B7H3 (CD276) is an immune checkpoint and is widely expressed on tumor cell. The B7H3/TROP2 ADC shows significant activity especially in small cell lung cancer and head and neck cancer. Toxicity reflects the payload. B7H3 levels by IHC didn’t correlate with outcome @NatureMedicine
𝐅𝐃𝐀 𝐂𝐥𝐞𝐚𝐫𝐬 𝐅𝐢𝐫𝐬𝐭-𝐄𝐯𝐞𝐫 𝐈𝐧 𝐕𝐢𝐯𝐨 𝐆𝐞𝐧𝐞-𝐄𝐝𝐢𝐭𝐢𝐧𝐠 𝐓𝐫𝐢𝐚𝐥 𝐟𝐨𝐫 𝐂𝐡𝐫𝐨𝐧𝐢𝐜 𝐇𝐞𝐩𝐚𝐭𝐢𝐭𝐢𝐬 𝐁 𝐢𝐧 𝐭𝐡𝐞 𝐔𝐧𝐢𝐭𝐞𝐝 𝐒𝐭𝐚𝐭𝐞𝐬
Precision BioSciences, Inc. has received IND clearance from the FDA for PBGENE-HBV, making it the first investigational in vivo gene-editing therapy to enter a clinical trial for chronic Hepatitis B virus (HBV) in the United States.
PBGENE-HBV is designed to eliminate cccDNA and inactivate integrated HBV DNA that current treatments cannot effectively address.
Read more in our latest clinical trial update 👇
https://t.co/LkQi87hcBs
#crisprmedicinenews #crisprmedicine #crispr #geneediting #chronichepatitisB #hbv #geneeditingtrial #meganuclease
Today marks the beginning of OpenRxiv, which replaces bioRxiv and medRxiv, the world's largest preprint platform for life and medical science @openrxiv
New "triple-action" CRISPR method enables simultaneous gene activation, repression & deletion in human T cells!
Head over to @CrisprMedicine to read the full interview with @RasmusBak's team at @AarhusUni that developed this new approach for cell therapy.
#CRISPR#GeneTherapy
𝐍𝐞𝐰 𝐓𝐫𝐢𝐩𝐥𝐞-𝐀𝐜𝐭𝐢𝐨𝐧 𝐂𝐑𝐈𝐒𝐏𝐑 𝐓𝐞𝐜𝐡𝐧𝐨𝐥𝐨𝐠𝐲 𝐄𝐧𝐚𝐛𝐥𝐞𝐬 𝐒𝐢𝐦𝐮𝐥𝐭𝐚𝐧𝐞𝐨𝐮𝐬 𝐆𝐞𝐧𝐞 𝐀𝐜𝐭𝐢𝐯𝐚𝐭𝐢𝐨𝐧, 𝐑𝐞𝐩𝐫𝐞𝐬𝐬𝐢𝐨𝐧, 𝐚𝐧𝐝 𝐃𝐞𝐥𝐞𝐭𝐢𝐨𝐧 𝐢𝐧 𝐏𝐫𝐢𝐦𝐚𝐫𝐲 𝐇𝐮𝐦𝐚𝐧 𝐓 𝐂𝐞𝐥𝐥𝐬
Scientists at Aarhus University have developed a method that allows for simultaneous activation, repression, and knockout of genes within a single cell. By leveraging orthogonal CRISPR/Cas systems, the team, led by Rasmus O. Bak, has demonstrated the potential of advanced CRISPR technologies for innovative applications in gene therapy and regenerative medicine.
Read more in our latest interview with co-first authors of the study, Amalie Dyrelund Broksø and Louise Bidstrup-Bendixen.
✒️ Interview by: Christos Evangelou, PhD, BCMAS
https://t.co/1tmpKAoxId
📸 Photo, from left to right: Simon Bøje Fammé, Louise Bidstrup-Bendixen, Amalie Dyrelund Broksø, and Rasmus O. Bak.
