Category Archives: Rare Diseases

By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets. Identifying

By Ivana Magovčević-Liebisch, CEO of Vigil Neuroscience, as part of the From The Trenches feature of LifeSciVC Patients and their care partners are at the center of our work in the life sciences industry – but at what point during

By Mike Cloonan, CEO of Sionna Therapeutics In 2016, following robust discovery efforts by Pfizer and others, the NBD1 domain of the CFTR protein was largely considered undruggable. They knew it was a key target – the most common genetic

By Deanna Petersen, CBO of AVROBio, as part of the From the Trenches feature of LifeSciVC Gene therapy’s tremendous potential to transform the treatment of both rare and common diseases has been understood for decades. But few may appreciate how

By Josh Brumm, CEO of Dyne Therapeutics, as part of the From The Trenches feature of LifeSciVC One morning in early June I was reading my LinkedIn feed and saw something that caught my eye – a university was establishing

Neuroscience has had a tough run of news lately. With Amgen deprioritizing neuro this year, Pfizer doing the same last year, and others like BMS, GSK, and AZ cutting back on CNS a few years earlier, it would seem dark

This blog was co-written by Doug Kerr, Head of Preclinical Research & Clinical Development, and Geoff McDonough, CEO, both of GenerationBio, as part of the From The Trenches feature of LifeSciVC. Our industry stands at the beginning of the most

Today we recognize and celebrate #RareDiseaseDay to raise awareness of rare diseases and their impact on patients’ lives. Nearly 1 out of 10 Americans lives with a rare disease – more than 30M people in the United States.  NORD has documented over

My experience as an adaptive ski coach has been nothing short of life-changing. I don’t say that with hyperbole. By bringing together my passion for the mountains with my profession as an investor in biomedical therapies, adaptive coaching completes the

The field of gene and cell therapy has witnessed an unprecedented few months, almost unimaginable only a few years ago. It’s worth pausing to reflect on some of the recent highlights: FDA approval of two chimeric antigen receptor (CAR) T-cell

This blog was written by Jose-Carlos Gutiérrez-Ramos, CEO of Synlogic, as part of the “From The Trenches” feature of LifeSciVC. When I was invited to contribute to this blog, I was determined to write first about patients with Rare Diseases and

Last week on February 28th – as I sat down to write this short blog– was Rare Disease Day 2015. Rare diseases, and there are estimated to be 7000 of them, impact almost 30 million people in the US alone, according to