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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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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. . on meeting clinical development and commercial milestones in the future, in addition to tiered royalty payments, upon the marketing of the product.

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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 uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

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Symvivo’s Oral COVID-19 Vaccine Enters Clinical Trials

XTalks

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. COVID-19 Clinical Trials.

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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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Data dive finds cheap diuretic could be Alzheimer’s drug

pharmaphorum

The research has focused on Alzheimer’s associated with an apolipoprotein E4 (APOE4) gene variant known to be a major risk factor for late-onset forms of the disease. All told, five drugs emerged with a gene expression signature that the researchers believed might help neutralise the disease, but the strongest candidate was bumetanide.