Interview 3 Mar 2023 Interview: how has the first gene therapy for hemophilia B transformed research? Hemophilia B, a rare disease that affects more than 230,000 people worldwide is caused by the lack of or defective clotting protein factor IX often due to a spontaneous mutation in the F9 gene, leading to excessive bleeding. Until recently, routine prophylactic infusions of factor IX replacement therapy to maintain enough clotting factor to prevent […] March 3, 2023 - 8 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
News and Trends 28 Feb 2023 What’s happening on Rare Disease Day? Rare Disease Day 2023 is on February 28. The day is dedicated to raising awareness for the approximately 300 million people around the world living with a rare disease. More than 6,000 rare diseases are characterized by a diversity of disorders and symptoms that vary not only from disease to disease, but also from patient […] February 28, 2023 - 8 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 28 Feb 2023 European Commission approves first gene therapy for hemophilia B The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […] February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
News and Trends 28 Feb 2023 GC Biopharma acquiring hemophilia rare disease pipeline GC Biopharma Corp., a South Korean provider of biopharmaceutical products, has signed an asset purchase agreement with Catalyst Biosciences to acquire three programs related to the orphan hematology disorders. The agreement includes Marzeptacog alfa (MarzAA), an engineered factor VIIa, which is ready for phase 3 clinical stage development. In its previous clinical development trials, MarzAA […] February 28, 2023 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
Opinion 28 Feb 2023 Life changing treatments for rare disease – a beacon of hope By Sander Slootweg, managing partner, Forbion More than 300 million people around the globe are affected by a rare disease. This number does not even take into account the impact on these individuals’ support units that includes family, friends and caregivers. A rare disease is defined as a disease that affects no more than 1 […] February 28, 2023 - 6 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
Best in Biotech 28 Feb 2023 5 advancements in rare disease research over the past year There are more than 7,000 rare diseases that affect hundreds of millions of people worldwide. As Rare Disease Day is observed on February 28, here are five recent advancements that have transformed research and boosted the scope for the development of novel therapies. FDA approves tissue-agnostic therapies for rare cancers Tissue-agnostic drug development has been […] February 28, 2023 - 6 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
Best in Biotech 28 Feb 2023 10 biotech companies making a difference in rare diseases February 28 is Rare Disease Day, which looks to raise awareness of rare diseases and bring about change for patients and families living with them. The event also brings attention to the need for improved access to diagnosis, as well as the need to develop more treatments to tackle a broader range of diseases. There […] February 28, 2023 - 11 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
News and Trends 24 Feb 2023Beyond Biotech podcast 35: Rare Disease Day As February 28 is Rare Disease Day, we have three guests on the podcast today all relevant to rare diseases. And they are Dan Mandell, CEO and co-founder of GRO Biosciences; Stefano Portolano, chief executive officer, Azafaros; and from Replay, Adrian Woolfson, executive chairman, president, and co-founder, and Lachlan MacKinnon, CEO and co-founder. Rare Disease […] February 24, 2023 Share WhatsApp Twitter Linkedin Email
News and Trends 23 Feb 2023 Moderna and Life Edit Therapeutics look to speed up gene editing therapies Moderna, Inc., a biotechnology company working on messenger RNA (mRNA) therapeutics and vaccines, and Life Edit Therapeutics Inc., an ElevateBio company focused on gene editing technologies and therapeutics, have announced a strategic research and development collaboration to discover and develop in vivo mRNA gene editing therapies. The partnership will combine Life Edit’s suite of proprietary […] February 23, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 21 Feb 2023 First patient enrolled in Pharming APSD trial Pharming Group N.V. says the first patient has been enrolled in its phase III clinical trial evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children with activated phosphoinositide 3-kinase delta syndrome (APDS). There is currently no approved treatment for this complex and progressive disease caused by genetic variants. At […] February 21, 2023 - 6 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 21 Feb 2023 FDA approves alpha-mannosidosis drug Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), says the U.S. Food and Drug Administration (FDA) has approved Lamzede (velmanase alfa-tycv) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare, progressive lysosomal storage disorder […] February 21, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 17 Feb 2023 Positive results for INOVIO recurrent respiratory papillomatosis trial INOVIO Pharmaceuticals has announced positive preliminary results from the second cohort of its phase 1/2 clinical trial evaluating INO-3107 for the treatment of HPV 6 and HPV 11-associated recurrent respiratory papillomatosis (RRP) in adults. In the second cohort of 11 patients who were administered INO-3107 via the exploratory side port needle, 10 of the 11 […] February 17, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email