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Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B. It is also developing ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; EB-201 for for epidermolysis bullosa (EB); ABO-301 for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.

IDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 1/2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung, bladder, gastric, and esophageal cancers; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; GSK101, a Pol Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other homologous recombination, and HRD mutations; and Werner Helicase inhibitors for tumors with high microsatellite instability. It offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Novartis International Pharmaceuticals Ltd., Amgen Inc., Gilead Sciences, Inc., Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.

Mustang Bio, Inc., a clinical-stage biopharmaceutical company, focuses on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. Its pipeline focuses on gene therapy programs for rare genetic disorders, chimeric antigen receptor (CAR) engineered T cell (CAR T) therapies for hematologic malignancies, and CAR T therapies for solid tumors. The company develops MB-117 and MB-217, a gene therapy program for X-linked severe combined immunodeficiency, a rare genetic immune system condition in which affected patients do not live beyond infancy without treatment. The company also develops MB-106 CAR T cell program for B cell non-hodgkin lymphoma and chronic lymphocytic leukemia; MB-101 CAR T cell program for glioblastoma; MB-108, a next-generation oncolytic herpes simplex virus. It has license agreements with Nationwide Children's Hospital, CSL Behring; Mayo Clinic; Leiden University Medical Centre; SIRION Biotech GmbH. The company was incorporated in 2015 and is headquartered in Worcester, Massachusetts.

Neoleukin Therapeutics, Inc., a biopharmaceutical company, develops immunotherapies for cancer, inflammation, and autoimmunity disorders using protein design technology. The company's lead product candidate is NL-201, a de novo protein designed to mimic the therapeutic activity of the cytokines interleukin (IL)-2/IL-15 for the treatment of various types of cancer, including renal cell carcinoma and melanoma. The company was formerly known as Aquinox Pharmaceuticals, Inc. and changed its name to Neoleukin Therapeutics, Inc. in August 2019. Neoleukin Therapeutics, Inc. was founded in 2003 and is headquartered in Seattle, Washington.

VBI Vaccines Inc., a commercial-stage biopharmaceutical company, develops and sells vaccines to treat immuno-oncology and infectious disease. It offers Sci-B-Vac, a prophylactic hepatitis B (HBV) vaccine. The company also develops VBI-2601, a protein based immunotherapeutic candidate for the treatment of chronic HBV infection; VBI-1901, a glioblastoma vaccine immunotherapeutic candidate, which is in Phase I/IIa clinical study to treat solid tumors; VBI-1501, a prophylactic cytomegalovirus vaccine candidate that has completed Phase I clinical trial; and VBI-2501 that is in preclinical trial to treat Zika virus. In addition, it develops coronavirus vaccine candidates that include VBI-2902, VBI-2901, and VBI-2905. The company has collaboration and license agreements with Brii Biosciences Limited; and the National Research Council of Canada to develop pan-coronavirus vaccine candidate targeting COVID-19, severe acute respiratory syndrome, and Middle East respiratory syndrome. The company was formerly known as SciVac Therapeutics Inc. and changed its name to VBI Vaccines Inc. in May 2016. VBI Vaccines Inc. is based in Cambridge, Massachusetts.