Lexeo to start phase I/II clinical trial for patients with FA cardiomyopathy
Gene therapy company Lexeo Therapeutics in 2022 plans to launch a phase I/II clinical trial of its adeno-associated virus-based therapy designed to intravenously deliver a functional frataxin gene for the treatment of Friedreich’s ataxia (FA) cardiomyopathy. At the American Society of Gene & Cell Therapy 2022 annual meeting, Lexeo presented new preclinical data supporting its FA cardiomyopathy program. Read More
COVID-19 mRNA-based vaccines benefit from decades of research
25 years ago Dr. Drew Weissman, PhD, an infectious disease expert at Penn Medicine, and RNA biologist Kati Kariko, PhD, began their collaboration in what would become a critical technology used in some of today’s COVID-19 mRNA-based vaccines. Weissman spoke with ScienceBoard.net at the American Society of Gene & Cell Therapy 2022 annual meeting in Washington, DC about their research journey. Read More
Next-generation AAV capsids hold key to developing life-changing therapies
Voyager Therapeutics’ discovery platform is identifying novel capsids targeting desired cells and tissues with greater specificity, at lower doses, and with fewer off-target risks than conventional adeno-associated virus (AAV) serotypes in non-human primates, according to Todd Carter, PhD, senior vice president for research. The company presented data on the platform at the American Society of Gene & Cell Therapy 2022 annual meeting. Read More
Genenta Science reports preliminary results of Temferon-glioblastoma study
Biotech company Genenta Science is developing a proprietary hematopoietic stem cell gene therapy, Temferon, for the treatment of a variety of solid tumor cancers, according to co-founder Dr. Bernhard Gentner, PhD. ScienceBoard.net spoke to Gentner at the American Society of Gene & Cell Therapy 2022 annual meeting in Washington, DC, about Temferon. Read More
Atsena Therapeutics’ gene therapy takes aim at childhood blindness
In an interview with ScienceBoard.net, Patrick Ritschel, CEO of Ophthalmology-based gene therapy company Atsena Therapeutics, shared an overview of the company's development of novel treatments for inherited forms of blindness at the American Society of Gene & Cell Therapy 2022 annual meeting in Washington, DC. Read More
How to address challenges in producing AAV vectors
There are challenges in producing sufficient quantities of gene therapy products, particularly when it comes to adeno-associated virus (AAV) vectors, contends Ines do Carmo Gil-Goncalves, PhD, head of vector production for Cevec Pharmaceuticals GmbH. Gil-Goncalves discussed how Cevec’s Elevecta platform can help overcome these challenges at the American Society of Gene & Cell Therapy 2022 annual meeting. Read More
Omega Therapeutics looks to tap 'nature's operating system' to control gene expression
Precision genomic medicine company Omega Therapeutics is looking to harness “nature’s operating system” for controlling gene expression and cell differentiation, according to Thomas McCauley, PhD, chief scientific officer. McCauley spoke with ScienceBoard.net at the American Society of Gene & Cell Therapy annual meeting in Washington, DC. Read More
Delivery of gene therapies remains a key challenge
Gene therapy holds great promise for treating inherited disorders, cancers, and other diseases, but delivery of these therapies remains a key challenge, contends Kevin Foust, PhD, senior vice president for preclinical and translational medicine at Jaguar Gene Therapy. Foust spoke with ScienceBoard.net at the American Society of Gene & Cell Therapy annual meeting in Washington, DC. Read More
Next evolution of CAR T-cell therapy is solid tumors
While chimeric antigen receptor (CAR) T-cell therapy has become a game changer for some patients with blood cancer, the next area of evolution for the technology is solid tumors, said Sangamo CEO Sandy Macrae, PhD, at the American Society of Gene & Cell Therapy 2022 annual meeting in Washington, DC. Read More
Sample management, characterization is a growing challenge in CGT
From academic research centers to small biotechs to large pharmaceutical companies, the numbers of biosamples and measurements are growing exponentially in the field of cell and gene therapy (CGT). Andrea O'Hara and David Lewandowski of Azenta Life Sciences, spoke with ScienceBoard.net at the International Society for Cell & Gene Therapy (ISCT) 2022 annual meeting about the challenge of sample management and characterization. Read More
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