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Avid Bioservices, Inc., a contract development and manufacturing organization, provides process development and current good manufacturing practices (CGMP) clinical and commercial manufacturing services of biologics for the biotechnology and biopharmaceutical industries. The company offers various services, including clinical and commercial drug substance manufacturing, bulk packaging, release and stability testing, and regulatory submission and support. It also provides various process development services, such as upstream and downstream development and optimization, analytical methods development, cell line development, testing, and characterization. The company was formerly known as Peregrine Pharmaceuticals, Inc. and changed its name to Avid Bioservices, Inc. in January 2018. Avid Bioservices, Inc. was incorporated in 1981 and is headquartered in Tustin, California.

COMPASS Pathways plc operates as a mental health care company in the United Kingdom and the United States. It develops COMP360, a psilocybin therapy that is in Phase III clinical trials for the treatment of treatment-resistant depression; and is in Phase II clinical trials for the treatment of post-traumatic stress disorder and anorexia nervosa. The company was formerly known as COMPASS Rx Limited and changed its name to COMPASS Pathways plc in August 2020. COMPASS Pathways plc was incorporated in 2020 and is headquartered in London, the United Kingdom.

Design Therapeutics, Inc. a biopharmaceutical company, researches, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.

Prelude Therapeutics Incorporated, a clinical-stage biopharmaceutical company, focuses on the discovery and development of novel precision cancer medicines to underserved patients. It is developing PRT1419, a myeloid cell leukemia-1 inhibitor, which is in Phase 1 clinical trial for the treatment of selected relapsed/refractory myeloid or B-cell malignancies; PRT2527, a cyclin-dependent kinase, which is in Phase 1 clinical trial for the treatment of advanced solid tumors; PRT3645, a cyclin-dependent kinase 4/6 inhibitor, which is in Phase 1 clinical trial for the treatment of advanced and metastatic solid tumors; and PRT3879, a SMARCA2 selective protein degrader, which is in Phase 2/3 clinical trial for the treatment of advanced and metastatic solid tumors with loss of SMARCA4 due to truncating mutation and/or deletion. The company's CDK9 program is a regulator of cancer-promoting transcriptional programs, including MCL1, MYC and MYB. Prelude Therapeutics Incorporated was incorporated in 2016 and is based in Wilmington, Delaware.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.