DISCLAIMER: This presentation was delivered at the Nashville Vision Seminar on February 10, This presentation is for informational purposes

Transcription

1 DISCLAIMER: This presentation was delivered at the Nashville Vision Seminar on February 10, This presentation is for informational purposes only and is copyrighted by the Foundation Fighting Blindness. While I did my best to deliver accurate information, the accuracy of information can t be guaranteed. Also, the information will inevitably change over time. Always consult a physician before undertaking any change in treatment. Thanks, Ben Shaberman 1

2 Retinal Disease Research Update Nashville Vision Seminar February 10, 2018 Ben Shaberman Director Science Communications 2

3 Era of Clinical Trials More than 25 Clinical Trials Underway For Inherited Retinal Disease Therapies Most IRD Studies Launched in the Last 5-7 Years Several Dozen Clinical Trials for AMD Dry and (Improved Wet) Treatments Leverage: FFB-Funded Research Attracting Commercial Investments 3

4 Gene Therapy So Far, So Good Therapeutic gene (i.e., cargo) AAV, Viral Delivery System (i.e., truck) 4

5 LUXTURNA Spark s RPE65 (LCA and RP) Gene Therapy First FDA-approved gene therapy for the eye or an inherited disease. 5

6 Gene Replacement Clinical Trials (partial list) Choroideremia Nightstar (vision benefits sustained for 3.5 years), Spark XLRP Nightstar (UK), MeriaGTx (UK), AGTC (US) Achromatopsia day blindness, AGTC and Tubingen Retinoschisis splitting of the retina, AGTC and NEI Usher syndrome type 1B Sanofi, lentivirus for delivery Stargardt disease Sanofi, lentivirus for delivery LCA (GUCY2D) University of Florida, Genzyme, planned 6

7 Optogenetic Therapies Restores light sensitivity to retina affected by advanced disease Two Clinical Trials: RetroSense (US), GenSight (UK) 7

8 Stem Cells Grow new retinal cells Replace or protect diseased retinal cells 8

9 Retinal Progenitors (neuroprotective for RP) Stem cells that have partially developed into retinal cells Henry Klassen, Ph.D., University of California, Irvine Injected into the vitreous release several growth factors Rescues cones 28 patients treated moved into Phase 2b 9

10 Retinal Progenitors (photoreceptor replacement for RP) ReNeuron (Mass Eye and Ear Infirmary) Transplant partially developed photoreceptors Functionally replace lost photoreceptors First patients treated Phase I/II Eric Pierce leading trial Significant funding from FFB 10

11 Opsis: Multi-Layered Retinal Patch Replacing RPE and Photoreceptors David Gamm University of Wisconsin Madison FFB-funded Induced pluripotent stem cells banked from super donors, whose immune system profiles closely match those of general public. 11

12 Two Major FFB-CRI Investments Nacuity (Dallas) up to $7.5 million NACA strong antioxidant to slow vision loss (RP, others) Developed at Johns Hopkins Similar to NAC, N-acetylcysteine (FDA-approved) SparingVision (France) up to 7 million RdCVF rod-derived cone viability factor (protein) Saves cones (RP, others) Developed at Institut de la Vision 12

13 Ophthotech Clinical Trials C5 Complement Inhibitor Complement part of the innate immune system Complement activation in some retinal diseases Complement attacks retina (RPE, subretinal space) Ophthotech has three Phase 2 Clinical Trials Dry AMD (US and Hungary) Wet AMD (US) Stargardt disease (US) 13

14 Benefits of Genetic Testing to Find Disease-Causing Gene Confirms diagnosis XLRP can look like cone-rod dystrophy Cone-rod dystrophy can look like Stargardt disease Recessive RP vs. dominant RP vs. XLRP not always clear Helps patient understand visual prognosis Confirms inheritance pattern (risk for children) Helps qualify people for clinical trials Helps identify future treatments that might work for patient e.g., vitamin A, DHA, lutein regimen Patients (and their families) want to know

15 Patient Registry Global, Free, Secure, Easy-to-Use Patient-Controlled 15

16 Resources (Ben)