Autolus Therapeutics
NASDAQ:AUTLAutolus Therapeutics plc, a clinical-stage biopharmaceutical company, develops T cell therapies for the treatment of cancer and autoimmune diseases. The company's clinical-stage programs include obecabtagene autoleucel (AUTO1), a CD19-targeting programmed T cell investigational therapy that is in Phase 1b/2 clinical trial for the treatment of adult ALL; AUTO1/22, which is in a Phase 1 clinical trial in pediatric patients with relapsed or refractory ALL; AUTO4, a programmed T cell investigational therapy for the treatment of peripheral T-cell lymphoma targeting TRBC1 and TRBC2; AUTO6NG, a programmed T cell investigational therapy targeting GD2 in development for the treatment of neuroblastoma; and AUTO8, a product candidate to treat multiple myeloma. It also focuses on developing AUTO5, a preclinical TRBC2 programmed T cell product candidate for the treatment of peripheral T-cell lymphoma. The company was incorporated in 2014 and is headquartered in London, the United Kingdom.
Precision BioSciences
NASDAQ:DTILPrecision BioSciences, Inc., an advanced gene editing company, develops in vivo gene editing therapies for gene edits, including gene elimination, insertion, and excision in the United States. The company offers ARCUS, a genome editing platform to DNA genome insertion, deletion, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; PBGENE-PMM for the treatment of m.3243 associated primary mitochondrial myopathy (PMM) which is expected to submit an IND and/or CTA. In addition, it develops PBGENE-NVS for sickle cell disease/beta thalassemia for insertion; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LL2 (insertion), a liver directed target; PBGENE-LL3, a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. The company has license and collaboration agreement with Caribou Biosciences, Inc.; license agreement with TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; development and license agreement with Eli Lilly and Company for the research and development of potential in vivo therapies for genetic disorders; Cellectis S.A.; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; Duke University; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.
Prevail Therapeutics
NASDAQ:PRVLPrevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company's lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson's disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.
Telesis Bio
NASDAQ:TBIOTelesis Bio, Inc., a synthetic biology company, manufactures and sells synthetic biology instruments, reagents, and associated products and related services, primarily to pharmaceutical and academic laboratories worldwide. The company offers BioXp 3250 system and BioXp 9600 system, that empower researchers to go from a digital DNA sequence to endpoint-ready synthetic deoxyribonucleic acid (DNA) and messenger ribonucleic acid (mRNA) with onboard next generation sequencing (NGS) library preparation; BioXp portal, an online portal that offers an intuitive guided workflow and design tools for building new DNA sequences and assembling them into vectors of choice, as well as mRNA constructs; and BioXp De Novo kits which contains the necessary building blocks and reagents, including proprietary gibson assembly branded reagents, for specific synthetic biology workflow applications. It also provides BioXp Select kits which offers to use non-telesis bio DNA while using the BioXp system to perform synthetic biology workflow applications such as cloning, mRNA generation from plasmid and cell free amplification; BioXp Next Generation Sequencing kits which contains the necessary reagents to go from DNA or RNA to a sequencer-ready library; Benchtop reagents that contains the reagents necessary to proceed specific synthetic biology workflow on the benchtop using products generated on the BioXp system; and custom gibson short oligo ligation assembly enzymatic DNA synthesis solutions, designed reduce timelines for constructing synthetic DNA, RNA, and proteins for development of mRNA-based vaccines, diagnostics, therapeutics, and personalized medicines. The company serves its products to government institutions, contract research organizations, and synthetic biology companies. The company was formerly known as Codex DNA, Inc. and changed its name to Telesis Bio Inc. in November 2022. Telesis Bio, Inc. was incorporated in 2011 and is headquartered in San Diego, California.
Voyager Therapeutics
NASDAQ:VYGRVoyager Therapeutics, Inc., a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial. In addition, the company develops VY-FXN01, which is in preclinical trial to treat friedreich's ataxia; and GBA1 gene replacement to treat parkinson's disease and is in preclinical trial. Further, it provides research program for the treatment of Huntington's disease. The company has collaboration and license agreements with Alexion; AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc; and Sangamo Therapeutics, Inc. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Lexington, Massachusetts.