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
News and Trends 29 Sep 2025 First-ever approval for Barth Syndrome treatment: what does this mean for ultra-rare disease therapeutics? FDA approval for Stealth BioTherapeutics’ elamipretide has been a long time coming. What does this mean for other ultra-rare disease companies? September 29, 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
Webinars 1 Sep 2025Organoids in cancer research: Paving the way for faster drug development across cancer indications Discover how patient-derived organoids enable smarter, quicker oncology drug development. September 1, 2025 Share WhatsApp Twitter Linkedin Email
Podcast 22 Aug 2025How RNAi is expanding from a therapy of choice for rare disease into treating common conditions Our guest this week is Paul Nioi, Senior Vice President of Research at Alnylam and we talked about the evolution of RNAi. August 22, 2025 Share WhatsApp Twitter Linkedin Email Listen Now Apple Music Spotify More
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
News and Trends 6 Aug 2025 Fusion proteins attract funds and biopharma partnerships Fusion proteins have drawn investors these past months attracting dollars from deep pockets in the pursuit of commercial success. August 6, 2025 - 9 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 16 Jul 2025 Eight rare autoimmune diseases biotechs are fighting to treat Discover eight rare autoimmune diseases you may not have heard of and what biotechs are doing to treat them. July 16, 2025 - 12 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
In Depth 28 May 2025 Myasthenia gravis treatments: what’s on the horizon? Following FDA approval of Imaavy, we explore promising biotech candidates in development for myasthenia gravis. May 28, 2025 - 10 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
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
Best in Biotech 28 Feb 2025 Rare Disease Day: seven drugs awaiting approval in 2025 As we observe Rare Disease Day today, let us take a look at seven drugs poised to be approved to address rare diseases in 2025. February 28, 2025 - 10 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email