A New Wave of Hope: Transformative Gene Therapy Advances
A Breakthrough in Gene Therapy for Childhood Blindness
In a significant step towards fighting childhood blindness, A Race Against Blindness has recently announced a new $1 million grant awarded to Axovia Therapeutics. This funding aims to bolster the clinical development of AXV-101, a groundbreaking investigational gene therapy designed to tackle retinitis pigmentosa (RP) linked to Bardet-Biedl Syndrome 1 (BBS1). This initiative marks a pivotal moment not just for the organization but for all families affected by this condition.
The Increasing Impact of A Race Against Blindness
A Race Against Blindness, a nonprofit founded by Dr. Stephen Johnston and Kristina Johnston, has demonstrated extraordinary commitment to this cause. The recent grant is the third significant award allocated for the AXV-101 project, resulting in total funding of $4 million since the program's inception in 2024. This continuous support exemplifies the drive to find solutions that can alter the course for affected children.
What is AXV-101?
AXV-101 is a codon-optimized adeno-associated virus (AAV9) gene therapy aimed at halting retinal degeneration in BBS1 patients. This innovative approach is particularly exciting as it is set to begin the first-in-human open-label dose-escalation trial in the United Kingdom after receiving the necessary Clinical Trial Application (CTA) approval. Just imagine the possibilities as this therapy transitions from the lab to the clinic!
The Johnston Family's Personal Journey
The mission of A Race Against Blindness is deeply personal for the Johnston family. Their son Luke was diagnosed with retinitis pigmentosa due to genetic issues with the BBS1 gene, which led them to advocate for impactful therapies and research. Their dedication has galvanized thousands of supporters across the nation, all united in the quest for effective treatments.
Strong Community Support
Dr. Johnston expressed that the extraordinary support from their donors and sponsors is impactful, stating, "Every dollar given reflects a united commitment to ensuring children with BBS1 have a future where blindness is not inevitable." This echoes the core mission of the organization, which is to transform research into real-world therapies that save sight.
The Vision for Improved Therapies
The urgency of their mission resonates deeply within the community, reinforced by conversations with families and patients facing the daily challenges posed by BBS. Professor Phil Beales, CEO of Axovia Therapeutics, emphasized this point by saying, "Every conversation with patients and families in the BBS community reinforces the urgency and importance of developing new therapies." Such strong advocacy is essential as AXV-101 begins to enter into clinical trials.
About A Race Against Blindness
A Race Against Blindness is on a mission to fund cutting-edge research focusing on progressive vision loss associated with BBS and RP. The organization combines clinical research funding with awareness campaigns and community engagement, striving to bring innovative fundraising solutions to life. Their achievements are not just about raising money; they are about inspiring hope for affected families.
Contact Information
For inquiries, you can reach out to Dr. Stephen Johnston, Co-Founder & President of A Race Against Blindness. You can also connect via email at press@araceagainstblindness.org. Together, we can work towards a future free from the shadows of vision loss.
Frequently Asked Questions
What is the purpose of the $1 million grant?
The grant is designed to support the clinical development of AXV-101, a gene therapy that aims to combat childhood blindness caused by BBS1.
What is AXV-101?
AXV-101 is an investigational gene therapy using a codon-optimized AAV9 to stop retinal degeneration in BBS1 patients.
Who founded A Race Against Blindness?
Dr. Stephen Johnston and Kristina Johnston established the organization to pursue treatments for retinitis pigmentosa and other genetic causes of blindness.
How can people support A Race Against Blindness?
People can contribute by donating, raising awareness, or participating in community fundraising events organized by A Race Against Blindness.
When will clinical trials for AXV-101 begin?
The first-in-human clinical trial is expected to commence shortly in the United Kingdom following the clearance of the Clinical Trial Application.
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