Leslie Gaber Associates’ Post

Wonderful wonderful! Well done Matthew Klein, MD, MS, FACS, Viji (VG) Senthilnathan, and team! Accelerated approval. Singe arm compared with external untreated natural history cohort. Clinically meaningful efficacy endpoint. INDICATIONS AND USAGE KEBILIDI is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. This indication is approved under accelerated approval based on change from baseline in gross motor milestone achievement at 48 weeks post-treatment. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. (1, 14)

This #DiabetesAwarenessMonth, we reaffirm our commitment to transforming the treatment of type 1 diabetes and celebrate our dedicated team and fellow researchers who are at the forefront of pioneering innovative solutions for more effective management of this disease. For the 5 million people in the US and EU living with type 1 diabetes, investing in research is key to improving quality of life and delivering better control of their disease. Discover more about our investigational candidate, Kriya-839, a potential one-time gene therapy for type 1 diabetes: https://bit.ly/4huwzSv #GeneTherapy #Type1Diabetes 

ICYMI Pfizer unveiled detailed Phase 3 AFFINE trial results at #ASH2024 for the treatment of adults with moderately sever to severe hemophilia A. These findings highlight the tolerability of giroctocogene fitelparvovec and its superior bleed protection compared to FVIII prophylaxis. To learn more about this potential gene therapy treatment, click here. https://bit.ly/4g6pqqv #GenomicMedicine #hemophiliaA

⚠️ VERVE-101, a CRISPR-based gene editing therapy for cardiovascular disease, faces safety concerns in its first-of-a-kind base-editing trial. Here's my breakdown of the therapy's milestones: 🌐 View milestones of other drugs with my free app: https://lnkd.in/dVGQ6-8S

Check out our newest white paper, where we explore the Cell and Gene Therapy (CGT) Access Model that was announced by CMS in January 2024. The stated aim of this model is to increase access to CGTs and outcomes-based agreements for state Medicaid programs, thereby helping to mitigate the inherent complexities and financial uncertainties associated with CGTs. This paper is a great resource for understanding the Access Model, process, timelines, and limitations. #genetherapy #celltherapy #Medicaid #OBA #CGTAccessModel Palak Desai, Prachi Bhatt, PharmD, MPH, BCPS, BCCCP, Tyler Engel https://lnkd.in/gHpSXHZh

Gene therapy trials in Inherited Retinal Diseases (IRDs) present unique challenges for biopharma drug developers. Choosing MERIT as your clinical endpoint expert gives you access to solutions based on experience supporting 31 #genetherapy trials. Learn about common challenges and how MERIT solves for these in our info sheet: https://lnkd.in/gmGWFCG8 #MERITCRO #YourClinicalEndpointExpert #genetherapy #clinicaltrials #inheritedretinaldiseases #clinicalresearch

Did you know that the ATTC network is made up of 4 Advanced Therapy Treatment Centres spread across the UK? ➡️ https://ow.ly/liux50U63c6 Coordinated by the Cell and Gene Therapy Catapult, the ATTC network operates within the NHS framework to address the unique and complex challenges of bringing pioneering advanced therapy medicinal products (ATMPs) to patients. Learn more 👆 #ATTCnetwork #PatientAccess #AdvancedTherapies

Excellent news and good to see the IMF finally being used as was originally intended. Perhaps this breakthrough deal with CSL Behring's Hemgenix (haemophilia B) will now give industry more confidence and an 'art-of-the-possible' mindset in order to create more managed access for promising new non-cancer medicines. #access #health and access to medicines # pricing # value # IMF # NICE # NHS commercial framework for new medicines

We've published final guidance recommending etranacogene dezaparvovec, the world's first #GeneTherapy for #haemophiliaB, on the NHS.      This one-off infusion, which could benefit around 260 adults with moderately severe or severe haemophilia B, is the first gene therapy we’ve looked at in our technology appraisal programme and our first recommended haemophilia treatment.     It's also the first pilot for the outcomes-based scheme in the 2024 VPAG (voluntary scheme for branded medicines pricing, access and growth), and also the first medicine to go in the Innovative Medicines Fund. 🔗 https://lnkd.in/geyKHpqR

 📢 Exciting News 📢 The European Commission has granted Orphan Drug Designation (#ODD) to our gene therapy product, VTX-806, for the treatment of cerebrotendinous xanthomatosis (#CTX), a rare #neurodegenerativedisease in patients.   Pre-clinical data of VTX-806 in mice demonstrated normalization of metabolic parameters in blood and brain, and of upregulated compensatory enzymes, supporting VTX-806’s potential as an alternative treatment option to stop, or reverse, disease progression over the long-term, or possibly even cure CTX patients!   Read more🗞️about #Vivet's ODD approval and next steps as the team press forward with anticipation of submitting ODD to the US FDA in 2024 and commence planning preparations to enter clinical development in late 2025 in the link in the comments below 👇   #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT #GeneTherapy #AAVectors