News

Lexeo Therapeutics announced that it raised $100 million in a financing round to help support the development of pipeline therapies, including LX1004, a potential gene therapy for CLN2 disease, also known as late infantile Batten disease. Other potential treatments supported through this Series B financing include LX2006, a gene therapy for…

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

Taysha Gene Therapies has secured an exclusive option from the University of Texas Southwestern (UTSW) to license worldwide rights to a clinical-stage gene therapy program for CLN7 disease, a form of late-infantile Batten disease. A first-generation CLN7 therapy is currently being evaluated in a Phase 1 clinical trial…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

A new mutation found in the CTSD gene does not associate with the rare CLN10 form of Batten disease, despite occurring in a girl in China with CLN10-like symptoms, according to the results of a recent case report. Nonetheless, the discovery of this new mutation “has significance” and will…