Drugs Gene Editing Genetic Disease 
Camargo
JULY 27, 2021
When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.
pharmaphorum
SEPTEMBER 28, 2022
Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The post Vertex, CRISPR prep filing for gene-editing blood disorder therapy appeared first on.
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pharmaphorum
OCTOBER 4, 2022
AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.
STAT News
SEPTEMBER 16, 2022
Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed inside the body — found the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively.
pharmaphorum
DECEMBER 6, 2020
A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.
pharmaphorum
FEBRUARY 17, 2022
Global Blood Therapeutics’ run of positive regulatory news for its oral sickle cell disease treatment Oxbryta has continued with an approval for the drug in Europe for patients aged over 12. The post GBT claims EU approval for sickle cell drug Oxbryta appeared first on.
pharmaphorum
JANUARY 10, 2022
Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. There is another $1 billion in downstream payments on offer, assuming the drug candidates all make it through to regulatory approval and hit sales targets.
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