Skip to main content

The Batten Disease Support and Research Association (BDSRA) will present a special international edition of its virtual Ask-An-Expert series today. The webinar will explore the latest treatment developments for this group of rare inherited neurological diseases, with a focus on gene therapy.

The free interactive event, “Let’s Have a Conversation About NCL Biotechnology and Clinical Research: Exploring the Current Landscape,” will take place at 5:00 p.m. EDT and will feature a panel of Batten disease experts.

Go here to register for the webinar presented in collaboration with the international chapters and affiliates of the organization. The disorder, also known as neuronal ceroid lipofuscinosis (NCL), is thought to occur in one in 100,000 people worldwide.

“The Batten Disease Support and Research Association and its international chapters and affiliates would like to address the current landscape surrounding the biotechnology industry, some of the recent announcements regarding the development of gene therapy and how these changes affect our mission to support Batten families , to fund and facilitate research and advocate for treatments and a cure,” the BDSRA said in its announcement.

recommended reading

Batten disease is an inherited lysosomal storage disorder characterized by an accumulation of waste products inside cells. This interferes with normal cell function and ultimately leads to cell death.

There are currently no approved disease-modifying treatments, although several treatments are available to relieve symptoms, including vision problems, seizures, cognitive and communication problems, behavioral changes, heart problems, and heart problems. movement and coordination problems.

There are also several experimental Batten treatments that are in various stages of development. They range from those that can help with symptom management to therapies that could prospectively address the underlying genetic cause of the disease.

For example, stem cell therapies are a potentially viable treatment option, in addition to immunosuppressants and lysosomal therapies.

Gene therapy could also become a potential treatment for Batten. To restore normal function, such therapies would transport an intact, healthy version of the mutated gene to cells via a benign viral vector.

Several gene therapy efforts are underway. For example, a recent study showed that gene therapy safely and effectively attenuated signs and symptoms of CLN7 disease in cells and a mouse model, and children with CLN5 are being recruited for a first clinical study in humans. man who will evaluate the experimental Neurogene Therapy gene NGN-101.

The BDSRA noted, however, that the biotech market has recently been beset by industry-wide economic, clinical and regulatory setbacks that have led biotech companies, which develop these treatments, to cut staff, downsize and reprioritize their research and development pipelines.

“We know that some of the latest developments in the biotech industry will be distressing and extremely frustrating for many of our Batten families, but BRDSA and its partner organizations around the world remain committed to continuing to provide education, resources, support and communication to our Beat Families through all the ups and downs,” the organization noted in its announcement.