News and Trends 2 Mar 2023 New lipid nanoparticle CRISPR delivery system developed Lipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein. Gene therapy is a potential mode of treatment for a wide variety of diseases caused by genetic mutations. While it has been an area of diverse and […] March 2, 2023 - 3 minutesmins - By Jim Cornall 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 Peter MacCallum Cancer Centre and Cartherics to work on CAR-T cell therapy for ovarian cancer Peter MacCallum Cancer Centre (Peter Mac) in Australia and Cartherics Pty Ltd have entered into a collaborative development program agreement (CDPA) to develop Cartherics’ proprietary autologous CAR-T cell therapy (CTH-004) for the treatment of ovarian cancer. The clinical trial to be conducted by Peter Mac will be the first time a CAR-T cell therapy product […] February 28, 2023 - 4 minutesmins - By Jim Cornall 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 2023 Resalis Therapeutics receives €10M to develop obesity and NAFLD treatment Resalis Therapeutics has completed a seed financing round of €10 million to establish a non-coding RNA (ncRNA)-based therapeutic approach for the treatment of metabolic diseases. The funding will support the finalization of IND-enabling studies for the Resalis Therapeutics’ lead program, RES-010, which Resalis will initially develop as a novel treatment for obesity and nonalcoholic fatty […] February 24, 2023 - 2 minutesmins - By Jim Cornall 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 24 Feb 2023 AELIX announces ‘important step towards HIV eradication’ AELIX Therapeutics S.L., a clinical-stage biotechnology company specializing in the discovery and development of immunotherapies for HIV infection, has announced positive topline results from its AELIX-003 trial. The study evaluated the safety, tolerability, immunogenicity and efficacy of AELIX Therapeutics’ HTI T-cell therapeutic HIV vaccine in combination with Gilead’s investigational Toll-Like Receptor 7 (TLR7) agonist, vesatolimod […] February 24, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 24 Feb 2023 Sanofi hemophilia drug gets FDA approval The U.S. Food and Drug Administration (FDA) has approved Sanofi’s ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia […] February 24, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email