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Avadel Pharmaceuticals plc operates as a biopharmaceutical company in the United States. Its lead product candidate is LUMRYZ, a formulation of sodium oxybate, which is in a Phase 3 clinical trial for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy. The company was formerly known as Flamel Technologies SA and changed its name to Avadel Pharmaceuticals plc in January 2017. Avadel Pharmaceuticals plc was incorporated in 2015 and is headquartered in Dublin, Ireland.

Rallybio 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.

Alaunos Therapeutics, Inc., a clinical-stage oncology-focused cell therapy company, develops adoptive TCR engineered T-cell therapies. It develops TCR Library, which is in Phase I/II clinical trial for 12 TCRs reactive to mutated KRAS, TP53, and EGFR from its TCR library for the treatment of non-small cell lung, colorectal, endometrial, pancreatic, ovarian, and bile duct cancers; hunTR, a human neoantigen T-cell receptor engine. The company has a license agreement with PGEN Therapeutics, Inc.; research and development agreement with The University of Texas MD Anderson Cancer Center; and a patent license agreement, and research and development agreement with the National Cancer Institute. The company was formerly known as ZIOPHARM Oncology, Inc. and changed its name to Alaunos Therapeutics, Inc. in January 2022. Alaunos Therapeutics, Inc. was founded in 2003 and is headquartered in Houston, Texas.

Entrada Therapeutics, Inc., a clinical-stage biotechnology company, develops endosomal escape vehicle (EEV) therapeutics for the treatment of multiple neuromuscular diseases. Its EEV platform develops a portfolio of oligonucleotide, antibody, and enzyme-based programs. Its therapeutic candidates, which include ENTR-601-44, which is in Phase I clinical trial for the treatment of Duchenne muscular dystrophy; and ENTR-701, which is in Phase 1/2 clinical trial for the treatment of myotonic dystrophy type 1. The company also offers ENTR-601-45 and ENTR-601-50, which are in preclinical trail for the treatment of Duchenne muscular dystrophy; and ENTR-501, an intracellular thymidine phosphorylase enzyme replacement therapy, which is in preclinical trail for the treatment of mitochondrial neurogastrointestinal encephalomyopathy. Entrada Therapeutics, Inc. has a strategic collaboration and license agreement with Vertex Pharmaceuticals Incorporated to research, develop, manufacture, and commercialize ENTR-701. The company was formerly known as CycloPorters, Inc. and changed its name to Entrada Therapeutics, Inc. in October 2017. Entrada Therapeutics, Inc. was incorporated in 2016 and is headquartered in Boston, Massachusetts.