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Aging And Schizophrenia Share Striking Similarities in 2 Types of Brain Cells

AuroBlog - Aurous Healthcare Clinical Trials blog

US researchers examined gene expression in more than a million brain cells collected post-mortem from 191 donors. Those with schizophrenia and older adults were found to have a comparitively reduced expression of specific genes in both […]

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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients. “We

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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. Ultragenyx Pharmaceuticals has an orthinine transcarbamylase (OTC) activator drug in Phase III trials.

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C4XD and AstraZeneca enter $402m deal to develop respiratory disease therapy

Pharmaceutical Technology

According to the deal, C4XD is entitled to get up to $16m in pre-clinical milestone payments before the commencement of the first clinical trial. . A key natural regulator, NRF2 controls antioxidant genesexpression. This includes an upfront payment of $2m from AstraZeneca.

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Rocket Pharmaceuticals Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial of RP-A501 for the Treatment of Danon Disease

BioTech 365

Rocket Pharmaceuticals Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial of RP-A501 for the Treatment of Danon Disease Rocket Pharmaceuticals Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial … Continue reading →

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US FDA clears Transcenta’s IND for Rett syndrome treatment

Pharmaceutical Technology

Neurogene stated that the FDA IND clearance allows it to commence a Phase II/II trial of NGN-401 in female paediatric Rett syndrome patients. The multi-centre, open-label, single-arm clinical trial has been designed to evaluate the efficacy, tolerability, and safety of a single dose of NGN-401 delivered using a one-time ICV procedure.

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Aardvark Therapeutics IPO Sets Sights on Satiating Hyperphagia and Extreme Hunger

XTalks

In a Phase II trial involving patients with hyperphagia, ARD-101 was well tolerated and achieved an approximate eight-point reduction in hunger scores. Encouraged by these results, Aardvark has initiated a Phase III clinical trial, known as the HERO (Hunger Elimination or Reduction Objective) trial, with topline data expected in early 2026.