Atsena Therapeutics is passionate about improving the quality of life of patients with vision loss. Our ocular gene therapy programs aim to reverse or prevent blindness in patients with GUCY2D-associated Leber congenital amaurosis (LCA1), X-linked retinoschisis (XLRS) and MYO7A-associated Usher syndrome (USH1B).
X-linked retinoschisis (XLRS) is a monogenic eye disease that causes progressive vision loss. We are leveraging one of our novel spreading capsids in a clinical program for XLRS.
GUCY2D-associated Leber congenital amaurosis (LCA1) is a genetic eye disease that affects the retina and results in early and severe vision impairment or blindness. Our ongoing Phase I/II clinical trial is evaluating a gene therapy, known as ATSN-101. We look forward to sharing updates on the advancement of this program into a pivotal clinical trial.
MYO7A-associated Usher syndrome (USH1B) is an inherited disease that affects the retina and the inner ear. We are evaluating a dual AAV vector-based gene therapy in a preclinical program to prevent blindness from USH1B.
While we develop life-changing gene therapies for inherited retinal diseases, we are committed to supporting patients and families affected by vision loss. Here are a few non-profit organizations that offer helpful resources to patients and families affected by inherited retinal diseases.
The mission of the Foundation Fighting Blindness is to drive the research for treatments and cures for inherited retinal diseases and bring together a passionate network of families to support this mission. Some of the Foundation’s valuable resources to support patients and families impacted by inherited retinal diseases are below. Find more at www.fightingblindness.org.
Educational Videos
Learn all about inherited retinal diseases, genetics, clinical trials, and related topics by watching or listening to the Foundation’s An Eye on Education video series.
Strength Through Communities
Find your local Foundation Fighting Blindness Chapter to connect with others impacted by inherited retinal disease, get access to local resources and learn how you can support the Foundation.
Genetic Testing
Learn more about genetic testing for inherited retinal diseases and how you may be able to receive genetic testing at no cost to you. Accurate and accessible diagnosis is critical to getting the treatment you need.
My Retina Tracker® Registry
With the goal of accelerating research and cures, the My Retina Tracker® Registry is a research database of people and families affected by rare inherited retinal degenerative diseases.
Low Vision Resources
Explore the latest advancements in vision-loss technology and disability and accessibility resources.
Hope in Focus is dedicated to generating awareness, raising funds for research, and providing support, education and outreach to the Leber congenital amaurosis (LCA) and rare inherited retinal disease community. A few of the great resources offered by Hope in Focus are below and can also be found at www.hopeinfocus.org.
Family Connections
Sometimes you just want to talk to someone who is in the same situation as you. Hope in Focus can help those with Leber congenital amaurosis (LCA) connect with another family or individual by phone or email.
Let’s Chat About…
Watch webinars from this free web series in easy-to-digest 30 to 40-minute Q&As with subject matter experts.
Atsena’s head of patient advocacy Kara Fick and founder Shannon Boye, PhD, participated in a Hope in Focus “Let’s Chat About…” webinar to discuss our approach to gene therapy and work in LCA. A replay can be viewed here.
The Usher Syndrome Coalition (www.usher-syndrome.org ) is the core of the global Usher syndrome community, working to connect those living with Usher syndrome to resources, research, and each other. The Coalition uses the power of two data collection tools to build the Usher syndrome community, leading to a better understanding of Usher syndrome, improved quality of life, and treatments for hearing, vision, and balance issues associated with Usher.
USH Trust
The USH Trust is the largest international contact database of individuals with Usher syndrome, and our most powerful tool to connect, inform, and support individuals within the Usher community. Those who join the USH Trust are also the first to learn about the latest research opportunities.
Usher Syndrome Data Collection Platform
Individuals with Usher syndrome who are passionate about research are invited to join the Usher Syndrome Data Collection Platform, or USH DCP, hosted by RARE-X. Health information entered into this secure platform will become part of a global database of de-identified information available to researchers worldwide. As the DCP grows, more researchers will become aware of Usher syndrome, leading to the development of clinical trials and treatments for the vision loss, hearing loss, and balance issues associated with Usher syndrome.
Connect with your community
USH Blue Book
The USH Blue Book is a private email group created to connect individuals with Usher syndrome, family members, and friends in a global network of support.
USH Yellow Book
A centralized directory of professionals worldwide, listed by location and profession.
Usher Syndrome Coalition Discord Server
Were you recently diagnosed with Usher syndrome? Are you looking to connect with others in the Usher community quickly and in real-time? The Discord Server is a private, accessible, online space where you can meet and chat with others.
To learn about gene therapy for inherited retinal diseases, click here to view an infographic from the American Society of Gene & Cell Therapy.
Contact Us
If you are interested in learning more about our activities within the patient community or wish to contribute ideas or questions, please reach out to our Patient Advocacy team at [email protected].
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