D26AC50003

THIS PROJECT PLATFORM WILL MITIGATE CURRENT DRUG-BASED TREATMENTS' LIMITATIONS BY AVOIDING OFF-TARGET AND SYSTEMIC SIDE EFFECTS WHILE PROVIDING A NOVEL, SCALABLE APPROACH TO CLINICAL APPLICATIONS WITH UNPARALLELED PRECISION AND SAFETY. A SONOGENETIC METHOD WILL BE USED TO ALLEVIATE SYMPTOMS OF A CLINICALLY RELEVANT INDICATION IN MICE AND LARGE ANIMAL MODELS.

San Diego, California Salk Institute For Biological Studies

TRADITIONAL WESTERN MEDICINE RELIES HEAVILY ON PHARMACEUTICAL APPROACHES USING DRUGS TO MANIPULATE CELL FUNCTIONS, BLOCK PATHOGENIC MECHANISMS, AND/OR PROMOTE RECOVERY OF DAMAGED TISSUES. HOWEVER, TWO FACTORS PLAGUE DRUG-BASED APPROACHES. FIRST, DRUG TARGETS CAN BE EXPRESSED IN MULTIPLE TISSUES (NOT ALL OF WHICH NEED MANIPULATION) LEADING TO OFF-TARGET EFFECTS. SECOND IS THE LACK OF TEMPORAL SPECIFICITY ? WITHOUT PRECISE ON/OFF CONTROL OF DRUG ACTIVITIES, ADDITIONAL UNWANTED SIDE EFFECTS OCCUR. SALK INSTITUTE HAS PIONEERED SONOGENETICS, WHICH USES MECHANOSENSITIVE ION CHANNELS (SONOCHANNELS) TO MODULATE CELLULAR FUNCTION NONINVASIVELY VIA ULTRASOUND. BECAUSE ULTRASOUND READILY PENETRATES SKIN AND UNDERLYING TISSUE, INCLUDING BONE, SONOGENETICS CAN BE USED TO TARGET ORGANS AND CELL TYPES ANYWHERE IN THE BODY. IT CAN ALSO BE DELIVERED OVER VERY SHORT OR LONG-TIME SCALES, WITH EXTREMELY RAPID ON/OFF KINETICS. AS A CLINICAL PROOF-OF-CONCEPT, SALK WILL TARGET PERIPHERAL SENSORY NEURONS AND USE ULTRASOUND TO PROMOTE NERVE REGENERATION AND ALLEVIATE NEUROPATHIC PAIN.

93.384 - ADVANCED RESEARCH PROJECTS AGENCY for HEALTH (ARPA-H)

Interior Business Center (IBC) [DOI]

La Jolla, California 92037-1002 United States

Single Zip Code

ARPA-H Open Office BAA (75N99223S0001)