12/4/2015 Financial Disclosures Treatments and Clinical Grant Support Consultant/Scientific Advisory Board Trials for Retinal � Foundation Fighting Blindness � Foundation Fighting Blindness � USFDA Office of Orphan Product � Spark Therapeutics Degenerations Development � AGTC � Lowy Medical Research Institute � Shire � Research to Prevent Blindness � California Institute for Jacque Duncan, M.D. � The Claire Giannini Foundation Regenerative Medicine UCSF Kearn Family Center for the � That Man May See, Inc. � UC Irvine/jCyte � Lawrence L Hillblom Foundation � Ocugen Study of Retinal Degeneration � Neurotech USA, Inc. � Okuvision December 4, 2015 � Sucampo AG � ISIS Pharmaceuticals Retinal Degenerations Recent Developments � Treatments for Patients with Severe � Diverse group of inherited diseases Vision Loss: � All associated with progressive loss � Restore Vision of photoreceptors � Prosthetic Devices � Stem Cells � Emerging technologies provide � Cure the Problem: Gene Therapy hope for patients with severe vision loss 1
12/4/2015 Retinal Prostheses Visual Prosthetic Devices Subretinal Prostheses Epiretinal Prostheses � Bypass the missing photoreceptors � Stimulate the retinal cells the photoreceptors talk to: bipolar and ganglion cells � Identify position, orientation, motion of objects � Identify windows, doorways Retina Implant AG TM Second Sight, Model I TM Second Sight Argus II System Retina Implant AG Alpha IMS System � 3x3 mm 2 subretinal microchip � 1,500 electrodes, no external camera � July 3, 2013: CE Mark approval granted � Clinical trial recruiting in Europe Humayun M et al, Ophthalmology 2012: 119; 779-788 2
12/4/2015 Second Sight Argus II System Stem Cell Therapy FDA Approval 2/14/2013 � March 2, 2011: CE Mark approval granted for Argus II � Potential to restore vision that has been System lost if stem cells are used to replace vision � February 14, 2013: US FDA grants Humanitarian Use cells Device Exemption � Current trial to prevent further vision loss in � January 2014: first commercial devices implanted at University of Michigan dry age-related macular degeneration and � Offering Argus II at UCSF: Stargardt disease by replacing RPE cells � Contact Scott Miller to refer patients who are NLP due � Clinical Trial at UCLA, BPEI, Mass Eye and Ear, to RP at 415-353-2865, fax 415-514-5870 Wills underway www.ClinicalTrials.gov Identifier: NCT01345006 www.ClinicalTrials.gov Identifier: NCT01344993 Leber’s Congenital Amaurosis � Early onset, severe vision loss � Many Genetic Mutations: � AR: RPE65 , AIPL1, TULP-1, GUCY2D/RETGC, CRB1, CEP290, LCA5, RDH12, RPGRIP1 � Account for 70% of identifiable mutations � AD: CRX � Genetic Testing: Project 3000 � 9 patients: STGD and AMD � www.carverlab.org � Stem cells survive in subretinal space up to median 22 months � No adverse safety events � Vision improved in treated eyes Schwartz SD et al, Lancet 2012: 379; 713-20 Schwartz SD et al, Lancet 2015: 385; 509-16 3
12/4/2015 LCA: RPE65 Gene Therapy RPE65 Phase 3 trial update � 3 Groups: 2 at U Penn, 1 at UC London, 3 pts � Subretinal AAV2 each � Sites: � Phase I safety results presented in May 2008 � University of Iowa: (PI: Stephen Russell) � Gene Therapy with RPE65 was well tolerated � CHOP/Penn (PI: Albert Maguire) � 1/3 in London and 3/3 at U Penn gained vision � Update: � Visual acuity � Spark, Therapeutics reported results from Pivotal � Visual field phase 3 clinical trial of SPK-RPE65 10/5/15 � Less nystagmus � Ability to move around Effective Treatment, but not a cure… SPK-RPE65 � First Randomized, Controlled Phase 3 trial of a gene therapy for a genetic disease � Met primary and 2 secondary endpoints � Demonstrated restoration of functional vision: change in bilateral mobility testing at 1 year � Also showed improvement in light sensitivity � Plan to file Biologics License Application with USFDA in 2016 Jacobson et al, NEJM 2015: 372; 1920-6 4
12/4/2015 Retinal Degenerations Update: � New treatments for retinal degenerations show potential to improve vision for patients with different stages of vision loss: � Prosthetic devices � Stem Cell trials: Stargardt, AMD � Gene Therapy: Leber’s Congenital Amaurosis � Emerging Technologies Provide Hope for Patients with Severe Vision Loss! Bainbridge et al, NEJM 2015: 372; 1920-6 5
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