Amaurosis congénita de Leber (LCA)
La LCA es un grupo de enfermedades retinianas hereditarias que causan ceguera o una pérdida de visión grave en la niñez temprana.
Latest News
-
Atsena Receives $150 Million in New Financing to Advance XLRS and Preclinical Gene Therapy Programs
Research NewsThe company has reported encouraging results from its LCA1 and XLRS gene therapy clinical trials.
-
LCA4 Gene Therapy Restores Meaningful Vision for Blind Children
Research NewsMeiraGTx hopes to apply for marketing approval in the UK for the gene therapy.
-
Atsena’s LCA1 Gene Therapy Licensed by Nippon Shinyaku for Markets in US and Japan
Research NewsThe companies are planning a Phase 3 clinical trial for the promising gene therapy.
-
Opus Genetics, Ocuphire Merger to Boost Advancement of Several IRD Gene Therapies
Research NewsMerger will provide additional capital for gene therapy development.
-
BlueRock Therapeutics and Foundation Fighting Blindness announce collaboration to expand the Uni-Rare natural history study of patients living with inherited retinal diseases
Foundation NewsCollaboration will add a new multi-gene cohort of patients living with inherited retinal diseases. Data insights from the new study cohort will inform the future clinical trial design for BlueRock’s pipeline of cell therapies for treating blindness.
-
Foundation Funds 25 New Grants Totaling $15.1 Million in FY23
Research NewsThe global leader in retinal degenerative disease research supports a total of 93 research grants in its portfolio.
-
Foundation Launching its Largest Natural History Study to Date for 1,500 People with Inherited Retinal Diseases Caused by Rare Mutated Genes
Foundation NewsUni-Rare Study will improve clinical understanding of more IRDs and boost development of potential therapies.
-
Opus Genetics Appoints Jennifer Hunt Chief Development Officer
In the PressBiopharma clinical development veteran to propel Opus’ AAV-based gene therapies for inherited retinal diseases toward patients.
-
RD Fund Launches Opus Genetics with $19M Seed Funding to Advance Gene Therapy Treatments for Blinding Conditions
In the PressInitial programs will focus on treatments for rare pediatric blinding conditions. Company formed to advance the work of scientific cofounders Dr. Jean Bennett, Junwei Sun and Dr. Eric Pierce.
-
Atsena Therapeutics Raises $55 Million Series A Financing to Advance LCA1 Gene Therapy Clinical Program, Two Preclinical Assets, and Novel Capsid Development for Ocular Diseases
In the PressRound was led by Sofinnova Investments with participation from Abingworth, Lightstone Ventures and all existing investors.
Company expands board of directors and plans to build out team.
-
X Ambassadors Teams up with Foundation Fighting Blindness and Two Blind Brothers to Launch "Music to Our Eyes" Exclusive Livestream Music Series
Foundation NewsThis exclusive conversation and acoustic performance by Sam and Casey Harris of X Ambassadors on August 20, will raise awareness and funds to find treatments and cures for blinding diseases.
-
Atsena Therapeutics acquires exclusive rights to Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis
In the PressCompany formed with $8.15 million Series 1; led by founding investors Hatteras Venture Partners and the Foundation Fighting Blindness
-
COVID-19 Resources
Foundation NewsThe Foundation Fighting Blindness is closely monitoring the COVID-19 situation and its impact on the IRD community.
-
Seeing hope: Ledyard nonprofit focuses on rare retinal diseases
NewsSofia Priebe, 17, is legally blind. Her parents have started a nonprofit, Sofia Sees Hope, to raise funds for research into and awareness of the rare genetic disease Leber congenital amaurosis, which causes her blindness.
-
ProQR Therapeutics Teams Up with the Foundation Fighting Blindness and Blueprint Genetics to Support the My Retina Tracker® Program for People Living with Inherited Retinal Diseases
Foundation NewsMy Retina Tracker Program is the highest volume IRD genetic testing program in the U.S.
-
Blueprint Genetics, InformedDNA and the Foundation Fighting Blindness launch an open access program for patients with inherited retinal disease in the United States
Foundation NewsThe program will offer patients with inherited retinal disease no-cost genetic testing and genetic counseling in the United States. Look for updated information on how to participate to be posted in mid-October, with program registration starting shortly thereafter.
-
Foundation Fighting Blindness Investing Nearly $6.5 Million in New Grants
Foundation NewsThe newly funded research efforts include several therapies that have strong potential to treat a wide range of inherited retinal diseases.
-
Foundation Fighting Blindness Urges Congress to Pass ‘Eye-Bonds’ Legislation
Foundation NewsBill Introduced in U.S. House Would Speed Up Cures for Blindness
-
Foundation Fighting Blindness and CheckedUp® Partner to Educate Retinal-Disease Patients About Research, Resources, and Emerging Therapies During Doctor Visits
Foundation NewsThe Foundation Fighting Blindness (the Foundation) and CheckedUp have formed a collaborative partnership to deliver patient-friendly diagnostic and disease-management information to people with retinal diseases such as age-related macular degeneration, retinitis pigmentosa, and Stargardt disease during their visits to eye doctors.
-
A Retinal Research Nonprofit Paves the Way for Commercializing Gene Therapies
In the PressAN EMERGING, vision-restoring gene therapy for a devastating retinal disease is poised for Food and Drug Administration (FDA) approval. If it gets the regulatory nod, it will be the first gene therapy to receive FDA approval for the eye or an inherited condition.
Related Resources
ProQR Announces Virtual Presentations at Scientific Conferences
ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced virtual presentations at the Ophthalmology Futures Retina Forum, European Society of Retina Specialists (Euretina) congress and the Annual Meeting of the American Academy of Optometry (AAOpt).
Podcasts
-
Eye on the Cure Podcast | Episode 83: Michel Michaelides
Eye on the CureProfessor Michaelides talks about the extraordinary vision improvements for young blind children receiving gene therapy for LCA4
-
Eye on the Cure Podcast | Episode 82: Aaron Nagiel, MD, PhD
Eye on the CureDr. Nagiel discusses how gene therapy is administered to the retina, the surgery and recovery for patients, and the vision improvements for young patients receiving LUXTURNA, the first FDA-approved gene therapy for the eye or an inherited condition.
-
Eye on the Cure Podcast | Episode 73: Dr. Zuhal Butuner
Eye on the CureDr. Zuhal Butuner talks about her company’s two emerging RNA therapies for LCA10 and USH2A.
.png)
