United Therapeutics Corporation, a biotechnology company, engages in the development and commercialization of products to address the unmet medical needs of patients with chronic and life-threatening diseases in the United States and internationally. The company offers Tyvaso DPI, an inhaled dry powder via pre-filled and single-use cartridges; Tyvaso, an inhaled solution via ultrasonic nebulizer; Remodulin (treprostinil) injection to treat patients with pulmonary arterial hypertension (PAH) to diminish symptoms associated with exercise; Orenitram, a tablet dosage form of treprostinil, to delay disease progression and improve exercise capacity in PAH patients; and Adcirca, an oral PDE-5 inhibitor to enhance the exercise ability in PAH patients. It also markets and sells Unituxin (dinutuximab) injection, a monoclonal antibody for treating high-risk neuroblastoma; and Remunity Pump, which contains a pump and separate controller for Remodulin. In addition, the company engages in developing RemoPro and Ralinepag for the treatment of PAH; Aurora-GT, a gene therapy product to rebuild the blood vessels in the lungs; and Nebulized Tyvaso, for the treatment of idiopathic pulmonary fibrosis, as well as xenografts, which are development-stage organ products. It has licensing and collaboration agreements with DEKA Research & Development Corp. to develop a semi-disposable system for the subcutaneous delivery of treprostinil; MannKind Corporation to develop and license treprostinil inhalation powder and the Dreamboat device; and Arena Pharmaceuticals, Inc. to develop Ralinepag. The company was incorporated in 1996 and is headquartered in Silver Spring, Maryland.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd, Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. It is developing ulixacaltamide, a small molecule inhibitor of T-type calcium channels that is in Phase III clinical trial for the treatment of essential tremor; PRAX-562 for the treatment of pediatric patients with developmental and epileptic encephalopathies (DEE); and PRAX-628 to treat focal epilepsy. The company also develops PRAX-222 for the treatment of pediatric patients with early-onset SCN2A-DEE; PRAX-020 to treat KCNT1 related epilepsies; PRAX-080 for the treatment of PCDH19; and PRAX-090 and PRAX-100 for SYNGAP1 and SCN2A-LoF. It has a license agreement with RogCon Inc.; a research collaboration and license agreement with Ionis Pharmaceuticals, Inc.; a strategic collaboration and license agreement with UCB Biopharma SRL; and collaboration with The Florey Institute to develop three novel ASOs. The company was incorporated in 2015 and is based in Boston, Massachusetts.

Pliant Therapeutics, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes novel therapies for the treatment of fibrosis and related diseases in the United States. The company's lead candidate is bexotegrast, an oral, small-molecule, dual selective inhibitor of avß6 and avß1 integrins, which is in phase 2b trials for idiopathic pulmonary fibrosis and in phase 2a trial for primary sclerosing cholangitis. It also develops PLN-1474, an oral, small-molecule selective inhibitor of avß1 for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis; PLN-101095, a dual inhibitor of integrins avß8 and avß1 for the treatment of solid tumors; and PLN-101325 for treatment of muscular dystrophies. Pliant Therapeutics, Inc. was incorporated in 2015 and is based in South San Francisco, California.

Pulmatrix, Inc., a clinical stage biotechnology company, focused on development of novel inhaled therapeutic products to prevent and treat respiratory and other diseases with unmet medical needs in the United States. The company offers iSPERSE, an engineered dry powder delivery platform, which enables delivery of small or large molecule drugs to the lungs by inhalation for local or systemic applications. It engages in developing PUR1800, a narrow spectrum kinase inhibitor completed Phase 1b clinical trials for the treatment of acute exacerbations in chronic obstructive pulmonary disease; PUR1900 for the treatment of allergic bronchopulmonary aspergillosis in patients with asthma and cystic fibrosis; and PUR3100, an iSPERSE formulation of dihydroergotamine which is in Phase 1 for the treatment of acute migraine. It has a license agreement with RespiVert Ltd. for access to a portfolio of kinase inhibitor drug candidates. The company was founded in 2003 and is headquartered in Bedford, Massachusetts.

Pieris Pharmaceuticals, Inc., a biotechnology company, discovers and develops biotechnological applications. The company focuses on the development of its 4-1BB bispecifics immuno-oncology (IO) programs. Its clinical pipeline consists of IO bispecifics, including S095012 (PRS-344), a bispecific Mabcalin compound targeting PD-L1 and 4-1BB in partnership with Les Laboratoires Servier and Institut de Recherches Internationales Servier; SGN-BB228 (PRS-346), a CD228 x 4-1BB bispecific antibody-Anticalin compound targeting CD228 and 4-1BB in partnership with Pfizer Inc.; and BOS-342 (PRS-342), a GPC3 x 4-1BB bispecific Mabcalin compound targeting GPC3 and 4-1BB in partnership with Boston Pharmaceuticals, which are in phase 1 studies. The company was founded in 2001 and is headquartered in Boston, Massachusetts.

Allarity Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing oncology therapeutics using drug-specific companion diagnostics generated by its drug response predictor technology. Its drug candidates include Stenoparib, a poly-ADP-ribose polymerase inhibitor that is in Phase 2 clinical trials for ovarian cancer; Dovitinib, a pan- tyrosine kinase inhibitor for the treatment of renal cell carcinoma; IXEMPRA (ixabepilone), a selective microtubule inhibitor in phase 2 for the treatment of metastatic breast cancer; LiPlaCis, a liposomal formulation of cisplatin, which is in Phase 2 clinical trials for metastatic breast cancer; and 2X-111, a liposomal formulation of doxorubicin that is in Phase 2 clinical trials for metastatic breast cancer and glioblastoma multiforme. Allarity Therapeutics, Inc. has a collaboration with Detsamma Investments Pty. Ltd. to develop Deflexifol for the treatment of solid tumors. The company was incorporated in 2004 and is headquartered in Boston, Massachusetts.

Palatin Technologies, Inc., a biopharmaceutical company, develops targeted receptor-specific therapeutics for the treatment of various diseases in the United States. The company's lead product is Vyleesi, a melanocortin receptor (MCr) agonist for the treatment of premenopausal women with hypoactive sexual desire disorder. It is also developing oral PL8177, a selective MC1r agonist peptide that is in Phase 2 clinical trial for the treatment of inflammatory bowel diseases. In addition, the company engages in the development of PL9643, a peptide melanocortin agonist active at multiple MCrs, including MC1r and MC5r for anti-inflammatory ocular indications, such as dry eye disease, which is in Phase 3 clinical trial; and melanocortin peptides for diabetic retinopathy. Further, it is developing PL8177, an oral peptide formulation for treatment of ulcerative colitis, which entered Phase 2 clinical trials. Palatin Technologies, Inc. was founded in 1986 and is based in Cranbury, New Jersey.