Engaging with people and their stories of life with a Rare Disease
I have worked on multiple Rare Disease projects throughout my career in the Biopharmaceutical and Consulting industry but I am still...
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Flipping the traditional drug development model on its head; Custom-designed CRISPR therapy is close to becoming a reality
Rich Horgan, the founder of the nonprofit Cure Rare Disease, is on a quest to develop a therapy for his brother Terry who was born with...
On the Transformative Potential of Advanced Therapeutics in Rare Disease
The burgeoning field of cell and gene therapies collectively referred to as Advanced Therapeutics (ADVTX), is at last beginning to...
5 key challenges for Biotech surrounding smaller patient populations and targeted therapies in Biotech - Part 2 of 5
Last week we introduced 5 key challenges that, based on interviews at various biotech congresses, appear to be at the forefront of...
Another major advancement in gene therapy shows promise for patients with Duchenne Muscular Dystrophy
Investment in gene therapy has continued to pay off as Sarepta Therapeutics recently announced positive results from phase 1/2a trial...
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