The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US
This surpasses the number of approvals supported in 2020
Four-year research collaboration combines Pfizer’s deep experience in global drug development, including programs utilizing messenger RNA (mRNA), lipid nanoparticles (LNP), and gene therapy, with Beam’s leadership in base editing and mRNA/LNP delivery technologies
Despite the challenges of pandemic disruptions, drug developers advance promising therapies for conditions, including Alzheimer's, diabetes and asthma
Investigational human monoclonal antibody in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy, a systemic, progressive and fatal condition
Both companies will combine their expertise to channel real-world evidence toward ground-breaking new treatments in rare diseases
TIB Molbiol excels in ultra-rapid assay development for emerging infectious diseases, strongly demonstrated during the Covid-19 pandemic
Dicerna develops RNAi-based therapies to selectively silence genes that cause or contribute to disease
Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth
The ground-breaking project, led by Genomics England and NHS England, was established in 2013 to sequence 100,000 whole genomes from NHS patients and their families
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