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Aerie Pharmaceuticals, Inc., a pharmaceutical company, focuses on the discovery, development, and commercialization of ophthalmic therapies for open-angle glaucoma, dry eye, diabetic macular edema, and wet age-related macular degeneration in the United States. Its products include Rhopressa, a once-daily eye drop to reduce elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension; and Rocklatan, a once-daily fixed-dose combination of Rhopressa and latanopros to reduce IOP to treat patients with open-angle glaucoma or ocular hypertension. The company also develops AR-15512 to treat signs and symptoms of dry eye; and AR-1105 and AR-14034 SR sustained-release implants focused on retinal diseases. It has a collaborative research, development, and licensing agreement with DSM Biomedical. Aerie Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Durham, North Carolina.

Aptose Biosciences Inc., a clinical-stage biotechnology company, discovers and develops personalized therapies addressing unmet medical needs in oncology in Canada. Its lead clinical program is APTO-253, which is a Phase I clinical trial for the treatment of patients with relapsed or refractory hematologic malignancies. The company has an agreement with CrystalGenomics, Inc. to research, develop, and commercialize CG026806, a non-covalent small molecule therapeutic agent, which is in preclinical stage for the treatment of acute myeloid leukemia and chronic lymphocytic leukemia/mantle cell lymphoma. The company was formerly known as Lorus Therapeutics Inc. and changed its name to Aptose Biosciences Inc. in August 2014. Aptose Biosciences Inc. was founded in 1986 and is headquartered in Mississauga, Canada.

AVROBIO, 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.

Passage Bio, Inc., a genetic medicines company, develops gene therapies for central nervous system diseases. It develops PBGM01, a functional GLB1 gene encoding ß-galactosidase for infantile GM1; PBFT02, a functional granulin (GRN) and gene encoding progranulin (PGRN) for the treatment of FTD caused by progranulin deficiency; and PBKR03, a functional GALC gene encoding the hydrolytic enzyme galactosylceramidase for infantile Krabbe disease. The company develops PBML04 for the treatment of metachromatic leukodystrophy; PBAL05 for the treatment of amyotrophic lateral sclerosis; and other program for huntington's disease. It has a strategic research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program; and collaboration agreement, and a development services and clinical supply agreement with Catalent Maryland, Inc. Passage Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.

Poseida Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing therapeutics for patients with high unmet medical needs. The company's development candidates for Heme Malignancies includes P-BCMA-ALLO1, which is in Phase I trial to treat patients with relapsed/refractory multiple myeloma; P-CD19CD20-ALLO1, which is in Phase I trial for treating B cell malignancies and other autoimmune diseases; P-BCMACD19-ALLO1, an allogeneic, off-the-shelf CAR-T product candidate in preclinical development for treating multiple myeloma; and P-CD70-ALLO1 under preclinical development to treat hematological indications. It is also involved in the development of P-MUC1C-ALLO1 that is in Phase I trial for treating a range of solid tumors, including breast, colorectal, lung, ovarian, pancreatic, and renal cancers; P-PSMA-ALLO1, an autologous chimeric antigen receptor T cell (CAR-T) product candidate that is in preclinical development for the treatment of patients with metastatic castrate resistant prostate cancer (mCRPC); and P-PSMA-101, an allogeneic CAR-T product candidate under Phase 1 clinical trial for treating mCRPC. In addition, the company engages in the development of P-FVIII-101, a clinical stage liver-directed gene therapy for the in vivo treatment of hemophilia A; P-OTC-101, a clinical stage liver-directed gene therapy for the in vivo treatment of ornithine transcarbamylase deficiency; and P-PAH-101, a clinical stage liver-directed gene therapy for the in vivo treatment of phenylketonuria. It has a research collaboration and license agreement with F. Hoffmann-La Roche Ltd, and Hoffmann-La Roche Inc. The company was incorporated in 2014 and is headquartered in San Diego, California.