List of RNA Therapies Companies in Israel - 2

Silexion Therapeutics is a pioneering clinical-stage oncology-focused biotechnology company engaged in discovering and developing proprietary treatments for KRAS-driven cancers. The company's platform technology is addressing difficult-to-treat solid tumors. Silexion's lead product candidate, SIL-204, consists of locally administered small interfering RNAs, or siRNA, in an extended-release formulation, as a first-line treatment of locally advanced pancreatic cancer patients combined with standard-of-care chemotherapy. SIL-204 is a siRNA product candidate following Phase 1 and Phase 2 clinical trials with Silexion's first-generation siG12D-LODER, also referred to as LODER™. Results from the LODER™ Phase 2 clinical trial showed a trend for differences between treatment groups with the LODER arm suggesting an overall survival advantage of 9.3 months. Silexion commitment to Pushing the boundaries of therapeutic advancements in the field of oncology is focused on its second-generation microparticle delivery system, SIL-204 a newly formulated product that will improve clinical efficacy. Silexion plans to conduct a Phase 2/3 trial of SIL-204 in locally advanced pancreatic cancer patients that harbor the KRAS G12D and G12V mutations. Silexion Therapeutics Ltd. is headquartered in Israel and partnered with well known GMP compliant siRNA facility, LGC Axolabs group located in Petaluma California, and worldwide experts extended-release formulation facility, EVONIK Corporation located in Birmingham, Alabama to upscale manufacturing.

SKIP Therapeutics develops RNA-based therapies for rare genetic disorders and common diseases – utilizing a proprietary computational discovery engine to optimize target selection and therapeutic design. We employ sequence-based molecules, called Antisense Oligonucleotides (ASO), to restore function of mutated genes in rare genetic disorders or to modify functionality of key genes in major disease pathways. Convergence of SKIP’s novel computational discovery engine alongside strong experimental capabilities form a unique venture in the landscape of RNA-based therapies. Thousands of rare genetic disorders affect millions of people worldwide, however the small number of patients for each disease, further segregated by patient-specific mutations, has hindered development of dedicated treatments. Recent advancements in RNA-therapeutics offer an expanded molecular toolkit that can be deployed rapidly and at low costs to restore protein function of mutated genes. Identifying sub patient-populations that could potentially be suitable for treatment with RNA-based therapeutics and matching molecular treatment-strategies across the vast landscape of known disease-causing mutations could be assisted by computational methods. To facilitate development of dedicated treatments, Skip Therapeutics has developed a bioinformatic pipeline that analyzes disease-causing mutations and recognizes the best molecular strategy (if any) to restore protein function, using splice modulating antisense oligonucleotides (ASOs). Analyzing mutation data at scale, enables identification of treatable patient cohorts and optimizes target selection. Top candidates from the computational analyses are experimentally validated and pursued for therapeutic development.