23:35 , Aug 22, 2018 |  BC Innovations  |  Translation in Brief

Confirming CRISPR

Two critiques of a 2017 human embryo gene editing study, including one suggesting large deletions at the target site misled researchers into thinking CRISPR-Cas9 corrected a genetic mutation, raise questions about what assays and screening...
21:30 , Jul 24, 2018 |  BC Innovations  |  Translation in Brief

Interaction analysis

In a collaboration with Merck & Co. Inc. (NYSE:MRK) and SomaLogic Inc., University of Cambridge researchers have identified associations and causal relationships between thousands of genetic variants, plasma proteins and disease states, which could reveal...
17:50 , Jul 20, 2018 |  BC Week In Review  |  Company News

PTC acquiring CNS gene therapy play Agilis

PTC Therapeutics Inc. (NASDAQ:PTCT) will acquire gene therapy company Agilis Biotherapeutics Inc. (Cambridge, Mass.), giving the rare disease company four products in development for CNS disorders. PTC will pay Agilis shareholders about $200 million up front,...
22:43 , Jul 19, 2018 |  BC Extra  |  Company News

PTC acquiring CNS gene therapy play Agilis

PTC Therapeutics Inc. (NASDAQ:PTCT) will acquire gene therapy company Agilis Biotherapeutics Inc. (Cambridge, Mass.), giving the rare disease company four products in development for CNS disorders. PTC will pay Agilis shareholders about $200 million up...
15:41 , Jul 18, 2018 |  BC Innovations  |  Distillery Techniques

Biomarkers

TECHNOLOGY: Gene profiling Mutations in hematopoiesis-associated genes could help predict the risk of AML. Genomic sequencing of 124 patients who later developed AML and 686 healthy volunteers identified associations between the disease and mutations in 22...
21:40 , Jul 10, 2018 |  BC Extra  |  Preclinical News

Models could help predict AML risk

In a Nature paper, a team led by researchers at University Health Network and Wellcome Sanger Institute developed two models to predict healthy subjects’ likelihood for developing acute myelogenous leukemia that could provide the basis...
00:43 , Jun 29, 2018 |  BC Innovations  |  Product R&D

Highly personal

Pact Pharma Inc. has raised $120 million in venture financing to tackle one of the most complex forms of personalized medicine proposed to date: neoantigen-targeted T cells. Backed by Alphabet Inc.’s GV, the company is...
21:36 , May 30, 2018 |  BC Extra  |  Preclinical News

Tet2 disruption improves Kymriah efficacy

In a Nature study, researchers at the University of Pennsylvania identified a patient with an enhanced response to CAR T therapy Kymriah tisagenlecleucel, and suggested targeting the epigenome by inhibiting Tet2 could improve the efficacy...
18:54 , May 10, 2018 |  BC Innovations  |  Strategy

Finding phenotype

NIH wants to find direct relationships between genes and disease more efficiently than GWAS or other standard approaches allow. The institute has teamed up with Inova Health System to create a searchable pool of genomes...
18:27 , Apr 5, 2018 |  BC Innovations  |  Emerging Company Profile

Stoking protein production

Stoke Therapeutics Inc. is developing ASOs to boost normal protein expression as disease-modifying therapies for autosomal-dominant diseases that are not amenable to recombinant protein or gene therapies. Co-founders Adrian Krainer and Isabel Aznarez previously discovered cell...