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Start-up to study peptides that modulate gene expression

Drug Discovery World

HBC Immunology (a subsidiary of Hofseth BioCare) has secured seed capital financing of approximately $900,000 from external investors to commence preclinical animal trials with its patented peptides, FT-002 and FT-005. HBCI’s CEO, Dr Bomi Framroze said: “HBCI is at the forefront of research in using peptides to modulate gene expression.

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Potential first-in-class therapeutic for urea cycle disorders enters trial

Drug Discovery World

Mutations in genes encoding urea cycle enzymes result in insufficient levels of these important proteins. CMP-CPS-001 is an antisense oligonucleotide (ASO) designed to amplify CPS1 mRNA by harnessing fundamental cellular gene expression control mechanisms.

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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. In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs).

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

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). 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.

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Data dive finds cheap diuretic could be Alzheimer’s drug

pharmaphorum

A data-mining study conducted by researchers in the US has found that an already-approved diuretic drug could have potential as a treatment for some patients with Alzheimer’s disease. The post Data dive finds cheap diuretic could be Alzheimer’s drug appeared first on.

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Astellas licenses companion drug for Pompe gene therapy

pharmaphorum

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease. The post Astellas licenses companion drug for Pompe gene therapy appeared first on.

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World Mental Health Day 2023: Drug discovery breakthroughs

Drug Discovery World

Over recent months, there have been a number of ground-breaking discoveries in drug discovery for mental health conditions. The article in the journal Psychopharmacology focuses on new data showing that ACD856, the lead clinical drug candidate in the NeuroRestore platform, exhibits antidepressant effects in various preclinical models.

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