BioScience Forum Presents: Precision CRISPR Therapies

Optimizing potency, specificity, and deliverability is critical in the development of all therapeutics, and perhaps especially so for those leveraging CRISPR technology to deliver in vivo genetic and epigenetic modifications. Scribe has established an approach for doing so, called CRISPR by Design™, to optimize its internal portfolio focused on cardiometabolic diseases as well as corporate collaborations in neurological, neuromuscular, and sickle cell diseases.

Using a novel, easy-to-deliver, and relatively safer starting point — CasX — the company has designed and engineered two platforms with the potential to become standard of care for genetic medicines: ELXR for epigenetic silencing of multiple genetic targets without cutting DNA, and XE for direct gene editing with no detectable off-target editing.

The lead program STX-1150 utilizes the Epigenetic Long-Term X-Repressor (ELXR) as the first CRISPR epigenetic editor that potently and durably silences the liver PCSK9 gene and has been shown to lower low-density lipoprotein cholesterol (LDL-C) after therapeutic dosing of <1mg/kg in non-human primates. This approach could offer a more effective alternative to existing LDL-C lowering treatments, particularly for patients at high risk of atherosclerotic cardiovascular disease (ASCVD). In addition, the STX-1200 program uses the XE (X-Editor) based approach to directly target the Lipoprotein(a) gene and has demonstrated >90% knockdown of Lipoprotein(a) levels in preclinical models. The strategy for in vivo delivery has included both adeno-associated virus (AAV) and lipid nanoparticles (LNP).

Dr. Khakoo will discuss the technical challenges and opportunities of the approaches utilized by Scribe across their pipeline, the latest preclinical data driving the programs forward, and steps being taken towards clinical evaluations.

Info: Network with local bioscience professionals while enjoying appetizers and drinks.

Info: Scientific presentation by distinguished guest speaker followed by a lively Q&A.

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