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

Pharmaceutical Technology

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. David Bumcrot PhD, CSO, CAMP4 Therapeutics.

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US FDA clears Transcenta’s IND for Rett syndrome treatment

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted clearance for Neurogene’s investigational new drug (IND) application for NGN-401 to treat Rett syndrome. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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bluebird seeks gene therapy trial restart after cancer scare

pharmaphorum

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. Shares in bluebird ticked up following the announcement.

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Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

Delveinsight

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). The drug is a small molecule, which selectively activates GABAA receptors located on the surface of nerve cells and away from the synapse. OV101 is considered as the first compound that targets disrupted tonic inhibition.

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Decibel Therapeutics Announces $82M Series D Financing to Advance Gene Therapies to Restore Hearing and Balance

The Pharma Data

DB-OTO, Decibel’s investigational gene therapy to restore hearing in children with congenital deafness due to a deficiency in the otoferlin gene, is in preclinical studies, and Decibel expects to initiate clinical testing in 2022. Thompson, M.D., a partner at OrbiMed, to its board of directors.

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Artificial intelligence could be new blueprint for precision drug discovery

The Pharma Data

online issue of Nature Communications, researchers at University of California San Diego School of Medicine describe a new approach that uses machine learning to hunt for disease targets and then predicts whether a drug is likely to receive FDA approval. the success rates in drug discovery?are

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World Glaucoma Week 2024: New Advances in Glaucoma Care

XTalks

Therapeutic Developments Related to Glaucoma The introduction of new classes of glaucoma medications, such as prostaglandin analogs, nitric oxide-donating drugs and Rho kinase inhibitors, has added to the pharmacological options available for managing IOP.