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NEWS

Arrowhead Enters a ~$2.2B Deal with Novartis for ARO-SNCA and TRiM-Based Therapies to Treat Neurodegenerative Diseases

Shots: Arrowhead has granted Novartis exclusive global rights to develop, manufacture, & commercialize ARO-SNCA, which leverages the TRiM platform for SC dosing & targeted gene delivery to treat synucleinopathies, incl. Parkinson’s disease As per the deal, Arrowhead will receive $200M upfront & ~$2B in development, regulatory, and sales milestones, with tiered royalties on sales up…
September 3, 2025

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NEWS

Nuevocor Reports the US FDA’s IND Clearance of NVC-001 for LMNA DCM

Shots: The US FDA has approved the IND application of NVC-001 to treat LMNA-related dilated cardiomyopathy (LMNA DCM), enabling a P-I/II trial to start in early 2026 Preclinical studies showed that NVC-001 significantly improved survival and cardiac function. They planned a P-I/II trial, a 52-week, open-label, multicenter ascending-dose study to assess the safety, tolerability, and…
June 11, 2025

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Vanotech
NEWS

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…
May 5, 2025

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Abeona Therapeutics
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,…
April 30, 2025

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SineuGene
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…
April 3, 2025

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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…
January 6, 2025

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VIEWPOINTS_Dr. Samiah Al-Zaidy_2023
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…
May 22, 2023

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Pharma Tech Conclave 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…
February 22, 2023

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