg, Marburg, and Dresden.

4. First in Human: PROCLAIM and upliFT-D Trials Bring Gene Therapy to Frontotemporal Dementia

Frontotemporal dementia (FTD) is the second most common form of dementia and typically affects patients under 65 years of age. FTD usually presents in either a behavioral or language variant, the latter of which can cause either a semantic type of dementia or a nonfluent aphasia type.

5. Homology Medicines Halts Development and Announces Layoffs Despite Positive Clinical Results

Following a report of promising data from a phase 1 clinical trial evaluating Homology Medicines’ HMI-103, an investigational gene therapy intended to treat classical phenylketonuria, the company announced that it would be halting all nonrequired development activities on all its programs to evaluate its strategic options. Homology also noted that it would be letting go of 87% of its employees. The company cited “the current financing environment” and the length of expected development timelines as motivating its decision.

6. AstraZeneca Subsidiary Agrees to Buy Multiple Gene Therapy Programs from Pfizer

Alexion, AstraZeneca Rare Disease, has agreed to purchase several early-stage gene therapy programs from Pfizer that are directed at rare disease indications for a total consideration equaling up to $1 billion before tiered royalties on sales. The deal will net Alexion multiple novel adeno-associated virus (AAV) capsids and may add Pfizer employees working on the relevant programs to Alexion’s team.

7. Kriya Therapeutics Adds Additional $150 to Series C Financing Round

The new funds bring Kriya’s total amount raised in the Series C round to $430 million. The company stated that the cash would be directed towards clinical translation of its existing gene therapy programs and scaling for its various platforms. Kriya has programs in development for ophthalmology, neurology, and metabolic disease indications that include an aggregate patient population of millions of people.

8. Solve GNE Agrees to Fund Gradalis’ GNE Myopathy Gene Therapy Program

Solve GNE noted that Gradalis is seeking to advance development of a novel lipid nanoparticle gene therapy directed at GNE myopathy or hereditary inclusion body myopathy that is being designed based on findings from earlier research. Solve GNE will provide funding to enable filing of an IND and a potential multicenter phase 1 clinical trial for the new therapy. “We have continued to develop our technology and will apply what we have learned as we move forward to establish a phase 1 trial treatment IND in coordination with Solve GNE,” John Nemunaitis, MD, the chief scientific officer of Gradalis, said in a statement.

9. Singapore-based Company CyoMed Therapeutics Garners US Patent for CAR-T Technology

The patent covers a CAR-γδ T-cell technology that CytoMed has exclusively licensed from Singapore’s Agency for Science, Technology, and Research (A*STAR), which retains ownership of the technology. “A*STAR works closely with the industry to translate our research into clinical applications,” Yeo Yee Chia, the assistant chief executive of A*STAR's Innovation & Enterprise Group, said in a statement. “Cancer is a devastating disease, and we believe that through CytoMed's licensing of A*STAR's CAR-γδ T cell technology, more cancer treatments can be developed to benefit patients.”

10. Nonviral Developer Intergalactic Therapeutics Lays Off All Staff, Seeking Buyer

Intergalactic Therapeutics has laid off all its workers, according to a post by an employee on LinkedIn. The nonviral gene therapy developer had recently announced it planned to begin clinical trials in 2024 for conditions that lead to blindness, making plans for a phase 1 trial of gene therapy IG-002, but it is now seeking a buyer, according to reports.

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