Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
Vironexis Biotherapeutics Inc. has come out of stealth mode, disclosing that it has more than 10 product candidates it’s been developing over the last three years. The therapies are built on the company’s AAV-based platform, Transjoin, which is designed to have patients' livers express bispecific antibodies that bind to both CD3 on T-cells and various targets on tumor cells.
Vironexis Biotherapeutics Inc. came out of stealth mode today, disclosing that it has more than 10 product candidates it’s been developing over the last three years. The therapies are built on the company’s AAV-based platform, Transjoin, which is designed to have patients' livers express bispecific antibodies that bind to both CD3 on T-cells and various targets on tumor cells.
Philadelphia-based Latus Bio Inc., co-founded by serial biotech entrepreneurs P. Peter Ghoroghchian and Beverly Davidson, launched on May 2 with two lead adeno-associated virus (AAV)-based gene therapy candidates and $54 million in a series A financing.
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.
Gene therapy developer Kate Therapeutics Inc. (KateTx), which is developing next-generation adeno-associated virus (AAV) vectors that target skeletal and cardiac muscle, has unveiled $51 million series A round and a licensing deal with Astellas Pharma Inc.
AAVantgarde Bio SrL raised €61 million (US$65 million) in series A funding to take forward two novel approaches to gene therapy that aim to overcome the packaging limits of adeno-associated virus (AAV) vectors. The company plans to move its lead program, in retinitis pigmentosa associated with Usher syndrome type 1b, into the clinic later this year. A second program, in Stargardt disease, is a couple of years behind it.
One of the challenges in designing genetic and cellular strategies is getting the therapy to the right place. This is even more complicated when it comes to the nervous system. The brain is a complex organ that contains the most differentiated and inaccessible cells in human biology. It is an impassable safe, protected by the blood-brain barrier.
When it comes to CAR T therapies, South Korea is trailing behind the U.S. and China, but the South Korean government sees cell and gene therapies as a space where the country can draw international investors, speakers said during the Bio Korea 2023 conference in Seoul on May 10.
Researchers based at Uppsala University in Sweden have created an adeno-associated viral (AAV) vector treatment for glioma or glioblastoma that extended survival in a mouse model of the brain cancer. Writing in the May 11, 2023, issue of Cancer Cell, the researchers reported that by introducing an inflammatory protein known as LIGHT to the tumor microenvironment using the AAV treatment, the mice were better able to fight the cancer.
