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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

A personal journey to Alltrna Michelle Werner’s career has spanned over 20 years in the pharmaceutical industry, where she developed her expertise in oncology drug development at leading companies such as Bristol Myers Squibb, AstraZeneca, and Novartis. Alltrna focuses on a mutation-driven approach.

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First Patient Dosed in Phase 3 REVEAL Trial of ION582 for Angelman Syndrome

The Pharma Data

Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. This mechanism has the potential to partially restore UBE3A protein production in neurons, addressing the root cause of the disorder. Source link

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Target-directed cancer: protein-ligand interactions  

Drug Target Review

We support the drug discovery projects of the CCDD with assay development and screening, biophysics, structural biology and recombinant protein production. With all these diverse methods we characterise protein-ligand interactions in many ways.

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Series A Financing of 570 Million Yen Procured by Epsilon Molecular Engineering: A Next-Generation Medium Sized Molecular Biotechnology-Based Drug Discovery Firm

The Pharma Data

.] “EME aims to discover medium sized molecular bio-drugs with new modalities using its proprietary VHH technology. We will leverage this financing to accelerate collaborative research with pharmaceutical manufacturers and internal research using its own pipeline. We support companies that lead regional vitalization.

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Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Agency IQ

For example, in describing how potency should be demonstrated for CGT products, the 2011 guidance simply stated that potency for gene therapy products is derived from “at least two biological activities for its potency: the ability to transfer a genetic sequence to a cell; and the biological effect of the expressed genetic sequence.”

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Driving efficiency across upstream bioprocess workflow

Drug Target Review

Transposon technologies are gaining traction due to their accuracy in integrating the gene of interest into the host genome, making them an important tool for accelerating large molecule drug development. Anis works within the strategy group in Life Sciences at Revvity, Inc. Budge JD, Knight TJ, Povey J, et al. Biotechnol Prog.

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JS Bio and Etta Biotech Advancing Strategic Partnership

The Pharma Data

Etta Biotech”), to set up a high titer transient protein expression platform for high quality protein production using JS Bio’s transient transfection media. JS Bio becomes the exclusive cell culture supplier for Etta Biotech’s transient transfection high titer protein expression platform.