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  3. Rhythm Pharma Axovia Ink Joint Research Collaboration For Bardet Biedl Syndrome
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  • 26 Oct 2024
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Rhythm Pharma & Axovia Ink Joint Research Collaboration for Bardet-Biedl Syndrome

Rhythm Pharma & Axovia ink joint research collaboration designed to advance the understanding of Bardet-Biedl syndrome

Overview

Rhythm Pharmaceuticals, Inc. and Axovia Therapeutics Ltd. announced a joint research collaboration designed to advance the understanding of Bardet-Biedl syndrome (BBS).

Words from the CEO: Axovia Therapeutics

  • “We believe there is a significant need and opportunity with the growing awareness of BBS to better understand the disease and its epidemiology,” said Prof. Phil Beales, Axovia Therapeutics’ chief executive officer and co-founder. 
  • “At Axovia, we are utilizing gene therapy to address the loss of sight and obesity experienced by patients with BBS, and we are pleased to be collaborating with Rhythm, a global company that has pioneered research in the melanocortin-4 receptor pathway and developed the first and only commercially available drug to help treat hyperphagia and severe obesity in patients living with BBS. Rhythm and Axovia Therapeutics are natural collaborators in such an effort.”

Words from the CEO: Rhythm

  • “Prof. Beales is a leading expert on BBS and was involved in creating the original diagnostic criteria for patients living with this disease,” said David Meeker, M.D., chairman, chief executive officer and president of Rhythm. 
  • “We are excited to work together with Axovia Therapeutics to advance our shared understanding of the disease and pool the knowledge we have gained from our respective screening efforts with the goal of further improving the lives of patients and their families living with BBS.”  

About Bardet-Biedl Syndrome

  • Bardet-Biedl syndrome (BBS) is a rare autosomal recessive ciliopathy that presents with a variety of signs and symptoms that evolve over time including visual impairment, renal disease, polydactyly, genital abnormalities, cognitive impairment, hyperphagia and early-onset, severe obesity arising from impairment of the hypothalamic MC4R pathway. 
  • In the United States, BBS affects approximately 4,000 to 5,000 individuals with similar prevalence in Europe.

About the Company: Axovia Therapeutics

  • Axovia Therapeutics is leading the development of therapies that address the genetic causes of blindness and obesity which are driven by ciliopathies. 
  • Ciliopathies are a group of more than 50 inherited genetic diseases linked to more than 950 genes that impact the function of cilia which are critical for protein transport and cellular signalling.

About the Company: Rhythm

  • Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients living with rare neuroendocrine diseases. 
  • Rhythm’s lead asset, Imcivree (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the US Food and Drug Administration (FDA) for chronic weight management in adult and paediatric 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).

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