Advancing a pipeline of ASO-based, RNA-targeting therapeutics with the aim to restore healthy protein levels

Cambridge, MA
Joined May 2018
Meet us in Boston as our VP, Head of Clinical Operations, Biljana Bazdar-Vinovrski joins an @IQVIA_Global panel on global clinical development, sharing insights on scaling teams, country selection & execution risks. For more conference information, visit: bit.ly/4hwcn4W
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RNA regulates #GeneExpression & protein levels in the body. Our #ASO therapeutics target regulatory RNAs (#regRNAs) to increase expression of deficient proteins toward the therapeutic range in #HaploinsufficientDiseases like #SYNGAP1. Learn more: camp4tx.com/science/regrna/
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Where does the search for a potential #regRNA therapeutic target begin?​ Hear from our Scientist II, Ali Al Abdullatif, MS in this month’s #CAMP4Fireside series as he explores how we assess regulatory regions & evaluate #ASOs for therapeutic development.​ bit.ly/4ekBntf
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Join us at @BiotechWkBoston as our Scientific Fellow, David Bumcrot, PhD, highlights our approach to targeting regulatory RNAs (#regRNAs) to upregulate SYNGAP #GeneExpression and address the root cause of #SYNGAP1-related disorder. Learn more: bit.ly/4hbJ7PE
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Join us September 28 from 12:00–1:30 PM ET for our virtual Analyst Day. We’ll share updates on our #regRNA-targeting approach and work toward potential #SYNGAP1 treatments, including our planned Phase 1/2 trial for our lead candidate.​ Register here: bit.ly/4d3YTdZ
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Pleased to announce we’ve received authorization in the United Kingdom to expand our Phase ½ human clinical trial of our lead candidate for patients with #SYNGAP1-related disorder. Read more in our press release: bit.ly/4cAKQwl
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Proud to sponsor @BioLogicPharmaS & Cure NDD’s LEAD Project, helping pediatric clinics implement guideline-aligned genomic testing for children with developmental delays. Together, we're advancing early diagnosis & access to genetic evaluation. More here: bit.ly/4wPC0C0
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Excited to announce we're hosting a virtual Analyst Day on Sept. 28, where our team will share updates on our #regRNA-targeting approach for #SYNGAP1, including our planned Phase 1/2 clinical trial for our lead candidate, followed by a live Q&A. Read more: bit.ly/4A2FiVw
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ICYMI: Our CMO, Yuri Maricich, MD, joined Jessica Lynn in this recent @Rareatives article for a conversation on how our #regRNA-targeting approach aims to upregulate #GeneExpression as a potential treatment for #SYNGAP1. Read more: bit.ly/4hWOdkQ
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We’re participating in two upcoming investor conferences in September, including 1x1 meetings at the @WellsFargo Healthcare Conference and a fireside chat with our CEO, Josh Mandel-Brehm, at the @Official_Cantor Global Healthcare Conference. Read more: bit.ly/4i1g3we
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Our CSO, Dan Tardiff, PhD, joined @BiotechVoyager podcast to discuss our #regRNA-targeting approach and the potential to address #HaploinsufficientDiseases like #SYNGAP1. Watch the replay: bit.ly/3UisytA
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#HaploinsufficientDiseases occur when there is only one functional gene copy, leading to 50% reduction in protein levels. Our RAP Platform® aims to upregulate #GeneExpression to restore protein levels & target disease at its source. Learn more: bit.ly/3O2chWU
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In this #CAMP4Fireside Series, our CSO, Dan Tardiff, PhD, discusses why #SYNGAP1 & #HaploinsufficientDiseases are ideal targets for gene upregulation & how our approach aims to increase #GeneExpression through our #regRNA-targeting platform. Learn more: bit.ly/4ekBntf
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ICYMI: Our CEO Josh Mandel-Brehm joined @CNBC Cures' @BeckyQuick to discuss our first-in-human trial for #SYNGAP1-related disorder, including new regulatory approvals in Australia & Argentina and our approach to restoring SYNGAP protein expression. ​Watch: cnb.cx/4wEgdxW
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Join us at @HansonWade’s Oligonucleotides for CNS Summit, where our CSO, Dan Tardiff, PhD, will highlight our SYNGAP program and its potential to upregulate #GeneExpression for #HaploinsufficientDiseases like #SYNGAP1. Learn more: bit.ly/4xbIL1X
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Today, we announced our Q2 2026 financial results and corporate highlights. Read more in our press release: bit.ly/4zf5dJ0
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Our SVP, Technical Operations, Satya Kuchimanchi, PhD, will participate in two conferences by @HansonWade & @CHI_Healthtech, presenting on CAMP4's ASO therapeutics approach & efficient design & process development. Stay up to date on our progress: bit.ly/3UrYfjS
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This #WorldRNADay, we celebrate RNA's transformative impact on genetic research. At CAMP4, we're leveraging regulatory RNAs (#regRNAs) to target the root causes of #HaploinsufficientDiseases by modulating #GeneExpression. Learn more: bit.ly/3O2chWU
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Hear our SVP, Platform, Alla Sigova, PhD, highlight the differences of our #regRNA-targeting approach & other approaches to addressing genetic disease. Learn how we're using #ASOs for the potential treatment of #HaploinsufficientDiseases: bit.ly/4ekBntf #CAMP4Fireside
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