Exciting News! AI Therapeutics has rebranded to OrphAI Therapeutics Inc. as we further our commitment to treating rare diseases. Our new name captures our evolution & dedication to those in need. https://lnkd.in/gX5gKkVF
OrphAI Therapeutics’ Post
More Relevant Posts
-
HDSA San Francisco Bay Area Chapter Raises Funds To Improve the Lives of Everyone Affected by Huntington's Disease
"DelveInsight's, "Huntington's Disease Pipeline Insight 2024" report provides comprehensive insights about 50+ companies and 50+ pipeline drugs in Huntington's Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space."
Huntington's Disease Pipeline Outlook Report 2024 (Updated)
openpr.com
To view or add a comment, sign in
-
HDSA San Francisco Bay Area Chapter Raises Funds To Improve the Lives of Everyone Affected by Huntington's Disease
"DelveInsight's, "Huntington's Disease Pipeline Insight 2023" report provides comprehensive insights about 50+ companies and 50+ pipeline drugs in Huntington's Disease pipeline landscape. It covers the Huntington's Disease pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Huntington's Disease pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space." https://lnkd.in/gCpbF6FM
Huntington's Disease Pipeline, FDA Approvals, Clinical Trials Development and Companies 2023
openpr.com
To view or add a comment, sign in
-
Saghmos Therapeutics is developing ST-62516, a Phase 3-ready, cardiorenal-metabolic modulator to reduce the risk of Acute Kidney Injury (AKI) and Major Adverse Cardiac and Kidney Events (MACKE) after contrast procedures such as percutaneous coronary intervention (PCI). Over one million PCI procedures are performed annually in the US in patients with unstable angina (heart attack). Comorbidities such as heart disease, kidney disease and diabetes are a threat to the safety of the PCI procedure. There are no approved pharmacologic agents to prevent or treat AKI and MACKE so this is an area of high unmet need. ST-62516 has multi-billion dollar revenue potential because it could benefit all PCI patients, not just those with comorbidities, to reduce the threat of AKI and MACKE after PCI.
Saghmos Therapeutics Announces Notice of Allowance for Second US Patent for Phase 3-Ready Cardiorenal Metabolic Modulator ST-62516
businesswire.com
To view or add a comment, sign in
-
42K I Global Medical Journal I 18th Year I Houston I Istanbul I Clinical Trials I Innovative Therapies I Patient Journey I Ethics
Vertex Pharmaceuticals announced today that FDA has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. “On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, MD FASN, CEO and President of Vertex. “TDT patients deserve new, potentially curative treatment options, and we look forward to bringing CASGEVY to eligible patients who are waiting.” #BetaThalassemia #geneediting #CASGEVY #Vertex 👉 Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia | Vertex Pharmaceuticals Newsroom (vrtx.com)
To view or add a comment, sign in
-
-
Medical Affairs and Clinical Development Recruiter | 90% CV to interview ratio | Biotech, Pharma, and CROs | USA & Canada
Clinical Trials Updates: The FDA grants fast-track designation to Certa Therapeutics systemic sclerosis drug candidate. Great news for the treatment of systemic sclerosis! Certa Therapeutics has been granted fast-track designation by the FDA for their drug candidate, FT011. This chronic autoimmune disease causes abnormal connective tissue growth, leading to skin and organ hardening, with no known cure. Certa Therapeutics' phase II trial showed a 60% clinically significant improvement in patients compared to a placebo group. These results are impressive and have led to the drug being fast-tracked for regulatory evaluation, meaning it could reach the market faster. Congratulations to Certa Therapeutics for reaching this milestone and providing hope for those suffering from systemic sclerosis. #clinicaldevelopment #regulatoryaffairs #medicalaffairs #inflammation #sclerosis #FDA https://lnkd.in/e6_DXrGa
Scleroderma treatment by Certa Therapeutics granted US FDA fast track
manufacturingchemist.com
To view or add a comment, sign in
-
BioInformant.com, World's Largest Publisher of Stem Cell News | Real Estate Investor | PatriotHoldings.com, Board Member, & Advisor | Former USA Rugby Player
iRegene Receives IND approval from U.S. FDA to Start Clinical Trial for Parkinson’s Disease - https://lnkd.in/gbKS7Z8M On June 20 (UTC-4), the U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene Therapeutics targeting Parkinson’s Disease. This marks a significant milestone as NouvNeu001 becomes the world’s first chemically induced allogeneic cell therapy product to enter clinical stages for Parkinson’s disease in the US. Previously, the “Combined Phase I/II Clinical Study” of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. On June 20, 2024, iRegene further announced IND approval of NouvNeu001 by the U.S. FDA for the treatment of mid-to-late stage of Parkinson’s disease. Read more at: https://lnkd.in/gbKS7Z8M #parkinsonsdisease #regenerativemedicine #stemcell
