CGTLive

CGTLive™ aims to provide healthcare professionals with essential, high-quality information about cell, gene, regenerative, and engineered medicines to ensure optimal patient care. This independent digital platform offers a comprehensive resource that highlights groundbreaking news and insights directly from leading medical conferences and researchers. It is specifically designed for busy healthcare professionals who are either considering or currently managing patients receiving these advanced therapies. The news platform features detailed coverage of emerging products and procedures, addressing the growing need for expertise in an ever-evolving field. CGTLive™ connects users with the latest clinical trial results, FDA updates and approvals, transformative research findings, and expert opinions. Additionally, through engaging video content, CGTLive™ presents lively discussions with knowledgeable experts on important topics relevant to today’s healthcare practitioners. CGTLive™ provides direct access to expert knowledge aimed at enhancing the lives of patients affected by genetic disorders and specific cancers, including lymphoma, Duchenne muscular dystrophy, spinal muscular atrophy, lysosomal storage disorders, retinitis pigmentosa, GM1 and GM2 gangliosidosis, mucopolysaccharidosis, sickle cell anemia, hemophilia, and more.

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Articles

Expanding Access to Whole Genome Sequencing in Parkinson Research

3 days ago | cgtlive.com | James Beck |Noah Stansfield |Isabella Ciccone

The PD GENEration study, which is led by the Parkinson’s Foundation in collaboration with the Global Parkinson’s Genetics Program (GP2), provides whole genome sequencing (WGS) and genetic counseling for patients with Parkinson disease (PD) across more than 8 countries in the Americas and Israel. In order to support expansion internationally, the study adapted counseling materials tailored to local languages, cultural contexts, and healthcare infrastructure.
World Ovarian Cancer Day 2025: Looking Back at Progress for Cell Therapy

1 week ago | cgtlive.com | Noah Stansfield

According to the World Ovarian Cancer Coalition, at a global scale ovarian cancer is the 8th most common cancer for women and the 8th most common cause of death from cancer for women. Each year around the world, approximately 207,000 women with ovarian cancer die. Although treatment options exist, substantial unmet need remains, and many companies and academic institutions are currently pursuing new treatment approaches for the disease.
Around the Helix: Cell and Gene Therapy Company Updates - May 7, 2025

1 week ago | cgtlive.com | Noah Stansfield

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more. Have a cell and gene therapy news update you’d like to share with our editorial team?
Clinical Stage Biotech Bloomsbury Genetic Therapies Shuttering its Doors

1 week ago | cgtlive.com | Noah Stansfield

Bloomsbury Genetic Therapies, a biotech company developing several gene therapy candidates for ultra-orphan rare diseases, is in the process of shutting down its operations.1The announcement came via a LinkedIn post made by the company’s cofounder and chief executive officer, Adrien Lemoine, MSc, who noted alongside the news that he will be stepping down from his role as CEO.
Neurophth is Tackling LHON-ND4 With Gene Therapy NR082 in Phase 1/2/3 GOLD Trial

1 week ago | cgtlive.com | Noah Stansfield

Neurophth is currently evaluating NR082 (also known as Opvika and esonadogene mvoparvovec), an investigational gene therapy intended for the treatment of Leber hereditary optic neuropathy (LHON) associated with mtND4 mutations, in the phase 1/2/3 GOLD clinical trial (NCT04912843).1 For Healthy Vision Month, observed annually in May by the patient and clinician communities, CGTLive® has decided to take a closer look at this ongoing clinical trial.
CGTLive®’s Weekly Rewind - May 2, 2025

1 week ago | cgtlive.com | Noah Stansfield

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field. The agency's decision was based on data from the phase 3 VIITAL study (NCT04227106). The chief scientific officer at Precision Biosciences discussed the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy.
Data Roundup: April 2025 Features Updates for Cardiomyopathy Gene Therapy, Myasthenia Gravis CAR-T, and More

1 week ago | cgtlive.com | Noah Stansfield

May 2, 2025Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team. Last month, April 2025, the CGTLive® team was diligently tracking the latest data readouts and published literature on cell and gene therapies within oncology, neurology, rare diseases, and more.
FDA Activity Recap: April 2025 Features Major Approval in RDEB, Multiple Breakthrough Therapy Designations, and More

2 weeks ago | cgtlive.com | Noah Stansfield

Last month, April 2025, the CGTLive® team was diligently tracking the FDA's activities related to the development of cell and gene therapies for the treatment of rare, complex, and otherwise challenging diseases and disorders. The agency has continued to ramp up its activities around these therapies as more of them progress through the pipeline in tandem.
Around the Helix: Cell and Gene Therapy Company Updates - April 30, 2025

2 weeks ago | cgtlive.com | Noah Stansfield

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more. Have a cell and gene therapy news update you’d like to share with our editorial team?
The Evolving Landscape of Gene and Cell Therapies in Parkinson Disease: Pipeline Highlights

2 weeks ago | cgtlive.com | Isabella Ciccone |Marco Meglio |Noah Stansfield

A version of this article originally appeared on our sister site, NeurologyLive. Cell and gene therapies are rapidly transforming the treatment paradigm for Parkinson disease (PD), with several investigational agents targeting underlying disease mechanisms such as lysosomal dysfunction, α-synuclein pathology, and dopaminergic neuronal degeneration.