The European Medicines Agency (EMA) has designated Neurogene’s gene therapy candidate for CLN5, a form of late childhood Batten disease, an orphan drug as a potential treatment for the disease.
“This regulatory designation is an important step in addressing the urgent and unmet need of families and patients living with CLN5, and follows the orphan drug designation previously granted in 2020 by the U.S. Food and Drug Administration (FDA). United for this same gene therapy candidate,” Rachel McMinn, PhD, Founder and CEO of Neurogene, said in a press release.
Experimental gene therapy uses a harmless adeno-associated virus (AAV) to deliver a functional copy of the CLN5 gene to a patient’s cells. AAVs are common carrier vehicles for gene therapies because they can easily enter cells, including cells in the brain and spinal cord, making them particularly useful for neurological disorders such as Batten disease. .
Symptoms of CLN5 and other types of late childhood Batten disease include seizures, progressive loss of motor and intellectual abilities, loss of vision, and premature death.
“With no treatment options available, the outcome is devastating for children and their families,” said Angela Schulz, MD, PhD, of University Medical Center Hamburg-Eppendorf in Germany.
Although the exact function of CLN5 remains unclear, mutations in the cause waste molecules called lipofuscins to build up to toxic levels inside cells, eventually killing them. Nerve cells seem particularly susceptible to damage caused by lipofuscins.
Preclinical studies have found evidence that CLN5 plays a role in fat metabolism and in maintaining myelin, the fat-rich substance that insulates nerve fibers. It can also affect protein transport and embryonic development.
Neurogene is investigating therapies for CLN5 and CLN7, another late childhood form of Batten disease. The company is currently conducting a natural history study (NCT03822650) of these disorders in collaboration with UT Southwestern Medical Center.
Instead of testing the safety and effectiveness of a given medical intervention, natural history studies are observational in nature, intended to help researchers better understand the course of a disease – in this case, CLN5 and CLN7 – so they can determine the most relevant results. to be measured in future clinical trials.
Orphan drug designation means Neurogene’s gene therapy can benefit from development incentives such as trial protocol assistance, reduced regulatory fees and 10 years of market exclusivity if the therapy receives regulatory approval.
“Neurogene is collaborating with Batten disease experts, regulatory authorities and healthcare providers to deliver safe and effective gene therapy for this disease as quickly as possible,” McMinn said.