An Italian group of researchers has used zinc finger editing to silence the PCSK9 gene and improve blood cholesterol levels in mice by applying a single dose of their modifier. The epigenetic-based method could be an alternative to genome editing.
Newly approved gene therapies targeting sickle cell disease will be the first focus of the U.S. Centers for Medicare & Medicaid Services’ (CMS) Cell and Gene Therapy Access Model, the agency said Jan. 30.
Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease. But science does not stop.
Investigators at the Chinese Academy of Sciences have generated a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells. The animal survived for only 10 days, and it is not the first live birth of a chimeric primate. But it is the first such chimera with contributions from an embryonic stem cell, and that stem cell contributed a far higher proportion of cells in the newborn than have been achieved in previous attempts at creating chimeras.
At the 30th Annual Congress of the European Society for Gene and Cell Therapy in Brussels this week, researchers presented both preclinical and clinical strategies for applying gene therapy to a functional HIV cure. At a Wednesday session on Infectious Diseases & Vaccines, Alessio Nahmad, of Tabby Therapeutics Ltd., described using B cells edited to express broadly neutralizing antibodies (bnAbs) 3BNC117 to deliver high titers of antibodies in mice.
A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.
The first gene therapy to treat severe hemophilia A was among the drugs recommended for European approval by regulators from the EMA’s CHMP at its monthly meeting. Manufactured by Biomarin Pharmaceutical Inc., Roctavian (valoctocogene roxaparvovec) was recommended for conditional marketing authorization in the EU for severe hemophilia A in adults who do not have factor VIII inhibitors and no antibodies to adeno-associated virus serotype 5.
Biotechs that tackle the effects of aging are beginning to make headlines: in January Altos Labs Inc. launched with a reported investment from Jeff Bezos. With Bezos getting involved with San Francisco-based Altos, the immediate reaction was that anti-aging biotechs would be there for the benefit of billionaires searching for eternal life.
Not so, according to London U.K.-based Genflow Biosciences plc, which hopes to show that fighting aging is really about improving health as people age.
Attempts to modernize the conceptual framework of brain function and dysfunction are one prerequisite for brain disorders to benefit from precision medicine. For the circuit-based insights that are slowly emerging to benefit patients, though, better targeting methods are needed.
In one of the biggest collaboration deals of the year, Shape Therapeutics Inc. entered a collaboration and license agreement with Roche Holding AG to develop gene therapies for targets in areas that include Alzheimer’s disease, Parkinson’s disease and rare diseases. Seattle-based Shape is eligible to receive an initial payment, development, regulatory and sales milestone payments that could exceed $3 billion in aggregate value.