The N-Lorem Foundation has partnered with Servier to develop novel antisense oligonucleotide (ASO) therapeutic candidates for people with extremely rare neurodevelopmental disorders. The collaboration aims to expand the number of patients with nano-rare neurological disorders that N-Lorem can treat. Under the agreement, N-Lorem will use its ASO technology platform to create…
News
The U.S. Food and Drug Administration (FDA) has issued draft guidance meant to help developers of human gene therapies involving genome editing bring treatments to patients more efficiently, including those with rare and life-threatening diseases. Some recommendations also may apply to other cell and gene therapy products,…
The Medicines and Healthcare products Regulatory Agency (MHRA) is seeking public feedback on a proposal to revamp how rare disease treatments are developed in the U.K. The regulatory body wants input on a new plan that would streamline the clinical testing and approval processes to help get therapies to patients…
EveryLife Foundation for Rare Diseases’ 15th Scientific Workshop recently brought together leaders from the rare disease patient, scientific, government, and biopharmaceutical communities to discuss practical, patient-centered solutions for developing therapies for rare diseases amid evolving FDA pathways and programs. Titled, “Making What Matters Count: Advancing…
SK pharmteco is partnering with Axle Informatics and the National Institutes of Health (NIH) to advance gene therapy programs for rare diseases. The collaboration, which was funded in part by the NIH, focuses on developing viral vectors that use modified viruses to deliver genetic material…
Through a series of long-term experiments using rats, a team of U.S. scientists has tracked how an AAV2-based gene therapy vector similar to the one used in Kebilidi (eladocagene exuparvovec-tneq), an approved gene therapy for AADC deficiency, travels through the brain. Results showed that capsid signals from the…
The National Organization for Rare Disorders (NORD) is teaming up with artificial intelligence (AI) company Openevidence to combine artificial intelligence (AI) with expert review to create new educational resources on thousands of rare diseases. “People living with rare diseases and the families who support them deserve access to…
Natera has launched Zenith genomics, its next-generation genetic testing platform designed to improve the detection of rare diseases, such as aromatic l-amino acid decarboxylase deficiency. The platform uses whole-genome sequencing — a laboratory process that reads a person’s entire genome, or complete DNA sequence — and interpretation technology…
Genetics sequencing company Illumina is teaming up with the Center for Data-Driven Discovery in Biomedicine (D3b) on a new project that aims to leverage genomic data from thousands of children to better understand the biological underpinnings of childhood rare diseases and cancers. With this initiative, D3b — a…
First Atom Therapeutics — newly rebranded from Asha Therapeutics — will use a drug design approach powered by artificial intelligence (AI) to develop entirely new therapeutic candidates for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency. The platform will also be used to create novel drugs…
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