#crisprmedicinenews #crisprmedicine #crispr #geneediting #crispra #crispri #geneticengineering
𝐄𝐟𝐟𝐢𝐜𝐢𝐞𝐧𝐭 𝐧𝐨𝐧-𝐯𝐢𝐫𝐚𝐥 𝐢𝐦𝐦𝐮𝐧𝐞 𝐜𝐞𝐥𝐥 𝐞𝐧𝐠𝐢𝐧𝐞𝐞𝐫𝐢𝐧𝐠 𝐮𝐬𝐢𝐧𝐠 𝐜𝐢𝐫𝐜𝐮𝐥𝐚𝐫 𝐬𝐢𝐧𝐠𝐥𝐞-𝐬𝐭𝐫𝐚𝐧𝐝𝐞𝐝 𝐃𝐍𝐀-𝐦𝐞𝐝𝐢𝐚𝐭𝐞𝐝 𝐠𝐞𝐧𝐨𝐦𝐢𝐜 𝐢𝐧𝐭𝐞𝐠𝐫𝐚𝐭𝐢𝐨𝐧
Scientists at Full Circles Therapeutics Inc. have developed a novel circular single-stranded DNA technology that enables highly efficient gene integration in various cell types, achieving up to 70% successful gene integration.
The system can carry genetic sequences of up to 20 kb in length, showing promise for CAR-T cell therapy development.
Their work was published in Nature Biotechnology this morning.
Read more in our interview with co-founder and CSO Hao Wu 👇
https://t.co/YldNSaRZ7H
Interview by Christos Evangelou, PhD, BCMAS
#crisprmedicinenews #crisprmedicine #breakingnews #cancertherapy #nonviralgenetherapy #geneediting #crispr
𝐄𝐧𝐠𝐢𝐧𝐞𝐞𝐫𝐞𝐝 𝐏𝐨𝐥𝐲𝐦𝐞𝐫𝐬 𝐒𝐡𝐨𝐰 𝐏𝐫𝐨𝐦𝐢𝐬𝐞 𝐟𝐨𝐫 𝐒𝐲𝐬𝐭𝐞𝐦𝐢𝐜 𝐦𝐑𝐍𝐀 𝐃𝐞𝐥𝐢𝐯𝐞𝐫𝐲 𝐭𝐨 𝐋𝐮𝐧𝐠𝐬: 𝐏𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥 𝐈𝐦𝐩𝐥𝐢𝐜𝐚𝐭𝐢𝐨𝐧𝐬 𝐟𝐨𝐫 𝐆𝐞𝐧𝐞𝐭𝐢𝐜 𝐋𝐮𝐧𝐠 𝐃𝐢𝐬𝐞𝐚𝐬𝐞𝐬
Scientists engineered nanoparticles capable of delivering genetic material to lung cells with high efficiency when administered intravenously. Based on biodegradable polymers, this delivery method has demonstrated promising performance in both human lung cells and mouse models, potentially overcoming the limitations of current gene therapy approaches for lung diseases.
Read more in our interview with Erin Kavanagh (Johns Hopkins Medicine):
https://t.co/kYUYpP58ct
Thanks to Christos Evangelou, PhD, BCMAS for the writeup!
#crisprmedicinenews #crisprmedicine #genedelivery #genetherapy #cysticfibrosis #lungdisease #mRNA
🔥Presidential Symposium III: Eyes to the future🔥
TNT with non-operative management for pMMR LARC
#ESMO24
🔎First results of NO-CUT trial
👉cCR 26%
👉DRFS30 97%, NOM 85%
👉ctDNA+ predicts recurrence
🧐Next step to select pts for NOM
@myESMO#ESMOAmbassadors
Neoadjuvant pembrolizumab in dMMR/MSI tumors: Results of the CRC cohort
#ESMO24
🔎IMHOTEP phs II
👉52% pCR
👉pCR rate increased 46% (1 cy) > 68% (2 cy)
👉significant downstaging
🧐Multiple Options for neoadj. therapy in MSI CRC, how to best select?
