Syngene International Limited’s Post

We are delighted to announce today, on Rare Disease Day, that Coave Therapeutics has received a grant from the The ALS Association to advance its investigational CTx-TFEB program as a potential treatment for all forms of ALS. CTx-TFEB is a novel, targeted genetic medicine approach designed using Coave’s unique ALIGATER™ platform to promote autophagy and reduce the accumulation of toxic protein aggregates in neurons in the brain and spinal cord of people with ALS and that lead to nerve degeneration, paralysis, respiratory failure and ultimately death. There is no cure. With this funding and invaluable support from the ALS Association, we are poised to move our CTx-TFEB program forward. Our goal is to establish a robust preclinical proof-of-concept, laying a strong foundation for its advancement into clinic trials in the near future. Read our press release: https://lnkd.in/euTD95Vt More about the ALS Barnett Drug Development Program: https://lnkd.in/ebWw8D9G #ALS #genetherapy #autophagy #clinicaltrials

This week is exciting! Two companies announced positive interim results from their respective clinical trials in two rare kidney diseases that have an exceptionally high unmet need for new treatment options.   📣 Dimerix Limited annouced positive interim results for DMX-200 based on the proteinuria efficacy endpoint in its Phase 3 ACTION3 trial in #FSGS.   📣 Regulus Therapeutics announced positive topline data for RGLS8429 based on imaging-based biomarkers in its Phase 1b MAD trial in autosomal dominant polycystic kidney disease (#ADPKD).   Through Spherix's tracking of these markets over the years, it is evident that nephrologists are frustrated by the challenges they face in managing these diseases and welcome new therapies to help fulfill the unmet medical needs of their patients. #marketdynamics #marketresearch #nephrology

Just published in our special issue "Genetics and Pathomechanisms of Amyotrophic Lateral Sclerosis (ALS)" A comprehensive review of ALS mechanisms and related therapeutic strategies from Dr. Lien Nguyen, UFlorida. Highlighted are the 2023 FDA-approved drug Qalsody (tofersen) for SOD1 ALS, the updated C9orf72 GGGGCC repeat-expansion-related mechanisms, and therapeutic targets.

On this Word Lupus Awareness Day, Biomedcode is proud to contribute in the preclinical development of novel lupus-targeting human therapeutics with our recently published humanized IL23A driven mouse model of #SLE (https://lnkd.in/dH_S2PSG), a valuable disease model for studying the aetiopathogenic role of human IL23A in SLE and for validating the #preclinical in vivo #efficacy of novel disease-related therapeutics. Let’s all help raise global awareness of the physical, emotional and economic impact that lupus can have and bring greater attention and resources to efforts to end lupus. #makelupusvisible, #worldlupusday, #CRO, #mousediseasemodel, #IL23, #SLE, #drugtesting, #autoimmunity, #guselkumab, #tremfya, #TghIL23A, #humanized

Developing drugs for multiple disorders simultaneously (hepatic and cardio-renal) within a single trial is important. It is therefore essential to use non-invasive tests such as biomarkers or imaging as evaluation criteria to stratify patient risk on the one hand, and to assess the efficacy and safety of a treatment on the other! This week, we are very pleased to present the article "MASLD and MASH at the crossroads of hepatology trials and cardio-renal metabolic trials." which offers an overview of this type of approach from the multi-stakeholder, multi-specialty Metabolic multi Organ Science Accelerating Innovation in Clinical Trials meeting held in December 2022. Faiez Zannad, Arun J. Sanyal, Javed Butler, João Pedro Ferreira, Nicolas GIRERD, Veronica Miller, Ambarish Pandey, Chirag R. Parikh, Vlad Ratziu, Zobair M. Younossi, Stephen A. Harrison. https://lnkd.in/eRecyvS7

Here are the updates from the 3rd and last day of the Huntington's Disease Therapeutics Conference 2024 - Day 3:

Here are the updates from the 3rd and last day of the Huntington's Disease Therapeutics Conference 2024 - Day 3:

CereMark applauds the recent FDA approvals of new therapeutics for Alzheimer's Disease, notably Eli Lilly's Kisunla, an antibody drug designed to target and remove amyloid beta plaques in the brain, and Eisai's Leqembi (created in partnership with Biogen), another therapy also focused upon removing amyloid plaques from the brain. These developments offer hope for the millions of Americans who suffer from AD, although the case is that millions of others are experiencing mild cognitive impairment without dementia and uncertainty regarding the course of their future neurodegeneration.  At CereMark Pharma, our efforts continue to develop comprehensive PET imaging agents capable of assessing pathology associated with cognitive decline and monitoring therapy-driven changes in the brain burden of both amyloid and tau proteins in a single PET scan. Our commitment lies in developing PET biomarkers with the potential to predict neurodegeneration and greater precision in its pathological development. CereMark is dedicated to leading this charge and the potential of our investigational PET imaging agent, F-18 Flornaptitril, for the challenge of better care and management of neurodegenerative diseases. https://lnkd.in/epWjfi6n #BrainHealth #MedicalImaging #Neurodegeneration #CMS #PatientCare

Biomarker endpoints can be used as primary, secondary or exploratory endpoints and can be divided into those demonstrating treatment effects on pathological processes or ‘target engagement biomarkers’. Our paper summarizes excellently the possibilities and opportunities of using fluid-based biomarkers in AD clinical trials.

DeepCure will unveil preclinical data on their BRD4 Inhibitor, DC-9476, at the EMBO 2024 Conference. DC-9476 targets macrophage activation syndrome (MAS), outperforming standard treatments like glucocorticoids in animal models. The inhibitor shows promise in restoring normal white blood cell counts and reducing MAS biomarkers, offering a potential alternative for patients with MAS and Still’s disease. Kfir Schreiber, CEO of DeepCure, is optimistic about DC-9476's ability to inhibit macrophage activation and address limitations of current therapies. Learn more: https://lnkd.in/d9YM2eph