BDSRA Webinar Focuses on Latest Treatments, Research

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by Mary Chapman |

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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 in treatment development for this group of rare inherited neurological conditions, with an emphasis on gene therapy.

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

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

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

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

There currently are no approved disease-modifying therapies, although several treatments are available that can ease symptoms, which can include vision problems, seizures, cognitive and communication issues, behavioral changes, heart problems, and movement and coordination problems.

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

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

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

There are several gene therapy efforts underway. For example, a recent study showed that gene therapy safely and effectively eased the signs and symptoms of CLN7 disease in cells and a mouse model, and children with CLN5 are being recruited for a first-in-human clinical study that will evaluate Neurogene’s experimental gene therapy NGN-101.

The BDSRA noted, however, that the biotech market has been beset recently by sector-wide economic, clinical, and regulatory setbacks that have resulted in biotech companies, which develop these treatments, reducing staff, downsizing, and reprioritizing their research and development pipelines.

“We know some of the latest developments in the biotech industry will be distressing and extremely frustrating to 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 Batten families through all the ups and downs,” the organization noted in its announcement.