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Orchard Therapeutics is a leading global fully integrated commercial-stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies. Orchard’s portfolio of autologous ex vivo gene therapy programs has demonstrated sustained clinical benefit in over 150 patients across five disease areas. These programs include Strimvelis®, the first autologous ex vivo gene therapy approved by the EMA in 2016, 3 programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (Wiskott Aldrich syndrome) and ADA-SCID (adenosine deaminase severe combined immunodeficiency), 2 other clinical programs in X-CGD (X-linked chronic granulomatous disease) and beta-thalassemia, as well as an extensive preclinical pipeline. The company is partnered with world-leading institutions in gene therapy, including University College London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University Hospitals, the University of California Los Angeles and Boston Children’s Hospital, and Telethon Institute of Gene Therapy/Ospedale San Raffaele. Orchard is a publicly traded company (NASDAQ: ORTX) with offices in the UK and the US, including London, San Francisco and Boston.
EpiVax Oncology is developing precision cancer immunotherapies based on mutanome-directed neo-epitopes, selected using proprietary and validated in silico predictive algorithms. Neo-epitopes are mutated and tumor specific epitopes, recognized as non-self, capable of generating highly potent specific T cell immunogenic responses against tumors, translating in improved clinical outcome. EpiVax Oncology is developing neo-epitope based therapeutic vaccines customized and specifically designed for each cancer patient, based on the genetic profiling (using next generation DNA sequencing) of each patient`s unique tumor and normal genome sequence (the mutanome). EpiVax Oncology`s ultimate purpose is to address major unmet medical needs inadequately addressed by other oncology and immuno-oncology treatments which are either only benefiting a very limited subset of patients, or prone to adverse events, or extremely costly and complex to manufacture. EpiVax Oncology`s ambition is to become a premier cancer precision medicine company, leveraging EpiVax`s world class excellence in computational-immunology, genomics and vaccine design, built over the last 20 years.
Pillar Biosciences aims to "Make precision medicine the first option for every patient" by developing and manufacturing targeted next-generation sequencing (NGS)-based assays and software for today`s high-throughput specialty NGS laboratories. Through our SLIMamp technology (a multiplex overlapping PCR chemistry) Pillar offers a streamlined, robust and economical workflow with high mapping and on-target metrics. In conjunction with the SLIMamp assay technology, Pillar Biosciences has an informatics pipeline called the Pillar Variant Analysis Toolkit (PiVAT) that is not only highly accurate, enabling somatic variant calls down to 1% allele frequency without use of Unique Identifiers (UIDs), but also fast and efficient, returning valuable sample data quickly. For higher-sensitivity applications such as cell-free DNA analyses, Pillar Biosciences has developed a dedicated assay technology for cell-free DNA that uses UID methodology, to work with the PiVAT pipeline to drive sensitivity down to 0.1%-0.2%. Current as of May 25, 2021.
Roche NimbleGen, Inc. is a Madison, WI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Axcella is designing and developing AXA Candidates, compositions of endogenous metabolic modulators, or EMMs, engineered in distinct ratios, designed to target and maximize the fundamental role that EMMs play in regulating multiple metabolic functions. Axcella`s AXA Candidates are generated from its proprietary, human-focused AXA Development Platform. Axcella believes its expertise and capabilities in EMMs position it to become a preeminent biotechnology company reprogramming metabolism to address a diverse set of complex diseases and support health. Axcella`s AXA Development Platform has already produced a pipeline of product candidates in programs targeting liver, muscle and blood. Axcella was founded by Flagship Pioneering.