@myESMO#ESMOAmbassadors
Checkmate 067 10 year data 🚨 presented by James Larkin with concurrent @NEJM publication. 10 years of ipi+nivo revolutionizing melanoma care and changing the way we treat cancer!! 10y MSS is >50%! #ESMO24@OncoAlert https://t.co/tHBIwqCppk
Perioperative durvalumab + neoadjuvant chemo & cystectomy in operable bladder cancer significantly delays (HR 0.68) event free survival and a 25% reduction in the risk of death. 10% ⬆️ pCR. It’s the 1st time immune therapy has an OS benefit in muscle invasive UC #ESM024@NEJM
Neoadjuvant immunotherapy in locally advanced MMR-deficient colon cancer:
#ESMO24
🔎3-year disease-free survival from NICHE-2
👉💯% DFS
👉all MRD pts cleared ctDNA
🧐Impressive results, ctDNA may be helpful to select for organ preservation
@myESMO#ESMOAmbassadors
mFOLFOX plus/minus Nivo & Ipi vs FLOT + nivolumab in metastatic G/GEJ adenocarcinoma
#ESMO24
🔎IKF-AIO-Moonlight trial
👉ORR 46 vs 32 vs 47 vs 56%
👉PFS: 5,8 vs 4 vs 6,6 vs 7 mo
👉mOS: 10 vs 7,6 vs 12,5 vs 14,6 mo
🧐Only FLOT + Nivo seems feasible & effective
@myESMO #ESMOAmbassadors
Today is #WorldSuicidePreventionDay
Every year, more than 700,000 people die due to suicide.
Every death is a tragedy, leaving lasting effects on families & communities.
But, with the right support and interventions, suicide can be prevented https://t.co/IfH4ZzJBkToday is #WorldSuicidePreventionDay
Every year, more than 700,000 people die due to suicide.
Every death is a tragedy, leaving lasting effects on families & communities.
But, with the right support and interventions, suicide can be prevented https://t.co/IfH4ZzJBkToday is #WorldSuicidePreventionDay
Every year, more than 700,000 people die due to suicide.
Every death is a tragedy, leaving lasting effects on families & communities.
But, with the right support and interventions, suicide can be prevented https://t.co/IfH4ZzJBkToday is #WorldSuicidePreventionDay
Every year, more than 700,000 people die due to suicide.
Every death is a tragedy, leaving lasting effects on families & communities.
But, with the right support and interventions, suicide can be prevented https://t.co/IfH4ZzJBkToday is #WorldSuicidePreventionDay
Every year, more than 700,000 people die due to suicide.
Every death is a tragedy, leaving lasting effects on families & communities.
But, with the right support and interventions, suicide can be prevented https://t.co/IfH4ZzJBkr
𝐎𝐩𝐞𝐧𝐂𝐑𝐈𝐒𝐏𝐑-1: 𝐆𝐞𝐧𝐞𝐫𝐚𝐭𝐢𝐯𝐞 𝐀𝐈 𝐌𝐞𝐞𝐭𝐬 𝐂𝐑𝐈𝐒𝐏𝐑
Researchers at @ProfluentBio used large language models to expand the sequence diversity of CRISPR-Cas proteins and generate novel, functional genome editors with improved properties compared to natural systems.
Earlier this year, they publicly released OpenCRISPR-1, the first AI-designed editor for precise genome editing. This novel AI-based approach for designing CRISPR gene editors could potentially expand the capabilities and applications of genome-editing technologies.
Read more in our interview with Profluent CEO and founder Ali Madani.
Interview by Christos Evangelou, PhD (@MedRight_Comms)
https://t.co/oWM1BLidvf
#crisprmedicinenews #crisprmedicine #crispr #AI #AIcrispr #LLM #artificialintelligence #machinelearning #geneediting #designernuclease
The photo shows the Profluent Bio team members involved in this study. Column 1 down: Aadyot Bhatnagar - Machine Learning Scientist, Jeffrey Ruffolo - Head of Protein Design, Stephen Nayfach - Head of Bioinformatics. Column 2 down: Ali Madani - CEO and Founder, Joseph Gallagher - Associate Director. Photos courtesy of Profluent Bio (Thermal PR)
🔥off the press:
@ESMO_Open
Pan-Asian adapted ESMO Clinical Practice Guidelines for the diagnosis, treatment and follow-up of patients with biliary tract cancer
👏Great consensus with our Asian friends & colleagues
👇my personal link to share:
https://t.co/kni1NdjdNk
@myESMO@ILCAnews@EASLnews #livertwitter
It's World #Hepatitis Day.
Here is how you can take action to protect yourself and your loved ones:
🔹 Get tested
🔹 Never share sharp instruments
🔹 Get treated
🔹 Vaccinate babies within 24 hours of birth
https://t.co/No4R4F6tP7