Amryt Pharma
NASDAQ:AMYTAmryt 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.
Evolus
NASDAQ:EOLSEvolus, Inc., a performance beauty company, focuses on delivering products in the cash-pay aesthetic market in the United States, Canada, and Europe. The company offers Jeuveau, a proprietary 900 kilodalton purified botulinum toxin type A formulation for the temporary improvement in the appearance of moderate to severe glabellar lines in adults. It also provides dermal filler products under the Estyme and Evolysse names. The company was incorporated in 2012 and is headquartered in Newport Beach, California.
Precigen
NASDAQ:PGENPrecigen, Inc. operates as a discovery and clinical-stage biopharmaceutical company that develops gene and cell therapies using precision technology to target diseases in therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. It operates through two segments, Biopharmaceuticals and Exemplar. The company offers therapeutic platforms consisting of UltraCAR-T to provide chimeric antigen receptor T cell therapies for cancer patients; AdenoVerse immunotherapy, which utilizes a library of proprietary adenovectors for gene delivery of therapeutic effectors, immunomodulators, and vaccine antigen; and ActoBiotics for specific disease modification. It also develops programs based on the UltraCAR-T platform, including PRGN-3005 in Phase 1b clinical trial to treat advanced ovarian, fallopian tube, or primary peritoneal cancer; PRGN-3006 in Phase 1b trial for patients with relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndromes; and PRGN-3007 in Phase 1/1b trial for the treatment of advanced receptor tyrosine kinase-like orphan receptor 1-positive, hematologic, and solid tumors. In addition, the company is developing programs based on the AdenoVerse immunotherapy platform comprising PRGN-2009 in Phase 2 trial for patients with HPV-associated cancer; and PRGN-2012 in Phase ½ trial to treat recurrent respiratory papillomatosis, as well as AG019, which is based on the ActoBiotics platform and in Phase 1b/2a trial, to treat type 1 diabetes mellitus. Further, it provides UltraPorator, a proprietary electroporation device; and develops research models and services for healthcare research applications. The company was formerly known as Intrexon Corporation and changed its name to Precigen, Inc. in February 2020. Precigen, Inc. was founded in 1998 and is headquartered in Germantown, Maryland.
Rallybio
NASDAQ:RLYBRallybio Corporation, a clinical-stage biotechnology company, engages in development and commercialization of life-transforming therapies for patients suffering from severe and rare diseases. Its lead product candidate is RLYB212, a monoclonal anti-HPA-1a antibody that has completed Phase I clinical trial for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT); and RLYB211 for the prevention of FNAIT. The company is also developing RLYB114, a pegylated complement factor 5 (C5)-targeted Affibody molecule in preclinical development for the treatment of complement-mediated ophthalmic diseases; RLYB116, an inhibitor of complement component 5 (C5) to treat several diseases of complement dysregulation which has completed phase 1 trial; and RLYB331, a preclinical antibody, for the treatment of severe anemia with ineffective erythropoiesis and iron overload. It entered into a strategic alliance with AbCellera to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases. Rallybio has collaboration with Exscientia for the development of small molecule therapeutics for rare diseases; and collaboration agreement with Johnson & Johnson to provide pregnant individuals therapeutic solutions at risk of fetal and neonatal alloimmune thrombocytopenia. The company was founded in 2018 and is headquartered in New Haven, Connecticut.