As an early investor in Dyne Therapeutics, CureDuchenne is pleased to share that Dyne announced positive data from their Phase 1/2 trial of DYNE-251 in individuals with #Duchenne amenable to skipping exon 51. Individuals treated with 20 mg/kg once every 4 weeks for 6 months showed a mean dystrophin expression of 3.7% of normal when unadjusted for muscle content; when adjusted for muscle contest, the mean was 8.72% of normal dystrophin. Improvements were observed in multiple functional endpoints in the 20 mg/kg cohort at 6 months, as well as the 10 mg/kg group which was escalated to 20 mg/kg during the open-label extension (6 months to 1 year). Dyne is initiating registrational cohorts and will update us later this year with their plans for pursuing FDA approval via the accelerated pathway. #exonskipping #CureDuchenne #DMD #FDA #ClinicalTrials https://lnkd.in/g94sfmix
CureDuchenne’s Post
More Relevant Posts
-
Last week, Argenica Therapeutics lodged our Appendix 4C Quarterly Cashflow Report for the 12-month period ended 30 June 2024. One key highlight from the report was the progress made in our Phase 2 Clinical Trial. Here is an overview of the highlight Access the full report here ➡️ https://bit.ly/4ftygiq #ASX #AGN #investorupdate #stroke #clinicaltrial
To view or add a comment, sign in
-
Cadrenal Therapeutics, Inc. (Nasdaq: CVKD) invites you to our presentation at the Sidoti Micro-cap Virtual Conference on August 15, 2:30-3:00 pm ET in Track 3. Douglas Losordo, MD, Chief Medical Officer of Cadrenal Therapeutics, will provide an overview of our progress in developing our lead candidate, tecarfarin. With FDA orphan drug and fast-track designations, tecarfarin is a novel oral vitamin K antagonist designed to meet critical unmet chronic anticoagulation needs in patients with implanted cardiac devices and rare cardiovascular conditions. We look forward to discussing our clinical milestones and business development strategy with investor participants. https://lnkd.in/eFsv-hQB #CadrenalTherapeutics #Anticoagulation #Tecarfarin #CardiovascularHealth #PharmaInnovation #Biotech #OrphanDrug #FastTrack #Healthcare #SidotiConference #ClinicalTrials #CVKD #LVAD
To view or add a comment, sign in
-
Chief Financial Officer at Cadrenal Therapeutics, Inc. (Nasdaq: CVKD) - advancing its late-stage drug candidate tecarfarin, a new blood thinner for patients with implanted cardiac devices and rare CV conditions
Cadrenal Therapeutics, Inc. (Nasdaq: CVKD) invites you to our presentation at the Sidoti Micro-cap Virtual Conference on August 15, 2:30-3:00 pm ET in Track 3. Douglas Losordo, MD, Chief Medical Officer of Cadrenal Therapeutics, will provide an overview of our progress in developing our lead candidate, tecarfarin. With FDA orphan drug and fast-track designations, tecarfarin is a novel oral vitamin K antagonist designed to meet critical unmet chronic anticoagulation needs in patients with implanted cardiac devices and rare cardiovascular conditions. We look forward to discussing our clinical milestones and business development strategy with investor participants. https://lnkd.in/g5yPtHDr #CadrenalTherapeutics #Anticoagulation #Tecarfarin #CardiovascularHealth #PharmaInnovation #Biotech #OrphanDrug #FastTrack #Healthcare #SidotiConference #ClinicalTrials #CVKD #LVAD
To view or add a comment, sign in
-
Do you want an update on what's been happening at Argenica Therapeutics? This morning, we released our most recent Investor Presentation, providing an update on our Phase 2 Clinical Trial. Read the full presentation here➡️ https://bit.ly/4f4NZ70 #clinicaltrial #investorupdate #AGN #ASX #stroke #strokeawareness
To view or add a comment, sign in
-
Chief Financial Officer at Cadrenal Therapeutics, Inc. (Nasdaq: CVKD) - advancing its late-stage drug candidate tecarfarin, a new blood thinner for patients with implanted cardiac devices and rare CV conditions
Today Cadrenal Therapeutics, Inc. (Nasdaq: CVKD) shared an exciting analysis of a clinical trial conducted by Abbott supporting our belief that Cadrenal's lead drug candidate tecarfarin could be an important next-generation anticoagulant for patients with LVADs. Quoted in the news release lead investigator of the trial, Dr. Mandeep Mehra, states, "…Tecarfarin could potentially be an important therapy for patients with LVADs who all require chronic anti-coagulation since it does not get affected by drug-drug interactions or changes in kidney function like warfarin and deserves further study." Read more: https://lnkd.in/gypE4XZc #cadrenal #nasdaq #cvkd #therapeutics #tecarfarin Quang Pham Matthew Szot
