Shots:
Remedium Bio has entered into a multi-target research & development collaboration with Eli Lilly to develop gene therapies targeting multiple targets for the treatment of Obesity & Type 2 Diabetes, leveraging Remedium’s Prometheus platform
As per the deal, Remedium will receive an upfront, equity investment as well as development & commercial milestone payments, with tiered…
Shots:
In this episode of the CXO Talks podcast, PharmaShots speaks with Rick Modi, CEO at Affinia Therapeutics
Driven by cutting-edge innovation, Affinia is pioneering and re-imagining adeno-associated virus serotype 9 (AAV9), a gene therapy delivery method for conditions affecting cardiac muscle, skeletal muscle, and the central nervous system
Rick articulates the diagnostic and therapeutic…
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…
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…
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…
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,…
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…
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…
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…
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…

