Rare Diseases – Curing The IncurableWhile not entirely solved, a lot of major deadly illnesses that have plagued humanity have become more manageable over the last century, from bacterial (antibiotics) and viral (vaccines) infections to diabetes (insulin).There is, however, a wide array of rare diseases which are still mostly incurable. Most of the time, this is because these diseases are not caused by an external threat, but by a biological dysfunction inside the very cells of the patient. This is quickly changing, thanks to progress in biosciences.On one hand, with the human genome sequenced and much more advanced analytical methods, scientists can finally understand what are the root causes of rare diseases. Even AI is now helping interpret the data and provide new insights .On the other hand, new technologies like gene editing open the possibility of fixing deficient cells or organs, restoring normal function.Rare diseases each affect less than 1 in 2,000 people, with more than 6000 different rare diseases. Despite their individual rarity, together, they affect around 4% of the population, with as many as 30 million people in just Europe .3/4 of rare diseases affect children, 72% are genetic in origin, and it takes on average 5 years for rare disease patients to get a diagnosis.CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a recently discovered tool for genetic editing. It allows for very precise and directed gene editing, and its discoverers have won the 2020 Nobel Prize .What sets CRISPR Therapeutics apart is the all-star team of founders, including Dr

Beam Therapeutics is developing BEAM-101 for the SCD indication in early-stage studies.

Beam Therapeutics appoints biotech executive Christi Shaw to its board of directors.