Latest Headlines

1. Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted To Nexiguran Ziclumeran (nex-z) For The Treatment Of Hereditary Transthyretin (ATTR) Amyloidosis With Polyneuropathy 11/25/2024

   Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is an in vivo CRISPR-based investigational therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of ATTR amyloidosis.

2. Eton Pharmaceuticals Announces Acquisition Of U.S. Rights To Amglidia (Glyburide Oral Suspension) 11/25/2024

   Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced that it has acquired the U.S. rights to Amglidia (glyburide oral suspension, known as glibenclamide in Europe) for the treatment of neonatal diabetes mellitus from AMMTeK.

3. Duality Biologics Announces Expansion Of Adcendo ApS Collaboration Through Option Exercise Of Strategic License Agreement 11/25/2024

   Duality Biologics ("Duality"), a clinical-stage biotech company focusing on the discovery and development of next generation antibody-drug conjugate therapeutics today announced the expansion of its ongoing collaboration with Adcendo ApS ("Adcendo"), a biotech company focused on the development of first-in-class antibody-drug conjugates (ADCs) for the treatment of cancers with high unmet medical need. 

4. GC Biopharma And Novel Pharma First Patient Dosed In Global Phase I Clinical Trial For MPS IIIA Treatment 11/25/2024

   GC Biopharma and Novel Pharma today announced that the first patient in the US has been dosed in multinational clinical trial with 'GC1130A', an innovative new drug for Sanfillippo syndrome type A (MPS IIIA).

5. Seven Top Pharma Companies Adopt PhaseV's Machine Learning Technology For Drug Development 11/25/2024

   PhaseV, a pioneer in software and machine learning (ML) for advanced clinical trial optimization, announced today that seven global pharmaceutical companies are now leveraging the company's ML platforms for drug development. Additionally, PhaseV is in advanced project discussions with five additional top pharma companies.

6. Alnylam Announces U.S. Food And Drug Administration Acceptance Of Supplemental New Drug Application For Vutrisiran For The Treatment Of ATTR Amyloidosis With Cardiomyopathy 11/25/2024

   Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Company’s supplemental New Drug Application (sNDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). Based on the Company’s use of a Priority Review Voucher, the FDA has set an action date goal of March 23, 2025, under the Prescription Drug User Fee Act (PDUFA).

7. A Molecular Trap For Exotic Metals Promises Improved Diagnostics And Faster Drug Development 11/25/2024

   A group of scientists from IOCB Prague, led by Dr. Miloslav Polášek, have created compounds that are up to a million times more stable than similar substances used in contemporary medicine to treat tumours or as contrast agents for magnetic resonance imaging.

8. USC Announces Strategic Collaboration With Autobahn Labs To Accelerate Drug Discovery 11/22/2024

   USC has embarked on a collaboration with Autobahn Labs, an accelerator for early-stage drug discovery, to identify and advance cutting-edge scientific findings into new therapies – with a special focus on critical unmet medical needs.

9. Phages, Towards A Targeted Alternative To Antibiotics 11/21/2024

   With the rapid development of antibiotics in the 1930s, phage therapy – using viruses known as bacteriophages or phages to tackle bacterial infections – fell into oblivion. But as the current rise in antibiotic resistance is making it increasingly difficult to treat bacterial infections, phage therapy is once again sparking interest among physicians and scientists – although it remains complex in practice because of the great diversity and specificity of phages.

10. Amneal Resubmits DHE Autoinjector New Drug Application And Receives U.S. FDA Approval Of Exenatide, Its First Generic Injectable GLP-1 Agonist 11/21/2024

    Amneal Pharmaceuticals, Inc. (“Amneal” or the “Company”), a global pharmaceutical company, today announced the advancement of two key strategic initiatives. First, the Company has resubmitted to the U.S. Food and Drug Administration (FDA) its new drug application (NDA) for dihydroergotamine (DHE) prefilled syringe autoinjector for the acute treatment of migraine with or without aura and cluster headache in adults.