AVROBIO
NASDAQ:AVROAVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Codiak BioSciences
NASDAQ:CDAKCodiak BioSciences, Inc., a clinical-stage biopharmaceutical company, focuses on developing exosome-based therapeutics for the treatment of oncology, neuro-oncology, neurology, neuromuscular diseases, infectious diseases, and other diseases. Its lead product candidates are exoSTING and exoIL-12 exosome therapeutic candidates for the treatment of various solid tumors. The company is also developing exoASO-STAT6, an antisense oligonucleotide targeting the STAT6 transcription factor; and exoVACC, a modular platform for constructing precision vaccines. It has a collaboration agreement with the Jazz Pharmaceuticals Ireland Limited, Sarepta Therapeutics, Inc., Kayla Therapeutics S.A.S., Washington University, Ragon Institute of MGH, MIT, and Harvard. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Freeline Therapeutics
NASDAQ:FRLNFreeline Therapeutics Holdings plc, a clinical-stage biotechnology company, develops transformative adeno-associated virus (AAV) vector-mediated gene therapies. It develops FLT180a for the treatment of hemophilia B.; FLT201 for the treatment of Type 1 Gaucher disease; and FLT190 for the treatment of Fabry disease. in addition, it has research programs for systemic gene therapy. The company was founded in 2015 and is headquartered in Stevenage, the United Kingdom.
Outlook Therapeutics
NASDAQ:OTLKOutlook Therapeutics, Inc., operates as a clinical-stage biopharmaceutical company, focuses on developing and commercializing monoclonal antibodies for various ophthalmic indications. Its lead product candidate is ONS-5010, an ophthalmic formulation of bevacizumab product candidate that is in Phase-III clinical trial for the treatment of wet age-related macular degeneration and other retina diseases. Outlook Therapeutics, Inc. has collaboration and license agreements with BioLexis Pte. Ltd. and Zhejiang Huahai Pharmaceutical Co., Ltd. The company was formerly known as Oncobiologics, Inc. and changed its name to Outlook Therapeutics, Inc. in November 2018. Outlook Therapeutics, Inc. was incorporated in 2010 and is based in Iselin, New Jersey.
SQZ Biotechnologies
NYSE:SQZSQZ Biotechnologies Company, a clinical-stage biotechnology company, develops cell therapies for patients with cancer, autoimmune, infectious diseases, and other serious conditions. Its lead product candidate is SQZ-PBMC-HPV, from its SQZ Antigen Presenting Cells platform, which is in a Phase I clinical trial as a monotherapy and in combination with immuno-oncology agents for the treatment of HPV16+ advanced or metastatic solid tumors, including cervical, head-and-neck, anal, penile, vulvar, and vaginal cancer. The company also develops SQZ-AAC-HPV, which is in a Phase I clinical trial for the treatment of HPV16+ advanced or metastatic solid tumors in monotherapy and in combinations with immune-oncology agents. Its additional platforms in development are SQZ Activating Antigen Carriers; and SQZ Tolerizing Antigen Carriers. The Company was incorporated in 2013 and is headquartered in Watertown, Massachusetts.