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Amryt Pharma plc, a commercial-stage biopharmaceutical company, focuses on acquiring, developing, and commercializing various treatments to help improve the lives of patients with rare and orphan diseases. The company provides metreleptin, an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy; oral octreotide, a long-term maintenance therapy in acromegaly patients; and lomitapide, an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the Homozygous familial Hypercholesteraemia. Its development candidates include Oleogel-S10 for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (EB), a rare and distressing genetic skin disorder affecting young children and adults; and AP103, a pre-clinical gene therapy for patients with dystrophic EB. The company sells its products in the Americas, Europe, and the Middle East. Amryt Pharma plc was founded in 2015 and is headquartered in Dublin, Ireland.

BioSpecifics Technologies Corp., a biopharmaceutical company, develops an injectable collagenase clostridium histolyticum for various indications in the United States and internationally. The company offers injectable collagenase for the treatment of Dupuytren's contracture and Peyronie's disease under the XIAFLEX and Xiapex brands. It also provides injectable collagenase to treat frozen shoulder, cellulite, canine lipoma, lateral hip fat, plantar fibromatosis, human lipoma, and uterine fibroids. The company has a development and license agreement with Endo Global Ventures. BioSpecifics Technologies Corp. was founded in 1957 and is headquartered in Wilmington, Delaware.

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.

Marinus Pharmaceuticals, Inc., a pharmaceutical company, focuses on development and commercialization of therapeutic products for patients suffering from rare genetic epilepsies and other seizure disorders. It offers ZTALMY (ganaxolone), an oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder for adult and pediatric patient populations in acute and chronic care, and in-patient and self-administered settings. The company's ZTALMY product candidate acts at synaptic and extrasynaptic GABAA receptors, a target for its anti-seizure, antidepressant, and anxiolytic potential. It is developing ganaxolone for treating genetic epilepsy disorders, such as PCDH19-related epilepsy, and tuberous sclerosis complex. Marinus Pharmaceuticals, Inc. has license agreement with Purdue Neuroscience Company and CyDex Pharmaceuticals, Inc.; and collaboration agreement with Orion Corporation and Tenacia Biotechnology (Shanghai) Co., Ltd. Marinus Pharmaceuticals, Inc. was incorporated in 2003 and is headquartered in Radnor, Pennsylvania.

Pandion Therapeutics, Inc., a clinical stage biopharmaceutical company, develops various therapeutics to address the unmet needs of patients suffering from autoimmune diseases. Its lead product candidate is PT101, an effector module comprising an engineered variant of wild-type interleukin-2 (IL-2) fused to a protein backbone that is in Phase 1a clinical trials for the treatment of various autoimmune and inflammatory diseases. The company's other product candidates comprise PT627, a systemic PD-1 agonist that is in preclinical studies for the treatment of autoimmune diseases; PT001, a bifunctional molecule combining its PD-1 agonist effector with a tether module that binds to mucosal vascular addressing cell adhesion molecule (MAdCAM) to drive tissue-selective immunomodulation in the gastrointestinal tract; and PT002, a tethered bifunctional molecule that utilizes its IL-2 mutein effector module coupled with a MAdCAM-tether module to deliver the IL-2 mutein effector to the gastrointestinal tract where MAdCAM is expressed. It has a license and collaboration agreement with Astellas Pharma Inc. to discover and develop novel compounds for autoimmune diseases of the pancreas, including type 1 diabetes. The company was formerly known as Immunotolerance, Inc. and changed its name to Pandion Therapeutics, Inc. in June 2017. Pandion Therapeutics, Inc. was incorporated in 2016 and is headquartered in Watertown, Massachusetts.