Skip Therapeutics (“Skip”), developer of computational platform for design of Antisense Oligonucleotide (ASO) based therapies and Sheba Medical Center (“Sheba”), one of the leading medical centers in the world, announced today that they have entered a collaboration for the development of ASO-based treatments for rare genetic diseases.

Under the terms of the agreement, Sheba will share with Skip its de-identified patient mutation repository and Skip will provide a bioinformatic assessment of the possibility to use ASOs to circumvent the mutation, restore protein function and constitute a relevant therapeutic path.

Skip Therapeutics employs its proprietary bioinformatic engine to identify the most promising ASO-based molecular strategy for treatment of each patient sub-population based on their specific mutations. Skip’s approach has already identified promising targets for which it is performing in-vitro functionality studies with patient cells in collaboration with leading hospitals and academic institutes around the world.

“We are pleased to enter into a collaboration agreement with the world-renowned Sheba Medical Center for the development of ASO-based treatments for rare genetic diseases. Israel is and will always be at the forefront of innovation and it is our privilege and obligation, especially at times like this, to direct Israel’s ingenuity at developing new treatment options and advancing medicine,” said Ariel Feiglin, PhD, Chief Scientific Officer, Skip Therapeutics. “Thousands of rare genetic disorders affect millions of people worldwide. Most disorders are the result of a mutation in a single gene and have a deterministic genetic cause. However, the small number of patients for each disease has hindered development of dedicated treatments. Recent advancements in RNA-therapeutics offer an expanded molecular toolkit that can be deployed rapidly and at relatively low costs to restore protein function of mutated genes. This presents exciting possibilities of drug development for small patient cohorts and eventually even for single patients. To effectively realize these possibilities, it is necessary to incorporate computational tools in the developmental process, and we believe that our computational approach will enable an effective and rapid development of ASO-based treatments.”

Dr. Dan Dominissini, deputy director of the Center for Cancer Research and director of the Hematology Laboratory at Sheba Medical Center, stated, “We are delighted to collaborate with Skip Therapeutics for the development of cutting edge ASO-based therapies for genetic diseases. Sheba Medical Center is a world-leading center dedicated to incorporating and developing innovative techniques and approaches for the benefit of our patients. Skip Therapeutics’ novel computational engine will enables us not only to determine the most appropriate therapeutic design for a given genetic disease, but also to directly identify treatable patient populations, thereby maximizing efficiency and minimizing costs. We look forward to propelling gene therapy forward as a result of this exciting collaboration.”

 

About Antisense Oligonucleotide (ASO)

ASOs are short strands of synthetic, chemically modified, RNA sequences that can be designed to intervene in a molecular process called “splicing” which determines the final RNA sequence that will be translated into a protein. ASOs can be used to manipulate the splicing process and restore function of a mutated gene, by promoting exclusion of the mutated region from the final RNA transcript or by restoring normal splicing where it has been lost. This approach has already proved beneficial, among others, for treating Duchenne Muscular Dystrophy, Batten Disease and Spinal Muscular Atrophy.

 

About Skip Therapeutics

Skip Therapeutics develops Antisense Oligonucleotide (ASO) based therapies for rare genetic disorders and common diseases – utilizing a proprietary computational discovery platform to optimize target selection and guide therapeutic design. Skip’s ASOs aim to restore function of mutated genes in genetic disorders by modulating splicing or by controlling gene expression levels. For non-genetic disease, Skip is leveraging its platform to modify functionality of key genes in major molecular pathways using ASOs. ASO development programs for Pulmonary, Renal and Retinal indications are currently being pursued and evaluated on patient cells through collaboration with leading labs and medical institutions. Skip is a portfolio company of FutuRx bio-accelerator.

 

About Sheba Medical Center

The largest and most comprehensive medical center in the Middle East, Sheba Medical Center, Tel Hashomer is generating global impact through its medical care, research and healthcare transformation. Sheba’s City of Health boasts an acute-care hospital, rehabilitation hospital, research and innovation hubs, medical simulation center and center for disaster response on one comprehensive campus in the center of Israel. Sheba serves as a true hospital without borders, welcoming patients and healthcare professionals from all over the world and consistently providing the highest-level medical care to all in need. Sheba has been ranked a World’s Best Hospital by Newsweek five years in a row (2019, 2020, 2021, 2022, 2023).

Source: https://www.prnewswire.com/news-releases/skip-therapeutics-and-sheba-medical-center-to-collaborate-for-the-development-of-rna-based-treatments-for-rare-diseases-301994662.html
About PackGene

PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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