Chengdu Origen and Vanotech Report the First Patient Dosing in P-I (VAN-2401) Trial of KH658 for Wet Age-related Macular Degeneration

Shots: Chengdu Origen & Vanotech has reported the first pts dosing in Vanotech-led P-I (VAN-2401) trial assessing KH658 for wet age-related macular degeneration (wet AMD) in the US The P-I trial will assess safety, tolerability, & efficacy of single suprachoroidal KH658 gene therapy in ~9 previously treated wet AMD pts that are responsive to anti-VEGF therapy…

ByRidhi Rastogi1 day ago

News

Abeona Therapeutics’ Zevaskyn Receives the US FDA Approval for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Shots: The US FDA has approved Zevaskyn (prademagene zamikeracel) for treating wounds in adult & pediatric RDEB pts based on the intra-pts controlled P-III (VIITAL) trial & has also granted RPD PRV to Abeona, which the company intends to monetize; Zevaskyn to be commercially available in Q3’25 The P-III Trial assessed Zevaskyn in RDEB pts,…

ByRidhi RastogiApril 30, 2025

News

SineuGene Therapeutics Reports the US FDA’s IND Clearance of SNUG01 for Amyotrophic Lateral Sclerosis

Shots: The US FDA has granted IND clearance to SNUG01 for ALS SNUG01 will be evaluated in a P-I/IIa dose-escalation & expansion trial to assess its safety, tolerability, & preliminary efficacy in ALS adults; SineuGene to partner with academics & institutions to lead clinical development of SNUG01 SNUG01, a TRIM72-targeted gene therapy, counteracts ALS through…

ByRidhi RastogiApril 3, 2025

THOUGHTSPOT

7 Cutting-Edge Technologies Revolutionizing Personalized Eye Care

Vision is one of our most precious senses, shaping how we experience the world around us. Yet, for millions, achieving clear sight is not as simple as slipping on a pair of glasses. Complex conditions like astigmatism, glaucoma, and retinal disorders often require personalized solutions—solutions that, until recently, were limited by technology. Today, a wave of…

ByAndrea LaceyJanuary 29, 2025

News

JCR Pharmaceuticals and Modalis Progress into the Next Phase of Joint Research Agreement to Develop Gene Therapy

Shots: JCR Pharmaceuticals & Modalis have reported successful validation of initial PoC to develop gene therapy targeting CNS disease and will advance to the next phase under a new joint agreement The new agreement aims at conducting preclinical studies for gene therapy using JCR's BBB-crossing J-Brain Cargo tech and for gene therapy payload using Modalis' CRISPR-GNDM…

BySenior EditorJanuary 6, 2025

VIEWPOINTS

Dr. Samiah Al-Zaidy, VP of Clinical Development at Passage Bio Shares Insights on Additional Interim Data from the Imagine-1 Study for GM1 Gangliosidosis

Shots: Samiah Al-Zaidy spoke about the interim clinical data from Imagine-1, P-I/II trial of PBGM01 for GM1 gangliosidosis presented at the 19th Annual WORLDSymposium 2023 She also talked about how the presentation at WORLDSymposium contributed to raising awareness about GM1 Gangliosidosis which is a rare and fatal disease The interview showcases Passage Bio’s mission to…

BySenior EditorMay 22, 2023

THOUGHTSPOT

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

Shots: With the healthcare sector moving ahead with its technological advances, there is still an unexplored section in therapeutics that requires much more attention. Through innovations, life science companies have evolved to make breakthrough discoveries in many focus areas, but when it comes to the rare disease segment, all our endeavors seem to be bleak…

BySaurabh ChaubeyMarch 14, 2023

EVENTS

Pharma Tech Conclave 2023

Chitkara University and DeepSynergy presents the Pharma Tech Conclave 2023. The conference will be held on 3rd Mar 2023 from 9:00 am to 5:00 pm IST at the Chitkara College of Pharmacy, Chitkara University, Chandigarh. The global pharmaceutical industry is undergoing transformational changes due to advancements in technological innovations that aim to fundamentally alter how patients…

BySenior EditorFebruary 22, 2023

VIEWPOINTS

Matt Killeen, CSO at Renovacor Shares Insights on Pipeline Expansion through its AAV Gene Therapy Program

Shots: Matt spoke about the new research program for multiple genetic segments of arrhythmogenic cardiomyopathy He also highlighted Renovacor’s research collaboration with the University of Utah’s Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) The interview shows Renovacor’s pipeline expansion to develop transformative therapies in genetically-driven cardiovascular diseases Smriti: Please give readers a brief insight…

BySenior EditorSeptember 12, 2022

THOUGHTSPOT

How Pharma 4.0 Influences Cell and Gene Therapy

Pharma 4.0 foresees a manufacturing environment that is mostly automated and barely depends on human labor. Production systems would be interconnected with each other, as well as with supply chain management systems and quality assessment systems, all orchestrated by machine-learning-driven AI control systems. As such, smart factories promise higher productivity, better quality, and enhanced agility.…

BySenior EditorMarch 7, 2022

Abeona Therapeutics’ Zevaskyn Receives the US FDA Approval for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

SineuGene Therapeutics Reports the US FDA’s IND Clearance of SNUG01 for Amyotrophic Lateral Sclerosis

7 Cutting-Edge Technologies Revolutionizing Personalized Eye Care

JCR Pharmaceuticals and Modalis Progress into the Next Phase of Joint Research Agreement to Develop Gene Therapy

Dr. Samiah Al-Zaidy, VP of Clinical Development at Passage Bio Shares Insights on Additional Interim Data from the Imagine-1 Study for GM1 Gangliosidosis

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

Pharma Tech Conclave 2023

Matt Killeen, CSO at Renovacor Shares Insights on Pipeline Expansion through its AAV Gene Therapy Program

How Pharma 4.0 Influences Cell and Gene Therapy

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