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
News and Trends 24 Feb 2023 Researchers discover how tumor cells become resistant to colorectal cancer chemotherapy Platinum-based chemotherapy, which is used to treat advanced colorectal cancer, accumulates in the healthy cells surrounding the cancer cells and, as a result, can reduce tumor sensitivity to treatment. This is demonstrated by a study published in the journal Nature Communications by the Hospital del Mar Medical Research Institute (IMIM-Hospital del Mar), in collaboration with […] February 24, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 23 Feb 2023 Depression trial gets green light Beckley Psytech Ltd has received investigational new drug (IND) approval from the U.S. Food and Drug Administration (FDA) for a global multi-site phase IIb study exploring the safety, efficacy and tolerability of two doses of its lead compound, BPL–003, in patients with treatment resistant depression (TRD). BPL-003 is the Beckley Psytech’s novel synthetic formulation of […] February 23, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 23 Feb 2023 Positive topline data from sepsis trial Modus Therapeutics AB has published positive top-line data from its phase 1b lipopolysaccharide (LPS) provocation study, a key step in evaluating the potential of its lead asset, sevuparin, as a treatment for sepsis and other conditions with systemic inflammation. In the study, healthy volunteers received LPS to induce a transient systemic inflammation reaction together with […] February 23, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email