CGTLive
Online/Digital
The Mission of CGTLive™ is to deliver highly relevant, quality information about cell, gene, regenerative, and engineered medicines to healthcare professionals in order to facilitate the best patient care possible. CGTLive™ offers an independent, comprehensive digital platform that covers practice-changing news and insight directly from top medical conferences and researchers. The platform is intended for busy healthcare professionals who are considering or currently treating patients with The Mission of CGTLive™ is to deliver highly relevant, quality information about cell, gene, regenerative, and engineered medicines.
The news platform offers an in-depth look at the multitude of new products and procedures in development with ever-expanding targets and mechanisms, to help provide clarity during an unprecedented time of growing demand for expertise. The CGTLive™ platform connects visitors with the most up-to-date clinical trial results, FDA updates and approvals, practice-changing research, expert insight, and more. Additionally, through a unique mix of video editorial, CGTLive™ offers dynamic discussions with authoritative insights, opinions, and perspectives on critical issues facing today's healthcare professionals.
CGTLive™ delivers direct access to expert insight to improve the lives of patients with genetic disorders and targetable cancers, such as lymphoma, Duchenne muscular dystrophy, spinal muscular atrophy, lysosomal storage disorders, retinitis pigmentosa, GM1 and GM2 gangliosidosis, mucopolysaccharidosis, sickle cell anemia, hemophilia, and much more. Source
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Media Outlet details
| Scope | National, Consumer |
|---|---|
| Language | English |
| Country | United States of America |
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Recent Articles
Search ArticlesAspen Neuroscience Completes ASPIRO Cohort 3 and 4 Dosing, Advancing Sasineprocel Toward Phase 3
Aspen Neuroscience announced the completion of dosing in Cohorts 3 and 4 of its ASPIRO phase 1/2a trial of sasineprocel (ANPD001), bringing the total number of patients dosed in the program to 15 as of June 30, 2026.¹ The milestone represents one of the largest reported clinical experiences for an autologous cell therapy in Parkinson's disease (PD) and advances the program's commercial-ready cryopreserved formulation into broader patient testing ahead of a planned phase 3 transition.
First Patient Dosed in Phase 2b/3 NAAVIGATE Trial of Gene Therapy Surabgene Lomparvovec in Diabetic Retinopathy
REGENXBIO Inc. announced the first patient has been dosed in the phase 2b/3 NAAVIGATE clinical trial of investigational surabgene lomparvovec (sura-vec; ABBV-RGX-314), a one-time gene therapy for diabetic retinopathy (DR), triggering a $100 million milestone payment from AbbVie under the companies' existing collaboration.¹ The dosing marks the transition of the sura-vec program into pivotal-stage development for DR following encouraging two-year data from the earlier phase 2 ALTITUDE trial.
FDA Approves Tregzi for Matched Donor HSCT in Hematologic Malignancies
The FDA approved allogeneic regulatory T cell-based immunotherapy with hematopoietic stem and progenitor cell (HSPC) and T cells-vldq (Tregzi; Orca Bio) on June 30, 2026, for use in matched donor hematopoietic stem cell transplantation (HSCT) with a myeloablative preparative regimen, for hematopoietic and immunologic reconstitution, and to improve chronic graft-versus-host disease (cGVHD)-free survival in adults with hematological malignancies.1 Tregzi received regenerative medicine advanced...
Around the Helix: Cell and Gene Therapy Company Updates – May 27, 2026
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.
Guess the Therapy Answer May 22, 2026
Answer: ETX101 See below for further reading on ETX101: ETX101 Gene Therapy Shows Early Developmental, Seizure Benefits in Dravet Syndrome May 13, 2026 — Interim findings from the ongoing POLARIS phase 1/2 program suggest that Encoded Therapeutics’ investigational gene therapy ETX101 may produce substantial seizure reductions and early neurodevelopmental improvements in young children with SCN1A-positive Dravet syndrome, supporting continued advancement of the program as it enters...
Evaluating Miv-Cel for Stiff Person Syndrome
This interview was originally published on our sister site, NeurologyLive®. Stiff person syndrome (SPS) is a rare, chronic autoimmune neurological condition characterized by progressive rigidity of the axial musculature and limbs, along with episodic, stimulus-triggered muscle spasms that can profoundly compromise a patient's ability to walk and maintain quality of life.
Around the Helix: Cell and Gene Therapy Company Updates – May 20, 2026
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.
Phase 3 HOPE-3 Data Reinforce Skeletal and Cardiac Benefits of Deramiocel in Duchenne Muscular Dystrophy
New phase 3 findings presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting further strengthened the case for deramiocel, an investigational allogeneic cell therapy from Capricor Therapeutics, as a potential treatment for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy and progressive skeletal muscle decline.1 The data come at a pivotal regulatory moment for the therapy, with the FDA currently reviewing the biologics license application (BLA) and a...
Assessing Miv-Cel for Myasthenia Gravis
This interview was originally published on our sister site, NeurologyLive®. CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy has emerged as a potentially transformative approach in antibody-mediated autoimmune neurological diseases, including myasthenia gravis (MG). The underlying mechanistic premise involves achieving deep depletion of CD19-expressing B-cells through a single treatment course, with the aim of producing a lasting immune reset.
Guess the Therapy Answer May 15, 2026
Answer: lonvoguran ziclumeran (lonvo-z, formerly known as NTLA-2002) See below for further reading on lonvo-z: Lonvo-z Advances Toward Approval as First In Vivo CRISPR Therapy for Hereditary Angioedema April 27, 2026 — Intellia Therapeutics has initiated a rolling biologics license application (BLA) submission to the FDA for lonvoguran ziclumeran (lonvo-z), an investigational one-time CRISPR-based gene editing therapy for hereditary angioedema (HAE), following phase 3 data showing substantial...