Joshua Lorenz-Guertin | Vice President, Research & Development
Sharp Therapeutics
Dr. Joshua Lorenz-Guertin is Vice President of Research and Development at Sharp Therapeutics. He holds a Ph.D. in Molecular Pharmacology from the University of Pittsburgh, where he specialized in GABAA receptor neuropharmacology. During this time, he developed an innovative dual-fluorescence reporter in collaboration with Sharp's co-founders and authored multiple papers and reviews on this receptor’s pharmacology, trafficking, and genetics. His academic accolades include an NIH F31 award, conference prizes, international travel grants, and leadership in the ASPET Young Scientist Committee. Joining Sharp in 2019 as a Senior Scientist, he led high-throughput drug screening campaigns, including the Niemann-Pick disease type C program. Dr. Lorenz-Guertin implemented primary neural cultures and techniques, including CRISPR-Cas9, and contributed to Gaucher disease and frontotemporal dementia projects. Upon being promoted to Associate Director, he drove the GBA1 program, leading to the selection of '901 as a clinical candidate for Gaucher disease—a first for the company. In his current role as VP of R&D, he oversees scientific innovation, strategic priorities, drug discovery, and pipeline advancement, including guiding '901 through IND-enabling studies and clinical trials to benefit patients.
Appearances:
Day 1 - Tuesday 28 October @ 14:45
Development of Oral, Small Molecule Treatments for Genetic Diseases
Sharp Therapeutics (Sharp) is a preclinical-stage company developing small-molecule therapeutics for genetic diseases. Sharp’s discovery platform combines novel high throughput screening technologies, with compound libraries computationally optimized based on the physics and biology of cellular trafficking defects and allosteric activation of proteins. The platform produces small molecule compounds that restore activity in mutated proteins giving the potential to treat genetic disorders with conventional pill-based medicines. Sharp will highlight the status of their clinical targets and program pipeline, including Gaucher disease, Niemann-Pick disease type C, and familial frontotemporal dementia. Our most advanced program is the ‘901 clinical candidate for Gaucher disease, a lysosomal storage disease caused by a deficiency in the GBA1 product enzyme. We highlight the potential advantages of ‘901 over currently available therapies for Gaucher disease including reduced patient burden & safety, CNS penetrance, and discuss ongoing clinical trial planning. In summary, Sharp is developing a series of orally-available, small molecule drugs for the treatment of rare genetic disease and is progressing towards first in human clinical trials.
Day 1 - Tuesday 28 October @ 14:45
Development of Oral, Small Molecule Treatments for Genetic Diseases
Sharp Therapeutics (Sharp) is a preclinical-stage company developing small-molecule therapeutics for genetic diseases. Sharp’s discovery platform combines novel high throughput screening technologies, with compound libraries computationally optimized based on the physics and biology of cellular trafficking defects and allosteric activation of proteins. The platform produces small molecule compounds that restore activity in mutated proteins giving the potential to treat genetic disorders with conventional pill-based medicines. Sharp will highlight the status of their clinical targets and program pipeline, including Gaucher disease, Niemann-Pick disease type C, and familial frontotemporal dementia. Our most advanced program is the ‘901 clinical candidate for Gaucher disease, a lysosomal storage disease caused by a deficiency in the GBA1 product enzyme. We highlight the potential advantages of ‘901 over currently available therapies for Gaucher disease including reduced patient burden & safety, CNS penetrance, and discuss ongoing clinical trial planning. In summary, Sharp is developing a series of orally-available, small molecule drugs for the treatment of rare genetic disease and is progressing towards first in human clinical trials.
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