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).
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
NeuExcell Therapeutics Unveils Breakthrough in Stroke Treatment at ASGCT 2024
In a groundbreaking development, NeuExcell Therapeutics has revealed a significant breakthrough in its NXL-001 product for stroke treatment. The announcement was made at the 2024 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, where the company...
Unraveling CRISPR Precision: BreakTag Illuminates Pathways to Improved Gene Editing
Introduction:The quest for precision in CRISPR-Cas9 gene editing takes a significant leap forward with the development of BreakTag, a method devised to enhance our understanding of DNA double-strand breaks (DSBs) induced by Cas9. This breakthrough, detailed in a...
Sumitomo Pharma Announces FDA Acceptance of Supplemental New Drug Application for Vibegron in Men with Overactive Bladder Symptoms Receiving Pharmacological Therapy for Benign Prostatic Hyperplasia
–Supplemental New Drug Application (sNDA) submission based on Phase 3 study of vibegron 75mg (GEMTESA) demonstrating statistically significant reductions in daily micturition and urgency episodes– –If approved, vibegron will be the first and only beta-3 agonist for...
Herpes cure with gene editing makes progress in laboratory studies
Herpes simplex virus. Credit: CDCResearchers at Fred Hutch Cancer Center have found in pre-clinical studies that an experimental gene therapy for genital and oral herpes removed 90% or more of the infection and suppressed how much virus can be released from an...
Related Services
