Gilbert, Arizona--(Newsfile Corp. - January 2, 2025) - A Race Against Blindness, an Arizona-based nonprofit organization, has awarded a $1 million grant to fund research and clinical trials focused on Bardet-Biedl Syndrome 1 (BBS1), a rare genetic disorder that leads to progressive blindness and metabolic complications in children.
Nonprofit funding sight-saving research for children.
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The grant will support Axovia Therapeutics, a UK-based biotechnology research company, in advancing the clinical trial of AXV-101, a gene therapy designed to address the vision loss and obesity associated with BBS1. The trial is scheduled to begin in the United Kingdom in mid-2025, with plans to expand to the United States in subsequent phases.
"This $1 million in funding proves what can be achieved when family and community unify around a singular cause for change," said Dr. Stephen Johnston, Co-Founder of A Race Against Blindness. "The support for this clinical trial is a symbolic milestone for the community affected by both vision loss and rare diseases."
Clinical Research to Address Bardet-Biedl Syndrome
Bardet-Biedl Syndrome (BBS) is caused by mutations in the BBS1 gene, affecting multiple organ systems and leading to significant vision loss and obesity. Retinal degradation, a hallmark of the syndrome, typically results in severe vision impairments or blindness by adolescence.
The AXV-101 clinical trials aim to halt the progression of vision loss by targeting the underlying genetic cause. Additionally, the therapy is designed to address metabolic dysfunctions linked to BBS1, potentially providing comprehensive treatment for those affected.
The funding from A Race Against Blindness helps enable Axovia Therapeutics to move forward with this critical research, marking an important step in the development of therapies for rare genetic disorders, including BBS and related conditions like retinitis pigmentosa.
Co-Founder, Stephen Johnston, with his son, Luke, who is going blind.
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A Personal Mission with Global Impact
A Race Against Blindness was founded by Dr. Stephen Johnston and Kristina Johnston when their son was diagnosed with BBS.
Through creative fundraising initiatives, the nonprofit has rallied thousands of supporters nationwide. These efforts have helped advance research into rare genetic conditions while raising awareness about the importance of addressing rare diseases.
"We began this journey to help our son, but it has grown into something far bigger," said Dr. Johnston. "Every dollar raised reflects hope. Our family will keep pushing, fundraising, and believing we can save the eyesight of children affected by RP/BBS, and eventually make even bigger impacts for the rare disease community as a whole. This journey continues."
The organization is deeply rooted in the rare diseases community, where research often goes underfunded. Through public support, A Race Against Blindness has demonstrated that meaningful change is possible even for the most challenging and overlooked medical conditions.
Advancing Rare Disease Research
The AXV-101 clinical trial will be a significant step forward in gene therapy for Bardet-Biedl Syndrome. This research highlights the broader applications of gene therapy in treating rare diseases. As advancements in genetic medicine continue, therapies like AXV-101 have the potential to transform the standard of care for conditions that previously had limited or no treatment options.
To learn more about A Race Against Blindness and its mission, and to join upcoming fundraising initiatives, please visit http://www.araceagainstblindness.org.
About A Race Against Blindness
A Race Against Blindness is a nonprofit organization based in Arizona dedicated to funding research and advancing treatments for progressive vision loss caused by rare genetic conditions such as Bardet-Biedl Syndrome (BBS) and retinitis pigmentosa (RP). Founded by Dr. Stephen Johnston and Kristina Johnston, the organization was inspired by their personal experience with their son's diagnosis of a vision-loss condition. A Race Against Blindness focuses on supporting clinical research, raising awareness, and engaging communities through creative fundraising efforts. By addressing the gaps in resources and attention often faced by rare diseases, the organization aims to bring hope and meaningful solutions to affected children and their families.
About Axovia Therapeutics
Axovia Therapeutics is a biotechnology research company focused on developing advanced gene therapies for ciliopathies, including Bardet-Biedl Syndrome (BBS). Specializing in adeno-associated virus (AAV) gene therapies, Axovia aims to address the root causes of genetic disorders by targeting neurological, metabolic, and retinal dysfunctions. With a commitment to improving the lives of patients and their families, Axovia is advancing innovative treatments like AXV-101, designed to prevent blindness and mitigate metabolic complications associated with BBS.
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Media Contact
Company Name: A Race Against Blindness
Contact Person: Dr. Stephen Johnston
Contact Number: (520)-548-1807
Email: steve@araceagainstblindness.org
Country: United States
Website: http://www.araceagainstblindness.org
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