In Depth 14 Oct 2025 Prader Willi syndrome: five much-anticipated therapies poised for approval The treatment drug to address insatiable hunger in people with Prader-Willi syndrome was greenlit this year. Discover five therapies in line for approval. October 14, 2025 - 10 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 10 Sep 2025 When rare diseases are not so rare after all: A closer look at where and why this happens Discover how in some communities around the world, the prevalence of rare diseases is higher due to factors like cousin marriages. September 10, 2025 - 12 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
Podcast 8 Aug 2025Day One DNA: How and why the UK is betting on whole genome sequencing for every newborn Recently the UK government announced plans to sequence the DNA of every baby born in that country. Why, how, and what will it cost? August 8, 2025 Share WhatsApp Twitter Linkedin Email Listen Now Apple Music Spotify More
Best in Biotech 24 Jul 2025 Which biotech companies are gaining traction in genetic diseases? Discover twelve companies focused on genetic diseases that managed to close funding rounds in the last two years. July 24, 2025 - 12 minutesmins - By Jules Adam Share WhatsApp Twitter Linkedin Email
Podcast 11 Jul 2025How OMass Therapeutics is tackling congenital adrenal hyperplasia, a rare genetic disorder This week we talk about Congenital Adrenal Hyperplasia and how OMass Therapeutics is approaching it with the company CEO, Ros Deegan. July 11, 2025 Share WhatsApp Twitter Linkedin Email Listen Now Apple Music Spotify More
News and Trends 12 May 2025 Pharma giants pull back on AAV research: what’s next for the gene therapy space? Biopharmas have been scaling back on AAV research for sometime now. Find out why and what’s on the horizon for drug delivery methods. May 12, 2025 - 8 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
Best in Biotech 29 Apr 2025 Six biotech companies leading the charge in hemophilia treatment Discover six hemophilia companies developing new treatments for the genetic bleeding disorder, from gene therapies to RNAi therapies. April 29, 2025 - 10 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
In Depth 7 Apr 2025 Will tRNA therapy be the next big thing in genetic disease treatment? Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment. April 7, 2025 - 12 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
Best in Biotech 20 Feb 2025 3 promising biotech approaches to treat Hunter syndrome, a rare genetic disorder Discover three promising approaches being investigated to treat Hunter syndrome, a rare genetic disorder affecting children. February 20, 2025 - 10 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
In Depth 21 Nov 2024 Huntington’s disease: a therapeutic field on a bumpy ride For years, biotechs have been trying to develop medicines to cure the disease but much to no avail. Now, as clinical studies advance, there is hope. November 21, 2024 - 9 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
Best in Biotech 21 Nov 2024 Eight areas where gene therapy shines Delve into the numerous applications of gene therapy, from cancer to hematological disorders and respiratory diseases. November 21, 2024 - 12 minutesmins - By Jules Adam Share WhatsApp Twitter Linkedin Email
News and Trends 23 Oct 2024 RNA editing in humans: the race begins with first successful trial Wave Life Sciences achieves RNA editing in the clinic, crossing a major milestone in the space. October 23, 2024 - 6 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email