Xin "Daniel" Gao

1/In one of the best articles that I’ve read recently - Washington Post’s excellent science reporter @Carolynyjohnson sheds light on a less known aspect of the Gene Therapy and CRISPR Gene Editing field. Although many Gene Therapies and CRISPR Gene Editing therapies are clinically proven and could generate major health benefits for patients, they are still being discarded and fail to reach patients only due to economical and regulatory hurdles. In her excellent piece Carolyn Johnson brings not only the voice of the physicians and of the patient community but also that of the scientists who are developing the genetic treatments for these rare genetic diseases including @kiranmusunuru who treated the baby KJ Muldoon with a custom made Base Editing treatment and that of Professor @davidrliu - the inventor of Base and Prime Editing and the co-founder of Beam Therapeutics (NASDAQ: $BEAM) and Prime Medicine (NASDAQ: $PRME) - all agreeing that the current situation is both unacceptable and heartbreaking! An outstanding example for this anomaly - that existing and clinically proven medical treatments for rare genetic diseases are not being offered to patients who are desperate for a cure, can be seen in many cases such as $PRME recent decision to discontinue its clinically proven PM359 program for treating CGD - a rare inherited immune deficiency disorder. Custom Gene Therapy and Gene Editing treatments are not a vague or futuristic concept but a reality! The regulators must get involved and intervene in order to immediately solve this issue which affects more than 300 million patients with rare genetic diseases and their families worldwide!! 🧵👇 $XBI