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Moderna (MRNA) Inks Deal to Develop Ultra-Rare Disease Therapy

Moderna (MRNA) inks a collaboration deal with the Institute for Life Changing Medicines to develop a free of charge mRNA-based therapy to treat Crigle...

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This story originally appeared on Zacks

Moderna, Inc. MRNA announced that it has entered into a new collaboration agreement with the non-profit Institute for Life Changing Medicines (ILCM) to develop an mRNA-based therapy for an ultra-rare disease, Crigler-Najjar Syndrome type 1 (CN-1). The company will develop a new therapeutic, mRNA-3351, for the disease under this collaboration and aims to provide it for free to patients.

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Per the press release, there are approximately 70 to 100 known cases of this ultra-rare disorder across the globe. The CN-1 disease is a genetically inherited disorder that results in an inability to properly convert and clear bilirubin from the body leading to jaundice and damage to the brain, muscles, and nerves. Per an article by the United States National Library of Medicine, CN-1 affects fewer than 1 in 1 million newborns worldwide and can lead to death in childhood due to kernicterus (brain damage).

Moderna will grant license rights for mRNA-3351 to ILCM with no upfront fees as well as without any downstream payments per the terms of the agreement. The clinical development of the candidate will be undertaken by ILCM and clinical studies are expected to begin next year.

Currently, phototherapy treatments are the standard of care and patients may be treated up to 12 hours a day throughout their life, which severely affects their daily routine.

Moderna believes that its mRNA-based therapeutics candidate has the potential to treat CN-1 as well as prevent lethal metabolic crises in the patients. Mutation in the human gene, UGT1A1, is responsible for CN-1 and mRNA-3351 encodes for this gene and is designed to treat the disease by restoring the missing or dysfunctional proteins in the gene.

The candidate enjoys Rare Pediatric Disease designation in the United States

Shares of Moderna have rallied 317.7% so far this year compared with the industry’s 2.8% rise.

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The prospect of the company’s diverse mRNA-based pipeline received a boost as its mRNA-based COVID-19 vaccine was found effective in providing protection against the deadly virus and its variants. Moderna’s mRNA-1273 and Pfizer PFE/ BioNTech’s BNTX Comirnaty are mRNA-based vaccines that are leading in the COVID-19 space. The demand for these vaccines has been significantly higher than other authorized vaccines, which have been developed using different technology, including J&J JNJ and AstraZeneca’s adeno-virus based COVID-19 vaccines.

The mRNA-based therapies have the potential to change the landscape of the healthcare system with their novel treatment design by altering the basic components of the human body, which may help treat a wider portfolio of diseases including many rare diseases. Moderna’s pipeline candidates are being developed for several rare diseases.

Last month, the company initiated a phase I/II study for its pipeline candidate, mRNA-3705, in patients with methylmalonic acidemia, a rare multi-organ disease. The company is also working to advance mRNA-based therapeutics for other rare diseases such as propionic acidemia, glycogen storage disease type 1a, and phenylketonuria.

Zacks Rank

Currently, Moderna carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.



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