Agnos Therapeutics Inc.

AGNOS THERAPEUTICS INC. — Grant R41EY038591

HUMAN IPSC DERIVED ORGANOID THERAPEUTICS FOR THE TREATMENT OF INHERITED BLINDNESS - PROJECT SUMMARY INHERITED RETINAL DISEASES (IRDS) ARE A GROUP OF GENETIC BLINDING DISORDERS THAT CAUSE PROGRESSIVE VISION LOSS, OFTEN RESULTING IN BLINDNESS BY EARLY TO MID-ADULTHOOD. IN ADDITION TO ECONOMIC BURDEN, BLINDNESS AND VISUAL IMPAIRMENT HAVE SEVERE IMPLICATIONS ON QUALITY OF LIFE FOR PATIENTS. THE MOST COMMON FORM OF IRD, RETINITIS PIGMENTOSA (RP), AFFECTS OVER 2 MILLION PEOPLE WORLDWIDE AND APPROXIMATELY 100,000 IN THE US ALONE. WITH CAUSATIVE MUTATIONS IDENTIFIED IN OVER 100 GENES SO FAR, THE GENETIC HETEROGENEITY UNDERLYING RP IS A MAJOR BARRIER TO TREATMENT. NO SINGLE GENE ACCOUNTS FOR MORE THAN 30% OF THE PATIENT POPULATION, HIGHLIGHTING A MASSIVE UNMET NEED FOR A GENE-AGNOSTIC THERAPEUTIC THAT CAN TREAT MANY FORMS OF RP. THE GOAL OF THIS PHASE I STTR PROGRAM IS TO DEVELOP AGN-001, A FIRST-IN-CLASS, GENE-AGNOSTIC, OFF-THE- SHELF RETINAL CELL THERAPY TO RESTORE PHOTORECEPTOR FUNCTION IN