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Rhythm Pharmaceuticals and Axovia Therapeutics Announce Joint Research Collaboration in Bardet-Biedl Syndrome

Rhythm Pharmaceuticals and Axovia Therapeutics Announce Joint Research Collaboration in Bardet-Biedl Syndrome

10/24/24, 10:59 AM
Location
https://purecatamphetamine.github.io/country-flag-icons/3x2/US.svgboston
Industry
therapeutics
biotechnology
health care
Type
partnership
Rhythm Pharmaceuticals, Inc. and Axovia Therapeutics Ltd. have announced a joint research collaboration in Bardet-Biedl syndrome (BBS) to advance the understanding of the disease and pool the knowledge gained from their respective screening efforts with the goal of improving the lives of patients and their families living with BBS.

Company Info

Company
RHYTHM PHARMACEUTICALS, INC.
Location
500 boylston street
boston, massachusetts, united states
Additional Info
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s lead asset, IMCIVREE (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity caused by these diseases, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). Both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare MC4R pathway diseases, as well as a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA.

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