Clinical hold for heart disease gene therapy
The FDA has placed a clinical hold on Tenaya Therapeutics’ phase Ib/IIa trial for TN-201, a gene therapy being investigated for MYBPC3-associated hypertrophic cardiomyopathy. The agency has requested a protocol amendment to “standardize activities related to patient monitoring and management of the immunosuppression regimen across trial sites.”
Tenaya says it is working with the FDA to solve the situation and does not expect the action to impact data milestones or development timelines for the therapy.
MeiraGTx and Eli Lilly deal
MeiraGTx has entered into a strategic collaboration with Eli Lilly focused on developing genetic medicines for ophthalmic diseases. The agreement grants Lilly exclusive worldwide rights to MeiraGTx’s adeno-associated virus (AAV) gene therapy program targeting the AIPL1 gene for the treatment of Leber congenital amaurosis type 4, a rare inherited retinal disorder caused by mutations in the AIPL1 gene that lead to early-onset vision loss.
Lilly will also gain access to MeiraGTx’s gene therapy manufacturing capabilities and technology platforms, including proprietary intravitreal capsids, promoters, and riboswitch regulatory systems, for use in developing therapies for additional retinal targets selected by Lilly. Under the terms of the agreement, MeiraGTx will receive an upfront payment of $75 million and is eligible to receive up to $400 million in potential development and commercial milestone payments, as well as tiered royalties on future product sales.
New cGMP license for Nanoform
Nanoform Finland Plc has received a European commercial cGMP manufacturing license from the Finnish Medicines Agency (Fimea). The authorization enables Nanoform to manufacture and release drug products for commercial use within the EU and covers the company’s facility in Helsinki, which includes capabilities for nanoparticle engineering, formulation, and aseptic filling.
The facility was previously licensed for clinical manufacturing, but the new certification expands its operations to include commercial-scale production. The company indicated that it can now produce and release finished drug products for clients seeking to manufacture in Europe under full regulatory oversight. The approval represents the final regulatory step required for Nanoform to transition from clinical to commercial manufacturing within the EU framework.
Novartis opens radioligand facility
Novartis has opened a new 10,000-square-foot radioligand therapy manufacturing facility in Carlsbad, California, as part of its ongoing expansion of radiopharmaceutical production capacity in the US. The site will support the company’s growing portfolio of targeted radioligand therapies, which combine a radioactive isotope with a ligand designed to deliver radiation directly to tumor cells while minimizing exposure to healthy tissue.
Novartis stated that the site will strengthen domestic supply chains and help meet increasing demand for radioligand-based cancer treatments. The investment forms part of Novartis’s previously announced five-year, $23 billion plan to expand its manufacturing and research footprint in the US, with a focus on advanced therapeutic platforms including radioligand, cell, and gene therapies.
Phase I findings for CRISPR Therapeutics
CRISPR Therapeutics has released early results from a phase I trial of CTX310, a single-dose gene-editing therapy being developed to lower unhealthy blood lipid levels. The treatment uses CRISPR/Cas9 technology delivered by lipid nanoparticles to switch off the ANGPTL3 gene in liver cells, which plays a role in regulating triglycerides and cholesterol.
The initial findings showed that participants who received CTX310 experienced strong and lasting reductions in ANGPTL3 protein levels, along with decreases in triglycerides, low-density lipoprotein cholesterol, and other harmful lipids. The reductions were dose-dependent and sustained over several months. The trial remains underway as the company continues to test additional dose levels and monitor long-term outcomes. CTX310 is one of several in vivo gene-editing therapies CRISPR Therapeutics is developing to address cardiovascular and metabolic diseases through one-time treatments that directly target the underlying genetic causes.
New tech for AAV yields
Ascend Advanced Therapies has developed two proprietary enhancers designed to improve adeno-associated virus (AAV) vector yields without affecting product quality. The enhancers were evaluated using the company’s EpyQ two-plasmid AAV production system in HEK293 cells and compared against a standard three-plasmid system. According to Ascend, one enhancer achieved up to a threefold increase in vector yield at Ambr15 scale and a 1.7-fold increase at Ambr15 and 2-l bioreactor scales in the three-plasmid system. The second enhancer demonstrated yield increases of up to 2.4-fold in the EpyQ system at Ambr15 scale and up to 2.1-fold in Ambr250 and 5-l bioreactors.
"Improving AAV productivity without compromising product quality is one of the most persistent challenges in gene therapy manufacturing," said Markus Hörer, Chief Scientific Officer at Ascend Advanced Therapies. "This enhancer represents a meaningful step forward – not just for our internal platform, but for our partners who depend on scalable and reliable AAV manufacturing. This advancement could allow twice as many patients to be treated per vector batch, reducing cost of goods."
Ascend plans to incorporate the enhancers into its internal process development and clinical manufacturing operations and make the technology available for external licensing. Patent protection has been filed covering the enhancer technology.
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