FDA lifts clinical hold on Rocket study
The FDA has lifted the clinical hold on Rocket Pharmaceuticals’ phase II trial of RP-A501, a gene therapy under investigation for Danon disease. The hold was initiated in May 2025 following the death of a patient who developed capillary leak syndrome after receiving the therapy.
Under the revised trial protocol, patients will receive a lower dose of RP-A501 at 3.8 × 10¹³ genome copies per kilogram. The therapy will be administered sequentially to three participants, with a minimum interval of four weeks between each dosing. The immunomodulatory regimen has also been modified: prophylactic use of a C3 complement inhibitor has been discontinued, while sirolimus, rituximab, and steroids remain part of the treatment plan. In addition, the threshold for initiating a C5 inhibitor has been lowered to allow earlier intervention if required.
The amended dosing and immune management strategy reflects findings from the phase I study, where lower doses demonstrated clinical activity with a more favorable safety profile.
Kite to acquire Interius BioTherapeutics
Kite, a Gilead company, says it will acquire Interius BioTherapeutics for $350 million. The acquisition centers on Interius’s in vivo CAR-T platform, which delivers genetic material directly to a patient’s T cells through a single intravenous infusion. The approach is designed to generate CAR-T cells within the body, removing the need for cell collection, genetic modification, and reinfusion that are required for conventional ex vivo CAR-T therapies. The in vivo platform also eliminates the requirement for preconditioning chemotherapy, offering the potential to simplify treatment administration and streamline manufacturing processes. Kite hopes the technology will reduce both the time and cost associated with CAR-T therapy development and delivery.
Following the completion of the transaction, Interius will be incorporated into Kite’s research operations in Philadelphia.
“In vivo therapy is a promising frontier with the potential to transform how we approach treating patients, shifting to more accessible and scalable solutions,” said Cindy Perettie, Executive Vice President of Kite. “By combining Interius’s teams and their novel platform with Kite’s deep expertise and footprint in cell therapy research, development and manufacturing, we aim to advance best-in-class in vivo therapies to bring them to patients more efficiently.”
Lack of funding forces Appia to close
Appia Bio CEO JeenJoo Kang says that the company has closed due to lack of funding. Appia was founded in 2020 to develop more broadly accessible allogeneic CAR T products and was close to filing an IND for clinical testing. A LinkedIn post from Kang says: “Looking back at the trail we have walked, I am grateful, proud, and sad to see the footprints on our path. I am grateful for the energy, time, and care of my colleagues -- our employees, the leadership team, our investors, our board and advisors. Thank you. I am proud of the distance and peaks of challenges that we have overcome to get here. And I am sad that, for now, we say good bye.”
The AA partnership
Andelyn Biosciences has entered into a partnership with Amplo Biotechnology to manufacture AAV–based gene therapies designed to address disorders of the neuromuscular junction. As part of the agreement, Andelyn will supply scalable, clinical-grade AAV material produced through its suspension-based AAV Curator platform, which is designed to support manufacturing consistency and efficiency. The partnership is intended to align Amplo’s therapeutic development efforts with Andelyn’s established capabilities in viral vector production, quality control, and regulatory-compliant processes.
FDA extends review for Regenxbio BLA
The FDA has extended the BLA review period for Regenxbio’s clemidsogene lanparvovec (RGX-121), an investigational one-time gene therapy for Mucopolysaccharidosis II (Hunter syndrome). The target date has moved from November 9, 2025, to February 8, 2026. The extension follows the company’s submission of 12-month data from its pivotal trial cohort of 13 patients in response to an FDA information request.
According to the company, the additional data are consistent with earlier findings related to both biomarker outcomes and neurodevelopmental measures. These results are scheduled to be presented at the International Congress of Inborn Errors of Metabolism in September 2025. The FDA has also completed a pre-license inspection and a bioresearch monitoring inspection without issuing observations, and no new safety concerns have been raised during the review process.
Three-year old patient treated with gene therapy for AADC defiency
Texas Children’s Hospital has treated its first patient with the newly approved gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. The therapy, eladocagene exuparvovec-tneq (Kebilidi), was delivered using a neurosurgical procedure that infused the treatment into specific areas of the brain. The three-year-old patient tolerated the six-hour surgery well, stayed in the hospital for two weeks of monitoring, and has since returned home. Doctors expect to see clear improvements in movement and development over the next several months.
AADC deficiency is a rare genetic disorder that prevents the brain from making essential neurotransmitters, causing severe motor and developmental problems. Only about 350 people worldwide are thought to be affected, and until Kebilidi’s approval in November 2024, there were no treatment options.
Texas Children’s played a leading role in the clinical trials that supported the therapy’s approval. The procedure was performed by Daniel J. Curry, Director of Functional Neurosurgery and Epilepsy Surgery.
Bacterial virus for delivery
Researchers at the University of Waterloo’s School of Pharmacy have developed a new gene delivery platform using a modified form of the bacterial virus M13. The system removes unnecessary viral and bacterial components, leaving a streamlined, single-stranded DNA phage capable of carrying therapeutic genes into human cells. By simplifying the genetic and structural design, the team believes both it will be more efficient and significantly less expensive than conventional approaches.
“There is a real need for customizable gene therapies to address the gap in treatments,” said Roderick Slavcev, a professor in Waterloo’s School of Pharmacy and the principal investigator of the study. "What’s exciting about M13 is that it is very simple genetically and structurally, a single-stranded DNA phage. This simplicity allows for a cost-effective, efficient, and controllable approach to gene therapy that may represent a step towards personalized gene therapy.”
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