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Aeglea BioTherapeutics, Inc., a clinical-stage biotechnology company, designs and develops human enzyme therapeutics for the treatment of rare metabolic diseases. The company's therapeutic candidates include pegtarviliase, a polyethylene glycol modified, which is in Phase I/II clinical trial for the treatment of patient with homocystinuria; and pegzilarginase, a recombinant human Arginase 1 that is in Phase III PEACE trial to evaluate the safety and efficacy for the treatment of Arginase 1 deficiency. In addition, its preclinical pipeline includes AGLE-325 for the treatment of cystinuria, as well as other research programs. The company was formerly known as Aeglea BioTherapeutics Holdings, LLC and changed its name to Aeglea BioTherapeutics, Inc. in March 2015. Aeglea BioTherapeutics, Inc. was founded in 2013 and is headquartered in Austin, Texas.

Athersys, Inc., a biotechnology company, focuses on the research and development activities in the field of regenerative medicine. Its clinical development programs are focused on treating neurological conditions, cardiovascular diseases, inflammatory and immune disorders, and pulmonary and other conditions. The company's lead platform product includes MultiStem cell therapy, an allogeneic stem cell product for the treatment of patients suffering from neurological damage from an ischemic stroke, as well as for acute respiratory distress syndrome, trauma complications, HSC transplant support, and other indications. It also develops MultiStem cell therapy to promote tissue repair and healing for animal patients. The company has license and collaboration agreements with Healios K.K. to develop and commercialize MultiStem cell therapy for ischemic stroke, acute respiratory distress syndrome, and ophthalmological indications, as well as for the treatment of liver, kidney, pancreas, and intestinal tissue diseases; and the University of Minnesota to develop MultiStem cell therapy platform. The company was founded in 1995 and is headquartered in Cleveland, Ohio.

Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.

Fortress Biotech, Inc., a biopharmaceutical company, develops and commercializes pharmaceutical and biotechnology products. The company markets dermatology products, such as Ximino an oral minocycline drug for the treatment of moderate to severe acne; Targadox an oral doxycycline drug for adjunctive therapy for severe acne; Exelderm cream for antifungal intended for topical use; Qbrexza a medicated cloth towelette for the treatment of primary axillary hyperhidrosis; Amzeeq; Zilxi; and Accutane capsules for severe recalcitrant nodular acne. It also develops late stage product candidates, such as intravenous Tramadol for the treatment of post-operative acute pain; CUTX-101, an injection for the treatment of Menkes disease; MB-107 and MB-207 for the treatment of X-linked severe combined immunodeficiency; Cosibelimab for metastatic cancers; CK-101 for the treatment of patients with EGFR mutation-positive NSCLC; CAEL-101 for the treatment of amyloid light chain amyloidosis; Triplex vaccine for cytomegalovirus; and CEVA101 for the treatment of severe traumatic brain injury in adults and children. The company's early stage product candidates include MB-102 for blastic plasmacytoid dendritic cell neoplasm; MB-101 for glioblastoma; MB-104 for multiple myeloma and light chain amyloidosis; MB-106 for B-cell non-hodgkin lymphoma; MB-103 for GBM & metastatic breast cancer to brain; MB-108; MB-105 for prostate and pancreatic cancers; and BAER-101. Its preclinical product candidates comprise AAV-ATP7A gene therapy; AVTS-001 gene therapy; CK-103 BET inhibitor; CEVA-D and CEVA-102; CK-302, an anti-GITR; CK-303, an anti-CAIX; and ONCOlogues, and oligonucleotide platform. It has collaboration arrangements with universities, research institutes, and pharmaceutical companies. The company was formerly known as Coronado Biosciences, Inc. and changed its name to Fortress Biotech, Inc. in April 2015. Fortress Biotech, Inc. was incorporated in 2006 and is based in Bay Harbor Islands, Florida.

Verrica Pharmaceuticals Inc., a clinical-stage dermatology therapeutics company, develops medications for the treatment of skin diseases in the United States. Its product pipeline comprises YCANTH (VP-102), which is in phase III clinical trial for the treatment of common warts; and has completed phase II clinical trial for the treatment of external genital warts. The company also develops VP-315, an oncolytic peptide-based injectable therapy, which is in phase II clinical trial for the treatment of dermatology oncologic conditions which includes basal cell carcinoma; and VP-103, a cantharidin-based product candidate for the treatment of plantar warts and is in phase II clinical trial. In addition, it offers YCANTH for the treatment of molluscum contagiosum. The company has a collaboration and license agreement with Torii Pharmaceutical Co., Ltd. for the development and commercialization of its product candidates for the treatment of molluscum contagiosum and common warts in Japan, including VP-102; and a license agreement with Lytix Biopharma AS to develop and commercialize VP-315 for dermatological oncology indications, such as non-metastatic melanoma and non-metastatic merkel cell carcinoma. Verrica Pharmaceuticals Inc. was incorporated in 2013 and is headquartered in West Chester, Pennsylvania.