To view or add a comment, sign in
-
🚀𝐀𝐜𝐜𝐞𝐥𝐞𝐫𝐚𝐭𝐢𝐧𝐠 𝐈𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧: 𝐊𝐲𝐯𝐞𝐫𝐧𝐚 𝐒𝐞𝐜𝐮𝐫𝐞𝐬 𝐀𝐝𝐝𝐢𝐭𝐢𝐨𝐧𝐚𝐥 𝐅𝐃𝐀 𝐅𝐚𝐬𝐭 𝐓𝐫𝐚𝐜𝐤, 𝐈𝐏𝐎 𝐨𝐧 𝐭𝐡𝐞 𝐇𝐨𝐫𝐢𝐳𝐨𝐧 🌐 𝗜𝗻 𝗝𝗮𝗻𝘂𝗮𝗿𝘆 𝟮𝟬𝟮𝟰, Kyverna Therapeutics accelerates into another FDA fast track following IPO plans. As KYV-101 moves through the clinical trial stage, Kyverna will have more regular and convenient communication with U.S. regulators thanks to the fast track tag—this time for refractory progressive multiple sclerosis. Additionally, if and when Kyverna files a new #drug application, it allows the #business to request for priority review, which shortens the FDA approval review period to six months. Recently, Kyverna was able to obtain a fast-track tag for myasthenia gravis and approve the entry of its autologous CD19-targeting cell treatment into a phase 2 open-label trial for #patients suffering from #multiplesclerosis. 𝑳𝒐𝒐𝒌 𝒂𝒕 𝒐𝒖𝒓 𝑪𝒐𝒓𝒓𝒆𝒍𝒂𝒕𝒆𝒅 𝑹𝒆𝒑𝒐𝒓𝒕👉:https://bit.ly/3RPw6Qb #FDAFastTrack #IPOJourney #BiotechAdvancement #healthcareinnovation #acceleratingsuccess #biopharma #prophecymarketinsights
To view or add a comment, sign in
-
We are thrilled to announce that Cadrenal Therapeutics, Inc. has received FDA Orphan Drug Designation (ODD) for tecarfarin, our novel blood thinner, aimed at preventing thromboembolism and thrombosis in patients with LVADs, RVADs, Biventricular Assist Devices, and Total Artificial Hearts. This designation not only provides potential market exclusivity for up to seven years after approval, but also opens up expanded partnering opportunities for tecarfarin. At Cadrenal, we are deeply committed to addressing unmet needs in anticoagulation therapy. With this second FDA ODD, we're one step closer to making a meaningful difference in the lives of patients with rare cardiovascular conditions who require chronic anticoagulation. Read more about this milestone: https://lnkd.in/gkdCV25y #CadrenalTherapeutics #FDAODD #OrphanDrugDesignation #novelbloodthinner #Tecarfarin #HealthcareInnovation #CVKD Quang Pham
To view or add a comment, sign in
-
“Venturing Forward, Innovating for Impact” | GP @ 1004 | Venture Capital, Strategic M&A Advisory, Investment Banking | Board of Directors | Healthcare, Life Sciences, and Longevity
Good start to the week. Merck (NYSE: MRK) and Harpoon Therapeutics, Inc. (NASDAQ: HARP) announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire Harpoon for $23.00 per share in cash for an approximate total equity value of $680 million. https://lnkd.in/gthYiVjA Novartis is set to acquire autoimmune-focused Calypso Biotech for $250 million upfront, laying claim to the first pharma buyout of 2024 just hours before the JP Morgan Healthcare Conference kicks off. https://lnkd.in/g9_CTYu9 Axonics, Inc. (NASDAQ: AXNX) entered into a definitive agreement to be acquired by Boston Scientific Corporation (NYSE: BSX) for $71 in cash per share, representing an equity value of approximately $3.7 billion. Johnson & Johnson (NYSE: JNJ) announced it has entered into a definitive agreement to acquire Ambrx Biopharma, Inc (NASDAQ: AMAM), a clinical-stage biopharmaceutical company with a proprietary synthetic biology technology platform to design and develop next-generation antibody-drug conjugates (ADCs), in an all-cash merger transaction for a total equity value of approximately $2.0 billion, or $1.9 billion net of estimated cash acquired. https://lnkd.in/gQ4uDSVF #biotechnology #mergersandacquisitions #jpm2024
To view or add a comment, sign in
-
The hereditary angioedema therapeutics market size is expected to see rapid growth in the next few years. It will grow to $13.47 billion in 2028 at a compound annual growth rate (CAGR) of 18.8%. https://lnkd.in/gwmKbFZY #marketresearch #marketintelligence #marketreport #industryanalysis #TheBusinessResearchCompany #TBRC #GlobalMarketModel
To view or add a comment, sign in
-
As more clinical trials in #Psychedelics emerge, it’s important to design studies with the patient’s condition in mind. Make sure to check out my team member’s presentation at the Psychedelic Therapeutics & Drug Development Conference to learn more! https://bit.ly/4bnRvXY
Presentation | Psychedelic Therapeutics & Drug Development Conference
To view or add a comment, sign in
5,659 followers