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Adverum Biotechnologies, Inc., a clinical-stage company, develops gene therapy product candidates to treat ocular diseases. Its lead product candidate is ixoberogene soroparvovec (ADVM-022), a single intravitreal injection gene therapy candidate used for the treatment of patients with wet age-related macular degeneration and diabetic macular edema which is in phase 2 clinical trials. The company is developing ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate for the treatment of blue cone monochromacy via a single IVT injection. Adverum Biotechnologies, Inc. has license and collaboration agreements with University of California; GenSight Biologics; Lexeo Therapeutics; and Virovek, Inc. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was incorporated in 2006 and is headquartered in Redwood City, California.

Krystal Biotech, Inc., a commercial-stage biotechnology company, discovers, develops, and commercializes genetic medicines for patients with rare diseases in the United States. It commercializes VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB). The company also develops KB105, which is in Phase 1/2 clinical trials for treating patients with deficient autosomal recessive congenital ichthyosis; KB104 for treating netherton syndrome; KB407 that is in Phase 1 clinical trials for treating cystic fibrosis; KB707 that is in Phase 1 clinical trials for the treatment of anti-PD-1 relapsed/refractory; KB408, which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study with ophthalmic B-VEC for treating for ocular complications of deb. Krystal Biotech, Inc. was founded in 2016 and is headquartered in Pittsburgh, Pennsylvania.

NanoString Technologies, Inc. develops, manufactures, and sells technology for scientific and clinical information in the fields of genomics and proteomics in the Americas, Europe, the Middle East, and the Asia Pacific. The company offers nCounter Analysis System, an automated, multi-application, digital detection, and counting system. It provides nCounter Pro and FLEX systems that include Prep Station, an automated liquid handling component that processes and prepares samples for data collection; and nCounter Digital Analyzer, which collects data from samples by taking images of the immobilized fluorescent reporters in the sample cartridge and processing the data into output files. The company also offers nCounter SPRINT Profiler, an instrument that provides liquid handling steps and the digital analysis through use of a microfluidic cartridge; and nSolver Analysis Software, a data analysis program that enables researchers to check, normalize, and analyze their data. In addition, it provides custom CodeSets; panels with oncology, immunology and infectious disease, and neuroscience applications. Further, the company offers Master Kits, cartridges, ancillary reagents, tips and reagent plates to setup and process samples in instruments; and Prosigna in vitro diagnostic kits. The company is also developing GeoMx DSP system to enable the field of spatial biology. It has collaboration with Lam Research Corporation for the development of NGS sequencing platform and related assays; and Parker Institute for Cancer Immunotherapy to develop CAR-T regimens that enhances patient outcomes for various types of cancer, as well as collaboration with Stanford Medicine, Acrobat Genomics, and Illumina Accelerator to discover new drug targets for gene editing-based therapeutics. NanoString Technologies, Inc. was incorporated in 2003 and is headquartered in Seattle, Washington. On February 4, 2024, NanoString Technologies, Inc., along with its affiliates, filed a voluntary petition for reorganization under Chapter 11 in the U.S. Bankruptcy Court for the District of Delaware.

Orchard Therapeutics plc, a gene therapy company, research, develops, and commercialize hematopoietic stem cell and gene therapies in the United Kingdom, Italy, France, and Germany. It offers OTL-200, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of patients with metachromatic leukodystrophy under the Libmeldy brand; and Strimvelis, a gammaretroviral vector-based gene therapy for the treatment of adenosine deaminase deficiency. The company's program for neurodegenerative disorders comprises clinical proof of concept-stage program, which includes OTL-203 for the treatment of mucopolysaccharidosis type I and OTL-201 for mucopolysaccharidosis type IIIA, or MPS-IIIA; and pre-clinical program, OTL-204 for the treatment of frontotemporal dementia with progranulin mutations. In addition, it develops program for immunological disorders consist of pre-clinical programs, which includes OTL-104 for Crohn's disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2; and OTL-105 for the treatment of hereditary angioedema. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom. As of January 24, 2024, Orchard Therapeutics plc operates as a subsidiary of Kyowa Kirin Co., Ltd..

Twist Bioscience Corporation engages in the manufacture and sale of synthetic DNA-based products. The company offers synthetic genes and gene fragments used in product development for therapeutics, diagnostics, chemicals/materials, food/agriculture, data storage, and various applications within academic research by biotech, pharma, industrial chemical, and agricultural companies, as well as academic labs; Oligo pools used in targeted NGS, CRISPR gene editing, mutagenesis experiments, DNA origami, DNA computing, data storage in DNA, and other applications; and immunoglobulin G proteins for customers focused on the pursuit of drug discovery and development. It also provides NGS tools comprising library preparation kits, human exome kits, and fixed and custom panels, as well as alliance panels, primarily used within diagnostic tests for various indications, population genetics research and biomarker discovery, translational research, microbiology, and applied markets research; and methylation detection kits for cancer, and rare and inherited disease study, as well as fast hybridization and full RNA sequencing workflow solution. In addition, the company provides synthetic SARS-CoV-2 RNA reference sequences as positive controls for the development of both NGS and reverse transcription-polymerase chain reaction assays; synthetic monkeypox controls; and various respiratory viral controls, including for influenzas, respiratory syncytial virus, rhinoviruses, SARS, MERS, and coronaviruses. Further, it offers SARS-CoV-2 Research Panels, such as the Twist Respiratory Virus Panel and the Pan-Viral Research Panel, for the detection of disease in a research setting; precision DNA libraries used for antibody engineering, affinity maturation, and humanization; and antibody optimization solution to enable simultaneous optimization of multiple characteristics of a given antibody. The company was incorporated in 2013 and is headquartered in South San Francisco, California.