List of Gene Therapy Companies in Israel - 5

Valuing Life, Granting Advanced Therapies to Patients- that Is Adva Biotechnology’s Vision and goal. Adva Biotechnology developed the ADVA-X3- a simple, smart, easy to use, automated, sensors-based, flexible cell manufacturing platform. The ADVA-X3 enables full manufacturing process based on the CAMP™ technology, of different applications, such as CAR-T, TCR, TIL, NK, Exosomes, Viruses and More.

ART BioScience was founded in December 2015 by Mr. Oren Ozeri and Mr. Alon Hazan, fathers of two children diagnosed with Duchenne. After co-founding the Israeli Duchenne Parent Association and increased involvement in following current research, Alon and Oren swore to do everything possible to save the lives of their children and dedicated their time and resources to trying to find a cure for the disease. The result of their intensive research and involvement in the field led them to the decision to establish ART, a company based on novel messenger RNA (mRNA) technologies for the development of new therapeutics that can potentially change the course of the disease. ​We are developing Dystrophin modified mRNA encapsulated in LNP (Lipid Nanoparticles) proprietary delivery system as: 1.) Protein Replacement Therapy 2.) CRISPR Cas9 mRNA as Gene Editing Therapy

At Gamida Cell, we are striving to enable patients with blood cancers and serious blood disorders to reach cures through advanced cell therapy. We work with a sense of urgency and deeply held responsibility to address the clear need for new treatment options. And we believe that cell therapies should be available to patients who can benefit from them.

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.

TargetGene is an innovative and agile biotechnology company in it’s development stage poised for expansion. TargetGene’s R&D is centered around creating efficient and supremely specific DNA editing solutions in living organisms. TargetGene, with it's patented and patent pending "T∙GEE"​ (Genome Editing Engine) platform is the first to invent RNA-guided gene-targeting. TargetGene is aiming to implement the "T∙GEE"​ platform for human gene-therapy with a focus on inherited diseases and cell-based immunotherapy. TargetGene’s platforms include In-Vivo assembling nucleases targeted to the cell's genetic material. Highly specific Targeting is achieved through use of the principle of nucleic acid hybridization. Keywords: Genome editing, Gene Targeting, Genome engineering, Nucleases, RNA-guided Genome Engineering (RGEN), Immunotherapy, Cell-based Therapy, Gene Thearapy, Immuno-oncology. Our new laboratories are situated in the Rehovot Science park, Oppenheimer 10, Rehovot, Israel.