News

Winners of the Australia and New Zealand Batten Disease Research Grant for 2021 have been announced by the Batten Disease Support and Research Association (BDSRA) Australia, the program’s sponsor. They are Anthony White, PhD, an associate professor and principal research fellow at QIMR Berghofer Medical Research Institute in Brisbane, Australia, and Anthony…

A screening of U.S.-approved therapies revealed that tamoxifen, an oral cancer therapy, is able to effectively reduce the accumulation of a fatty molecule called globotriaosylceramide, or Gb3, in cellular models of two forms of Batten disease. It worked without compromising cell survival, a study showed. That study found that…

Enrollment in a clinical trial testing NGN-101 — an experimental gene therapy for CLN5, a type of late-infantile Batten disease — is expected to start early next year at the University of Rochester Medical Center (URMC). “This trial will move research forward in developing a potentially disease-modifying treatment for…

Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease access to care. During the pandemic, governors used emergency authority to waive certain state licensure requirements, giving healthcare providers…

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…