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Informed Design of Bioanalytical PCR Assay Testing Parameters

PPD

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type.

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Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Alta Sciences

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

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Biopharma Money on the Move: December 2 – 8

The Pharma Data

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. CEO and founder Min Li knows a thing or two from his time running GSK’s neuro R&D group before the company shuttered the program in 2017.

RNA 52
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2021 AAN highlight impact and breadth of expanding neuroscience portfolio

The Pharma Data

FDA and global approvals for our groundbreaking therapies in SMA and NMOSD, Roche’s data at AAN reflect our continued commitment to meaningful therapeutic progress for people living with neurological disorders,” said Levi Garraway, M.D., Following U.S. Roche’s Chief Medical Officer and Head of Global Product Development. Claims Database.

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Roche to present new data in multiple sclerosis and neuromyelitis optica spectrum disorder at MSVirtual2020

The Pharma Data

From the success of our first-in-class B-cell MS therapy OCREVUS, we are poised to continue advancing the science in MS with our new investigational BTK inhibitor fenebrutinib, and in NMOSD with the recent FDA approval of ENSPRYNG.”. Roche’s Chief Medical Officer and Head of Global Product Development.

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GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome

The Pharma Data

In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. About Ultragenyx.

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Driving efficiency across upstream bioprocess workflow

Drug Target Review

As discussed above, recent advances in gene editing tools and transposon-based vectors have facilitated targeted and consistent cell line engineering for the development of protein expressing CHO cell lines. 2017 Nov;33(6):1468-1475. Epub 2017 Sep 7. Cell Gene Therapy Insights 2017; 3(2), 131-158, 10.18609/cgti.2017.014.