Arabella White’s Post

🙌 Exciting news for liver disease, as Gilead Sciences acquires CymaBay Therapeutics! The acquisition gives Gilead ownership of Seladelpar, a drug to treat Primary Biliary Cholangitis (PBC), a disease which affects the bile ducts that run through the liver. The therapy is currently under review by the FDA, following a recent Phase III RESPONSE trial, which showed statistically significant results versus a placebo. As a fellow therapy developer in the liver disease space, this news is heartening – demonstrating that persistence, adaptability, and focused execution are key to advancing novel medicines for patients. Read more about the acquisition at STAT ⬇️ #biotech #genomics #LiverDisease

iRegene Receives IND approval from U.S. FDA to Start Clinical Trial for Parkinson’s Disease - https://lnkd.in/gbKS7Z8M On June 20 (UTC-4), the U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene Therapeutics targeting Parkinson’s Disease. This marks a significant milestone as NouvNeu001 becomes the world’s first chemically induced allogeneic cell therapy product to enter clinical stages for Parkinson’s disease in the US. Previously, the “Combined Phase I/II Clinical Study” of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. On June 20, 2024, iRegene further announced IND approval of NouvNeu001 by the U.S. FDA for the treatment of mid-to-late stage of Parkinson’s disease. Read more at: https://lnkd.in/gbKS7Z8M #parkinsonsdisease #regenerativemedicine #stemcell

iRegene Receives IND approval from U.S. FDA to Start Clinical Trial for Parkinson’s Disease - https://lnkd.in/g6vekNF6 On June 20 (UTC-4), the U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene Therapeutics targeting Parkinson’s Disease. This marks a significant milestone as NouvNeu001 becomes the world’s first chemically induced allogeneic cell therapy product to enter clinical stages for Parkinson’s disease in the US. Previously, the “Combined Phase I/II Clinical Study” of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. On June 20, 2024, iRegene further announced IND approval of NouvNeu001 by the U.S. FDA for the treatment of mid-to-late stage of Parkinson’s disease. Read more at: https://lnkd.in/g6vekNF6 #parkinsonsdisease #regenerativemedicine #stemcell

TL1A is a high-value target with implications across several auto-immune inflammatory diseases, including ulcerative colitis and Crohn’s disease. In October 2023, Roche (SIX: ROG) agreed to acquire rights to RVT-3101, Roivant Sciences’ (NASDAQ: ROIV) TL1A-targeting antibody, for $7.1 billion upfront and $150 million in upcoming milestone payments. Visible Alpha consensus estimates for RVT-3101 project peak global revenues of nearly $2.5 billion in 2032 (not adjusted for risk). #VisibleAlpha #biotech #pharma #autoimmunediseases https://lnkd.in/dZh8VkHe

Yesterday's decision by the FDA to accept CSF heparan sulfate as an endpoint to support #AcceleratedApproval in #SanfilippoSyndrome marks a significant victory for patients. This pivotal moment follows our collaborative workshop in February 2024 with the Reagan-Udall Foundation for the FDA, focusing on "Developing Biomarkers to Support Accelerated Approval in Rare Diseases." Thank you to the Reagan-Udall Foundation for the FDA for spearheading the discussion, alongside the FDA, Cure Sanfilippo Foundation, National MPS Society, The Ryan Foundation, National Organization for Rare Disorders and the academics and co-sponsors, Denali Therapeutics, Orchard Therapeutics - U.S., Allievex Corporation, and REGENXBIO Inc., who joined us. We are optimistic that this is the beginning of rethinking the approval pathway for ultrarare diseases by using disease-causing biomarkers as primary endpoints to support accelerated approvals.  #RareDisease #GeneTherapy #MPS

The FDA has just approved donanemab (Kisunla), a monoclonal antibody developed by Eli Lilly and Company to slow the progression of early symptomatic Alzheimer’s disease. Donanemab helps remove amyloid plaque buildup in the brain, a key factor in Alzheimer’s. Clinical trials have shown significant results, with a 35% lower risk of disease progression over 18 months. While not a cure, donanemab marks the second entry into the first class of disease-modifying drugs for Alzheimer's. This incredible progress is the result of more than 20 years of biomarker research and highlights the importance of public-private collaboration in advancing such medical breakthroughs. The FNIH is committed to continuing its work in developing robust, shareable data sets and sharing knowledge, expertise, and resources to allow researchers to expand their thinking and add new knowledge to solve complex problems like Alzheimer’s and other neurodegenerative diseases. Link here: https://lnkd.in/gcdswNp7 #Alzheimers #AlzheimersResearch #Donanemab #NeurodegenerativeDisease #BiomarkerResearch #AMPAD #BiomarkersConsortium #FNIH

🌟Exciting News on 1st FDA-approved NASH Treatment!   A 40-year barrier in NASH drug discovery has been broken by Madrigal's Rezdiffra™ (resmetirom) becoming the 1st FDA-approved NASH treatment (https://lnkd.in/egBDf6ya.) Benefit from that breakthrough with Eurofins Discovery’s cutting-edge NASH drug discovery services, ranging from comprehensive target- and translational-based portfolios to in vivo efficacy testing. Our established program uses advanced cellular and animal models closely resembling clinical conditions. All are designed to identify promising compounds for novel NASH drug discovery.   With more than 40 targets currently being explored against NASH, including THR-β (resmetirom’s target), FXR, FGFR1, PPAR, and GLP1R, the potential of the field is immense.   Our Metabolic Dysfunction-Associated Steato-Hepatitis (MASH, formerly as NASH) LeadHunter® Panel provides pivotal targets driving steatosis, hepatitis, to fibrosis in NASH progression. Combined with translational liver fibrosis cell model, BioMAP® fibrosis panel and highly clinically relevant humanized liver chimeric mouse models, this leads to a promising approach for your next NASH therapy.   💡 Accelerate your drug discovery journey for NASH with us!   Learn more about our MASH Panel: https://lnkd.in/gyCmByxp   Speak to an Expert Today! https://lnkd.in/gBqd2kzn   #NASH #DrugDiscovery #EurofinsDiscovery #MedicalBreakthroughs

At VascularStrategies, we are passionate about helping sponsors develop life-saving therapeutics for cardiovascular disease. To do this, we offer specialty and proprietary functional assays, useful for all phases of drug development, spanning bench to bedside. Our research and validated assays, and expert resources support you in moving your drug forward as quickly and cost-effectively as possible. Don’t miss out on #cardiovascularresearch updates. Follow us! #atherosclerosis #clinicalresearch #assays #drugdevelopment #hdl #frat

Please consider participating in an important Phase 2a clinical trial for patients with non-tuberculous mycobacterial pulmonary disease (NTM-PD) sponsored by Spero Therapeutics. Eligible participants are either treatment-naïve or have received treatment for NTM-PD in the past and responded well. The study will compare two doses of oral SPR720 monotherapy, 500 mg and 1000 mg, with placebo (no active drug). The goal of Study SPR720-202 is to lay the groundwork for a later-stage and longer-term trial evaluating SPR720, in combination with current standard-of-care agents. For more information on eligibility, click here. https://bit.ly/3UlhsQw

Vertex Pharmaceuticals announced today that FDA has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. “On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, MD FASN, CEO and President of Vertex. “TDT patients deserve new, potentially curative treatment options, and we look forward to bringing CASGEVY to eligible patients who are waiting.” #BetaThalassemia #geneediting #CASGEVY #Vertex 👉 Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia | Vertex Pharmaceuticals Newsroom (vrtx.com)