Ionis Pharmaceuticals, Inc. engages in the development and commercialization of human therapeutic drugs using antisense technology. It operates through the Ionis Core segment. The Ionis Core segment exploits a novel drug discovery platform to generate a pipeline of drugs. The company was founded by Stanley T. Crooke, David J. Ecker, Christopher K. Mirabelli, and Brett P. Monia in 1989 and is headquartered in Carlsbad, CA.

Alnylam is the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States. Incorporated in 1980 as AntiVirals, shortly before going public the company changed its name from AntiVirals to AVI BioPharma soon with stock symbol AVII and in July 2012 changed name from AVI BioPharma to Sarepta Therapeutics and SRPT respectively. As of 2018, the company has one approved drug (see the Products section below).

Part of uniQure NV, uniQure IP BV is a Dutch company that provides research and development services of human gene based therapies.

Voyager Therapeutics is a gene therapy company developing treatments for fatal and debilitating diseases of the central nervous system (CNS). It is committed to advancing the field of AAV (adeno-associated virus) gene therapy through innovation and investment in vector optimization and engineering, dosing techniques, as well as process development and production. The company’s initial pipeline is focused on CNS diseases in dire need of effective new therapies, including Parkinson’s disease, a monogenic form of amyotrophic lateral sclerosis (ALS), and Friedreich’s ataxia.

Neurimmune Holding AG is a private company headquartered in Schlieren, Switzerland, with domestic subsidiaries, that manufactures pharmaceutical products. The firm was founded in 2006.\nThe company says this about itself: Neurimmune is a biopharmaceutical company translating human immune memory into transformative antibody therapeutics. Neurimmune develops drug candidates for CNS and related protein aggregation diseases including Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, frontotemporal dementia, dementia with Lewy bodies and ATTR cardiomyopathy. Neurimmune discovered aducanumab, a human monoclonal antibody that removes amyloid beta from brains of patients with Alzheimer's disease, and licensed it to Biogen. With its RTM™ technology, Neurimmune also discovered the anti-miSOD1 antibody AP-101 for ALS and the anti-ATTR antibody NI006 for ATTR cardiomyopathy, programs being currently evaluated in clinical trials. Neurimmune has three additional antibody programs in preclinical development, and has recently expanded the spectrum of its treatment modalities by adding a small molecule program and programs involving vectorized expression of human antibody genes.

AC Immune SA is a clinical-stage biopharmaceutical company, which engages in the discovery and development of therapeutic and diagnostic products for neurodegenerative diseases. It leverages proprietary technology platforms to discover, design, and develop novel, proprietary medicines for prevention, diagnosis, and treatment of neurodegenerative diseases associated with protein misfolding. The company was founded by Jean-Marie Lehn, Claude Nicolau, Roscoe Brady, Fred van Leuven, Ruth Greferath, Andrea Pfeifer, and Alexey V. Eleesiv on February 13, 2003 and is headquartered in Lausanne, Switzerland.

Chase Therapeutics is an early-stage, product-based specialty pharmaceutical company focused on the development of better treatments for brain disease.

Sanofi Genzyme is the specialty care global business unit of Sanofi, focused on rare diseases, multiple sclerosis, immunology, and oncology. They help people with debilitating and complex conditions that are often difficult to diagnose and treat. They are dedicated to discovering and advancing new therapies, providing hope to patients and their families around the world. Genzyme became part of Sanofi in 2011. While continuing to focus on rare diseases, they also established ourselves as a leader in the field of multiple sclerosis. In 2016, their focus expanded to include Sanofi’s programs in oncology and immunology. Their approach is shaped by a long history of developing highly specialized treatments and forging close relationships with physician and patient communities. Founded as Genzyme in Boston in 1981, they rapidly grew to become one of the world’s leading biotechnology companies, recognized for their pioneering development of treatments for rare genetic disorders and their innovative contributions to medical science and biomanufacturing.

