NEW YORK – EyeDNA Therapeutics this week said its investigational gene therapy for PDE6b-mutated retinitis pigmentosa, HORA-PDE6b, has received rare pediatric disease designation from the US Food and Drug Administration. PDE6b retinitis pigmentosa is caused by PDE6b gene mutations, which lead to dysfunctional proteins that hinder the eye from converting light into electrical signals, often leading to blindness. There are no approved treatments for this rare, inherited retinal dystrophy.
HORA-PDE6b uses an adeno-associated virus 5 vector to deliver a nonmutated functional version of the PDE6b gene into the subretinal space, aiming to induce transgene expression and create functional PDE6b proteins in photoreceptor cells. EyeDNA is currently testing the gene therapy in a Phase I/II trial, hoping to show that HORA-PDE6b will delay or stop retinal degeneration in PDE6b-deficient patients, and it is exploring ways of achieving accelerated approval in the US and Europe. With rare pediatric disease designation, if HORA-PDE6b is approved, EyeDNA is eligible for a priority review voucher, which it can use to expedite review of another regulatory application or sell the voucher to another company.
Atsena Therapeutics this week said it has dosed nine patients in its Phase I/II clinical trial of a gene therapy for X-linked retinoschisis (XLRS) caused by mutations in the RS1 gene, a rare disease that leads to blindness.
In the LIGHTHOUSE trial, Atsena is evaluating the safety and tolerability of ATSN-201, which uses the company's novel adeno-associated virus capsid to deliver and spread a normal copy of the RS1 gene from the subretinal injection site to photoreceptors in the central retina. Nine adults were treated with three dose levels of ATSN-201 in Part A, clearing the way for the next stage of the study. Overall, the open-label, dose-escalation and dose-expansion clinical LIGHTHOUSE trial is to enroll 21 male patients 6 years old and older.
The Huntsman Cancer Institute at the University of Utah this week said it will participate in the myeloMATCH clinical trial sponsored by the National Cancer Institute. Within the trial, patients with acute myeloid leukemia and myelodysplastic syndrome are matched to a treatment arm based on genomic profiling and are assigned to different tiers of treatment based on their responses to therapies monitored using measurable residual disease testing.
Eli Lilly this week said that its beta-amyloid-targeting drug Kisunla (donanemab) has been approved by the National Medical Products Administration in China as a treatment for early Alzheimer's disease. Kisunla was approved in the US in July and has since been approved in Japan and Great Britain, as well.
The Medical University of South Carolina this week said it will expand its cell therapy programs and establish a new Integrated Center for Cellular Therapy to integrate research and clinical activities. The new facility, which is expected to open in early 2027, will house twice as many faculty and extend cell therapy services throughout the MUSC Regional Health Network and its affiliates.
Tempus this week said that it has signed agreements for in-network provider status with Blue Cross Blue Shield of Illinois, Blue Shield of California, and laboratory benefit manager Avalon Healthcare Solutions. Under its agreement with Avalon, Tempus is a participating provider with BCBS North Carolina, BCBS South Carolina, BCBS Vermont, Capital BlueCross, and CareSource in four markets. Patients with these insurance plans will now have access to Tempus' comprehensive molecular profiling portfolio.
Lucid Diagnostics said this week that it has formed a partnership with the first responder-focused healthcare provider VitalExam to offer firefighters access to Lucid's EsoGuard Esophageal DNA Test that is used for the early detection of esophageal precancer. Lucid noted that it has been pursuing partnerships through a direct contracting initiative to increase near-term revenues from the EsoGuard assay and complement the company's efforts to secure reimbursement for the test. In September, the firm also inked a deal with Front Line Mobile Health to explore opportunities to promote EsoGuard among firefighters and other first responders.
Roswell Park Comprehensive Cancer Center this week opened its new cell and gene therapy manufacturing center. The $98 million GMP Engineering and Cell Manufacturing, or GEM, facility is designed to support cancer cell therapy research and development at Roswell Park and is the first cell and gene therapy hub in the state of New York. The GEM facility, which includes 20 specialized clean rooms, is supported by a $30 million investment from Empire State Development.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.