Cancer & Disease Research
Overcoming the blood-brain barrier for gene therapy delivery with AAV capsids
Current gene delivery to the central nervous system continues to be a challenge, according to Amy Pooler, PhD, vice president of neuroscience at Sangamo Therapeutics. Pooler spoke with ScienceBoard.net at the American Society of Gene & Cell Therapy 2022 annual meeting about how Sangamo’s adeno-associated virus (AAV) capsids are designed to overcome the blood-brain barrier. Read More
From China to Princeton: Yibin Kang's journey as an immigrant scientist to disrupt cancer metastasis
It's been an amazing odyssey of scientific discovery for Yibin Kang, PhD, Warner-Lambert/Parke-Davis professor of molecular biology at Princeton University. Born in a small fishing village in China, Kang from an early age had a strong interest in nature and science -- a passion that led to his becoming a cancer biologist in the U.S. Read More
Decibel Therapeutics develops treatments to restore and improve hearing, balance
Decibel Therapeutics is helping to break down the barriers to understanding the molecular pathways and cell physiology inside the inner ear by integrating single-cell genomics, bioinformatic analyses, and precision gene therapy technologies, contends CEO Laurence Reid, PhD. Reid spoke with ScienceBoard.net at the American Society of Gene & Cell Therapy 2022 annual meeting in Washington, DC. Read More
Mustang Bio focuses on CD20 antigen for CAR T-cell therapy
Biopharmaceutical company Mustang Bio is focused on a CD20-targeted autologous chimeric antigen receptor (CAR) T-cell therapy for non-Hodgkin lymphoma, chronic lymphocytic leukemia, and Waldenstrom macroglobulinemia. Dr. Bruce Dezube, senior vice president at Mustang Bio, spoke to ScienceBoard.net at the American Society of Gene & Cell Therapy 2022 annual meeting about the targeting of the CD20 antigen. Read More
Graphite Bio sees its gene editing tech as step above CRISPR
While the use of CRISPR is an effective gene editing tool on its own, Dr. Josh Lehrer, CEO of Graphite Bio, contends that the technology doesn’t correct the underlying genetic defect. CRISPR is the “cut” function and Graphite Bio’s technology is the “cut and paste” capability, Lehrer told ScienceBoard.net at the American Society of Gene & Cell Therapy 2022 annual meeting in Washington, DC. Read More
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
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