$NTLA and $REGN report initial data from ph1 study of #ATTR for NTLA-2001. All patients across the two doses showed 92-94% TTR reduction at day 28.
#crispr #genomics #genetherapy #biology #biotech
At both dose levels, $NTLA-2001 was generally well tolerated. 2 patients reported transient infusion reactions, which was the only observed treatment-related adverse event.
$NTLA-2001 is an in-vivo administered #genetherapy.
It is one of the first few in-vivo #GeneTherapy being tested. Congrats to the $NTLA and $REGN teams.

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More from @BioCompounding

Sep 17
An Israeli biotech company, Renewal Bio that recently created mouse embryos using stem cells, has announced plans to make human embryos to harvest tissue for organ transplants and anti-aging procedures.

#Stemcells #biotech #biology #Syntheticbiology #Aging #transplant
The scientist behind the work clarified that:
The synthetic embryos were not "real" embryos and did not have the potential to develop into live animals. But that is partly because they don't have the technology, right now, to do so.
He admits to taking his "ground-breaking" technology to the next level and creating human embryos. The purpose is to harvest tissue to be used in transplant treatments meant to lengthen a person's life and health.
Read 5 tweets
Sep 13
The US Govt just announced and EO for advancing #biotechnology and #biomanufacturing for a #Sustainable , safe and secure #bioeconomy.
There are 11 areas they will be looking to fund and develop within this niche.
Read the full article here - whitehouse.gov/briefing-room/…
Here is a quick summary:
1) Coordinate investment in key R&D areas to further societal goals
2)Foster a biological data ecosystem, while adhering to principles of security, privacy, and responsible conduct of research
3) Expand domestic #biomanufacturing production capacity and processes, including #piloting and #prototyping efforts to accelerate the translation of basic research results into practice
4) Boost #sustainable #biomass production
Read 7 tweets

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