Clinical Development Gene Genetic Disease Protein 
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.
Pharmaceutical Technology
AUGUST 17, 2022
oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . Merck will also make royalty payments on any approved products developed out of the partnership.
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Drug Discovery World
AUGUST 28, 2023
DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials. It is the method for removing unwanted debris, e.g., old proteins, and breaking them down so their constituent parts can be used again. In fact, some variants of Parkinson’s and ALS are genetic in origin.
The Pharma Data
OCTOBER 27, 2020
The Massachusetts-based biotech is investing in their proprietary “Gene Traffic Control” system, which tells the cells when to express which genes where and in what order within the chromatin regulatory system. AavantiBio has a unique opportunity to change the lives of those living with FA and other rare diseases,” Cumbo said.
The Pharma Data
NOVEMBER 8, 2020
The submission includes data from the ENLIGHTEN clinical development program in schizophrenia, and pharmacokinetic (PK) bridging data comparing ALKS 3831 and Zyprexa (olanzapine), to support an indication for schizophrenia. Progeroid Laminopathies are similar, caused by a number of mutations in the lamin A and/or Zmpste24 genes.
Drug Discovery World
FEBRUARY 28, 2023
Interestingly, at least 80% of all rare diseases originate from monogenic mutations, meaning that they arise from mutations in a single gene 4. Recent progress on mRNA therapeutics – with at least five programmes in clinical studies – is encouraging, and I hope that we will only see more scientific advancement in the years to come.
Drug Discovery World
MARCH 6, 2024
Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.
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