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. 2021 Apr 1;10(4):23.
doi: 10.1167/tvst.10.4.23.

Tackling the Challenges of Product Development Through a Collaborative Rare Disease Network: The Foundation Fighting Blindness Consortium

Todd A Durham  1 , Jacque L Duncan  2 , Allison R Ayala  3 , David G Birch  4 , Janet K Cheetham  1 , Frederick L Ferris 3rd  5 , Carel B Hoyng  6 , Mark E Pennesi  7 , José-Alain Sahel  8   9   10 ; Foundation Fighting Blindness Consortium Investigator Group
Affiliations

Tackling the Challenges of Product Development Through a Collaborative Rare Disease Network: The Foundation Fighting Blindness Consortium

Todd A Durham et al. Transl Vis Sci Technol. .

Abstract

The Foundation Fighting Blindness, a 501(c)(3) nonprofit organization, established an international consortium of inherited retinal disease specialists in 2016, with a mission to accelerate the development of treatments for rare, inherited retinal degenerations, such as retinitis pigmentosa, Stargardt disease, Leber congenital amaurosis, Usher syndrome, choroideremia, and achromatopsia. The Consortium accomplishes its mission by evaluating novel outcome measures, sharing standardized study protocols and datasets, and disseminating findings. Having established research infrastructure in the first 3 years, including 39 global research sites, the network is now poised to expand its infrastructure for trials of new therapies in partnership with industry. This model represents an innovative approach to overcome challenges of therapeutic development for rare diseases.

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Conflict of interest statement

Disclosure: T.A. Durham, None; J.L. Duncan, Acucela, Abbvie/Allergan, Inc. (F), Biogen (F), and consultant fees from DTx Therapeutics (C), Eloxx, Gyroscope (C), ProQR Therapeutics, Inc. (C), Spark Therapeutics, Inc.(C), SparingVision, Second Sight Medical Products (C), Inc., Vedere Bio (C), Foundation Fighting Blindness (C), Astellas (C), AGTC (C), and Editas (C); A.R. Ayala, None; D.G. Birch, Nightstar, Inc. (F), Allergan (F), AGTC (F), Roche- 4D Molecular Therapeutics (F), ProQR Therapeutics (F), Foundation Fighting Blindness (F), AGTC (C), Nacuity (C), ProQR (C), Editas (C), Iveric, Roche(C); J.K. Cheetham, Foundation Fighting Blindness (C); F.L. Ferris III, receives support from Bausch and Lomb (F), Foundation for Fighting Blindness (F), JAMA Ophthalmology (F), Diabetic Retinopathy Clinical Research Network (F), Janssen Research and Development, LLC (F), Viewpoint Therapeutics, Inc. (F), EMMES (F), Genentech (F), Novo Nordisk, Apellis (F), Genentech/Roche (F), SemaThera, PTC Therapeutics (F), Regenera, Novartis (F), Eyevensys (F), Kodiak (F), Merck (F), 4D Molecular Therapeutics (F), and Clear Side Biomedical (F); C.B. Hoyng, received clinical support from Bayer (F), Gyroscope (F) Gemini (F) and is a member of advisory board of Allergan (S), Gemini (S) and Horama (S); M.E. Pennesi, receives support from FFB (F), clinical trial support from AGTC (F), Biogen (F), Editas (F), ProQR (F), Sanofi (F), is a consultant for: Adverum (C), AGTC (C), Allergan (C), Astellas Pharmaceuticals (C), Biogen (C), Blue Rock (C), Iveric Bio (C), Gensight (C), Novartis (C), RegenexBio (C), Roche (C), Spark Therapeutics (C), Viewpoint Therapeutics (C), advisory board for: Horama (S), Eyevensys (S), Nayan (S), Nacuity Pharmaceuticals (S), Ocugen (S), DTx Therapeutics (S), Vedere (S), Sparing Vision (S), Atsena (S), and Endogena (S); J.-A. Sahel, FFB (F) the Gilbert Family Foundation (F), Biogen (C), Spark (C), Stargazer (C), Atsena (C), Meira (C), Pixium Vision (C), GenSight (C), Novartis (C), Opsis, Savir (C), Tilak (C), Tilak (I), Pixium (I), Gensight (I), Sparing Vision (I)

References

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