Question: “What are some of the challenges observed in nonclinical studies that could later affect IND submissions?” Find the answer to this question & more from our #CBEROTP Virtual Town Hall on nonclinical assessment for cell and #GeneTherapy products. https://bit.ly/46AOMHo
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Decoding Methoxsalen: A Comprehensive Study of its R&D Trends and Mechanism on Drug Target https://lnkd.in/gF8EVQuZ
Decoding Methoxsalen: A Comprehensive Study of its R&D Trends and Mechanism on Drug Target
synapse.patsnap.com
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AFHEA, AMRSC, FCPS, MPhil (Cantab) | Research Associate, Broad Institute of MIT and Harvard | PhD Student in Chemistry, University of Cambridge | Former President of the Graduate Student Body
New Publication Alert! 🔬💊 I We started with a null library with excellent pharmacokinetics (DMPK) but no initial target potency. By applying MolDQN, a generative model, we successfully integrated activity against α-synuclein aggregation in Parkinson’s disease. #MachineLearning #DrugDiscovery #Innovation #Pharmacokinetics #ParkinsonsDisease
Using Generative Modeling to Endow with Potency Initially Inert Compounds with Good Bioavailability and Low Toxicity
pubs.acs.org
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First CRISPR-created therapeutic approved! 28/29 and 39/42 response rates... The cells are modified and infused back so no direct delivery of editing agent...which also make it expensive and lengthy. But it could be a permanent fix so definitely worth it! Great achievement well done Vertex! https://lnkd.in/g6p88UP4
UK approves Vertex, CRISPR therapy for sickle cell disease, beta-thalassemia in world first
https://endpts.com
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Very interesting review about the use of microdoses and isotopic microtracers for clinical intravenous pharmacokinetic data provision. Congratulations to the team for the great job! Thanks to Graeme Young End of
Translation for leading the team work of this manuscript 🙂 Graeme Young, Douglas Spracklin, Alex James, Mette Graulund Hvenegaard, Mette Lund Pedersen, David Wagner, Katrin Georgi, Hanno Schieferstein, Inga Bjørnsdottir, Andrea A. Romeo, Ken Cassidy, Georges Da violante, Stefan Blech, Simone Schulz, Filip Cuyckens, Mai Anh Nguyen, Graeme Scarfe First published: 29 November 2023 https://lnkd.in/erFiDurS https://lnkd.in/efayMfp9 A review of the use of microdoses and isotopic microtracers for clinical intravenous pharmacokinetic (IV PK) data provision is presented. The extent of application of the varied approaches available and the relative merits of each are highlighted with the aim of assisting practitioners in making informed decisions on the most scientifically appropriate design to adopt for any given new drug in development. It is envisaged that significant efficiencies will be realized as IV PK data in humans becomes more routinely available for suitable assets in early development, than has been the case prior to the last decade.To view or add a comment, sign in
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Applying binary solutions to a multifactorial problems is something which should be written on top of every single prescription ever.
https://lnkd.in/gn2vF-7j Insights on CYP2C19 and phenoconversion Interesting #pgx paper on my favorite topic - phenoconversion. In Invitae's YouScript CDST, we estimate cumulative expected increases or decreases in exposure based on summing exposure change ranges seen for the binary components in the published literature. Sometimes this results in an increase or decrease in exposure in a different range - like a change from moderate to potent inhibition - and sometimes it does not. We already have 40 million patients on 5+ medications with the number anticipated to double by 2040. We may not have the perfect solution for estimating phenoconversion yet, but applying binary solutions to a multifactorial problem is not the answer. #pharmacogenetics #precisionmedicine is #patientsafety
PharmGKB Blog
pharmgkb.blogspot.com
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FDA new guidance on #cellandgenetherapies is groundbreaking and an important read for any companies engaging in this science for human use. Great article breaking it down. Those looking to provide draft guidance comment should definitely give this a read...
On Jan. 29, 2024, the FDA released the final version of the guidance document on human #genetherapy products incorporating #genomeediting. This article by Dark Horse Consulting Group Inc.'s Brent Morse and Nathan Manley shares what changed between the draft and now final version of the guidance, and what open questions remain for the industry and scientific community to help resolve. #cellandgenetherapy #FDA #cmc #drugproduct https://lnkd.in/eQBpbdNf
FDA's Updated Guidance On Human Genome Editing: New Implications & Remaining Questions
cellandgene.com
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The #PatientJourney for #CellandGeneTherapy is complex and challenging. Drug developers can improve #PatientAccess with an effective Patient Services Hub, which can significantly benefit the many stakeholders in a CGT program. Resource below. #gotomarketstrategy AmerisourceBergen
Improving Access and Support for Cell and Gene Therapies
resources.industrydive.com
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Self-motivated scientist with extensive knowledge in immunology, gene and cell therapy, lentivirus transduction, and self-assembled nanomaterials
#genetherapy This is a comprehensive article
On Jan. 29, 2024, the FDA released the final version of the guidance document on human #genetherapy products incorporating #genomeediting. This article by Dark Horse Consulting Group Inc.'s Brent Morse and Nathan Manley shares what changed between the draft and now final version of the guidance, and what open questions remain for the industry and scientific community to help resolve. #cellandgenetherapy #FDA #cmc #drugproduct https://lnkd.in/eQBpbdNf
FDA's Updated Guidance On Human Genome Editing: New Implications & Remaining Questions
cellandgene.com
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Clinical Pharmacist /Collaborative Practice Agreement / Educator- Speaker/ MTM / Prior Authorizations/ Medical Writer/ Medicolegal Consulting/ 5 Board certifications in Pharmacy
In this clinical trial with healthy participants, compared with 1 intranasal naloxone dose administered at 0 and 2.5 minutes, 1 dose at 0, 2.5, 5, and 7.5 minutes significantly increased naloxone plasma concentration at 10 minutes, whereas 2 doses at 0 and 2.5 minutes significantly increased naloxone plasma concentration at 4.5 minutes. Additional research is needed to determine optimal naloxone dosing in the community setting. #naloxone #opioidcrisis
Intranasal Naloxone Repeat Dosing Strategies and Fentanyl Overdose
jamanetwork.com
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As we roll into 2024, what do the experts believe is next for #drugdevelopment and #clinicaltrials? According to Curavit's CEO, Joel Morse, #healtheconomics and outcomes research (#HEOR) will be come a lot more common in decentralized and hybrid clinical trials. Check out what Joel and other experts have to say on the matter in this PharmaVoice article! #DCTs #clinicalresearch #DTx #virtualsite https://lnkd.in/edNCtJha
PharmaVoice’s Crystal Ball: What’s next in drug innovation and clinical trials
pharmavoice.com
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