To view or add a comment, sign in
-
-
iRegene Receives IND approval from U.S. FDA to Start Clinical Trial for Parkinson’s Disease - https://lnkd.in/g6vekNF6 On June 20 (UTC-4), the U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene Therapeutics targeting Parkinson’s Disease. This marks a significant milestone as NouvNeu001 becomes the world’s first chemically induced allogeneic cell therapy product to enter clinical stages for Parkinson’s disease in the US. Previously, the “Combined Phase I/II Clinical Study” of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. On June 20, 2024, iRegene further announced IND approval of NouvNeu001 by the U.S. FDA for the treatment of mid-to-late stage of Parkinson’s disease. Read more at: https://lnkd.in/g6vekNF6 #parkinsonsdisease #regenerativemedicine #stemcell
To view or add a comment, sign in
-
-
Nirogacestsat: A Discarded Alzheimer’s Drug Becomes the First Approved Gamma-Secretase Inhibitor | https://lnkd.in/gyS4XkrH -2023 Molecule of the Year Nominee (#6/10)- Gamma-secretase is most well-known as a target for Alzheimer’s disease, though such programs have not been successful to date. Nirogacestat is a gamma-secretase inhibitor that Pfizer originally intended to treat Alzheimer’s disease, but has found new life at Pfizer spinout SpringWorks Therapeutics as a treatment as a first-in-class drug for rare and aggressive desmoid tumors (DTs). The molecule was first highlighted as a 2023 Molecule of the Month when it received FDA priority review based on Ph. III data that indicated a reduction in disease progression by 71% in desmoid tumors. In Nov. 2023, nirogacestat received the first FDA approval for a treatment for desmoid tumors after receiving Fast Track, Breakthrough Therapy and Orphan Drug designations, highlighting the importance and urgency for a therapeutic for this particular indication. Continue to the full article to learn how it was discovered, how gamma-secretase inhibition translates to anti-tumor activity, how various confusing assay disconnects were encountered in its discovery and development, and more. Not at one of the 150+ institutions with full Drug Hunter access? Learn how Drug Hunter helps you solve your greatest R&D challenges and request access here: https://lnkd.in/gjZdCZUc
nirogacestat
drughunter.com
To view or add a comment, sign in
-
I am an expert in Communication and Corporate Documentation in the health sector. I help you transform the data of your studies into useful information and disseminate the results of your research.
#Investigación │ Diez ensayos clínicos que deberemos monitorear en la primera mitad de 2024 👇 1. COMPANY: Vertex Pharmaceuticals DISEASE: Acute pain TREATMENT TYPE: Small molecule 2. COMPANY: Alnylam Pharmaceuticals DISEASE: Transthyretin amyloidosis cardiomyopathy TREATMENT TYPE: Small interfering RNA 3. COMPANY: Roche DISEASE: Lung cancer TREATMENT TYPE: Antibody 4. COMPANY: Amylyx Pharmaceuticals DISEASE: ALS TREATMENT TYPE: Small molecule 5. COMPANY: Johnson & Johnson DISEASE: Major depressive disorder TREATMENT TYPE: Small molecule 6. COMPANY: Gilead Sciences DISEASE: Lung cancer TREATMENT TYPE: Antibody-drug conjugate 7. COMPANY: Sanofi DISEASE: Multiple sclerosis TREATMENT TYPE: Small molecule 8. COMPANY: Pfizer DISEASE: Duchenne muscular dystrophy TREATMENT TYPE: Gene therapy 9. COMPANY: Moderna DISEASE: Influenza, COVID-19 TREATMENT TYPE: mRNA vaccine 10. COMPANY: Beam Therapeutics DISEASE: Sickle cell TREATMENT TYPE: Base editing https://lnkd.in/der2CggX
10 clinical trials to watch in the first half of 2024
biopharmadive.com
To view or add a comment, sign in
-
In a press release and call to investors today, March 19, 2024, Design Therapeutics, Inc. provided an update on the progress of GeneTAC™ program in FA. “Our lead program in FA has a new drug product, DT-216P2, that we have designed to have an improved pharmacokinetic and injection site safety profile, which positions us to resume clinical development for this debilitating, degenerative, neuro-muscular genetic disease. This work builds on encouraging data in FA patients from our previous clinical trial." - Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics Design is advancing DT-216P2 for FA, with plans to complete GLP studies by year-end 2024 to start patient trials in 2025. Click here to read the full press release: https://bit.ly/4cmnatS or to access the presentation to investors: https://bit.ly/3IHQhdB
To view or add a comment, sign in
-