Shanghai Shengyuan Gene Development Co., Ltd., established on 1998-03-18, Business scope includes bioengineering,Technology transfer in the field of genetic engineering,to develop,consult,Production, supply and marketing of services and related reagents and equipment,.

Shanghai Bode Gene Development Co., Ltd., established on 1998-03-22, Business scope includes bioengineering,Technology transfer in the field of genetic engineering expertise,technology development,technical consulting,Technical services and manufacture of related reagents and equipment,Sales;Bioengineering and Genetic Engineering Products,electronics,Chemical Products(In addition to dangerous goods)import and export business. [Items subject to approval by law,Business activities can only be carried out after approval by relevant departments]

The Board of Regents of The University of Texas System, a subsidiary of The University of Texas at Austin, is an American company located in Austin, TX. The firm oversees college activities.

The University of California is the nation's best public university system, delivering top-notch research and innovation to California (and the world).

Institut National de la Sant? et de la Recherche M?dicale provides medical services. The firm specializes in biomedical research and public health research. The company is headquartered in Paris, Cedex, France.

Hangzhou Puti Biotechnology Co., Ltd., established on 2013-03-29, Business scope includes technology research and development,Technical Services,Technical consultation and transfer of technological achievements:Biological product,medicine,Chemical Products;wholesale,retail:cosmetic,Chemical Products(Removal of hazardous chemicals and precursors),Laboratory equipment,medical instruments(Limited to one category,Class II),laboratory consumables;Import and export of goods and technology(law,Except for items prohibited by administrative regulations,law,Projects that are restricted by administrative regulations can only be operated after obtaining a license).

Shorewood Intellectual Property Holdings BV, a subsidiary of WestRock Co., is a company headquartered in the Netherlands. The firm provides investment services.

Scribe Therapeutics is a biotech company that develops and engineers new therapeutics based on CRISPR. The company’s first technology, called X-Editing (XE) molecules, are highly engineered CRISPR enzymes that provide combined aspects of greater efficacy, specificity, and deliverability than currently available CRISPR genome editing technology. It also a long-term, engineering approach that can help establish CRISPR-based therapies as a new standard of clinical care and expand access to groundbreaking treatments that will impact millions of lives. Scribe Therapeutics is overcoming the limitations of current genome editing technologies by developing custom-engineered enzymes and delivery modalities as part of a proprietary, evergreen platform for CRISPR-based genetic medicine. The company built for exceptional specificity within the genome – enabling therapeutic precision and allele-specific targeting. Scribe Therapeutics was founded in 2018 and is headquartered in Alameda, CA, USA.

Prilenia Therapeutics Development Ltd., a subsidiary of Prilenia Therapeutics BV, is a company headquartered in Herzliya, Israel, that operates clinical stage biotech company. The firm was founded by Michael R. Hayden.\nThe company says this about itself: Prilenia is a clinical stage biotech startup founded in 2018 with the purpose of improving the lives of patients and their families by developing treatments for neurodegenerative and neurodevelopmental disorders. Prilenia is based in Naarden, the Netherlands and Herzliya, Israel.

Guangzhou Zhongda Nansha Science and Technology Innovation Industrial Park Co., Ltd., established on 2011-12-07, Business scope includes technical services,technology development,technical consulting,Technology Exchange,technology transfer,Technology Promotion;Technology intermediary service;software development;Data processing and storage support services;Information Technology Consulting Services;Medical Research and Experimental Development;Information system integration service;Engineering and Technology Research and Experimental Development;Conference and Exhibition Services;Non-residential real estate leases;Sales of Class II Medical Devices;Wholesale medical masks;Medical Mask Retail;Indoor environment detection;Health food production;Health food sales;pharmaceutical production;Pharmaceutical commissioned production;food production;Food additive production;Production of formula food for special medical purpose;Sales of formula food for special medical purposes;Production of Class II Medical Devices;Production of medical masks