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ACADIA Pharmaceuticals NASDAQ:ACAD

ACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization innovative medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases in the United States. The company offers NUPLAZID (pimavanserin) for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis; and DAYBUE, a novel synthetic analog of the amino-terminal tripeptide of insulin-like growth factor 1 for treatment of Rett Syndrome. It also develops Pimavanserin that is in Phase III ADVANCE-2 study to treat the negative symptoms of schizophrenia; ACP-101 whixh is in Phase III for the treatment of hyperphagia in Prader-Willi syndrome; ACP-204 which is in Phase II for the treatment of Alzheimer's disease psychosis; ACP-2591 that is in Phase I for Rett syndrome and Fragile X syndrome; preclinical antisense oligonucleotide programs; and other programs for neuropsychiatric symptoms. It has a license agreement with Neuren Pharmaceuticals Limited to develop and commercialize trofinetide for Rett syndrome and other indications; and a license and collaboration agreement with Stoke Therapeutics, Inc. to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases of the CNS. The company was formerly known as Receptor Technologies, Inc. and changed its name ACADIA Pharmaceuticals Inc. in 1997. ACADIA Pharmaceuticals Inc. was incorporated in 1993 and is headquartered in San Diego, California.

Celcuity NASDAQ:CELC

Celcuity Inc., a clinical stage biotechnology company, focuses on the development of targeted therapies for the treatment of various solid tumors in the United States. The company's CELsignia diagnostic platform uses a patient's living tumor cells to identify the specific abnormal cellular process driving a patient's cancer and the related targeted therapy for the treatment. Its drug candidate includes Gedatolisib, which selectively targets various class I isoforms of PI3K and mammalian target of rapamycin and focus on the treatment of patients with hormone receptor positive, HER2-negative, advanced or metastatic breast cancer, and metastatic castration resistant prostate cancer. It had a license agreement with Pfizer, Inc. for the development and commercialization rights to Gedatolisib. The company was founded in 2011 and is headquartered in Minneapolis, Minnesota.

Parabilis Medicines NASDAQ:PBLS

Vor Biopharma NASDAQ:VOR

Vor Biopharma, Inc., a clinical-stage company, develops engineered hematopoietic stem cell (eHSC) therapies for cancer patients. It is developing VOR33, an eHSC product candidate that is in phase 1/2 to treat acute myeloid leukemia (AML) and other hematological malignancies. The company's VOR33 eHSCs lacks CD33, a protein that is expressed by AML blood cancer cells. The company's eHSCs targeted therapies, such as CAR-Ts, bispecific antibodies, and antibody-drug conjugates provide treatment for blood cancers. Vor Biopharma, Inc. has a collaboration agreement with Akron BioProducts to develop and manufacture cGMP nucleases. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.