FDA Approves PTC Therapeutics’ Gene Therapy Kebilidi for AADC Deficiency
The gene therapy has been approved in the UK and European Union since 2022 under the brand name Upstaza.
Around the Helix: Cell and Gene Therapy Company Updates – November 13, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Neurogene’s Rett Syndrome Gene Therapy NGN-401 Demonstrates Efficacy in Low-Dose Cohort, But Serious Adverse Event in High-Dose Group Raises Concerns
Neurogene pointed out that the SAE is consistent with the known risks of AAV vector-based gene therapy.
Anelloviruses, a Potential Alternative to AAV for Gene Therapy
Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics, discussed research presented at ASGCT 2024.
Improving Cell and Gene Therapy with Innovative Nonviral Delivery Vectors
An overview of novel drug-delivery vehicles and their potential role in improving gene and cell therapies.
Eureka Therapeutics Tackling Advanced Hepatocellular Carcinoma With T-Cell Therapy ECT204 in Phase 1/2 ARYA-3 Clinical Trial
Following up on Liver Cancer Awareness Month, which is observed annually in October by the patient and clinician communities, CGTLive has decided to take a closer look at this ongoing study.
Gastric Cancer Awareness Month 2024: Looking Back at Progress for Cell Therapy
In observance of Gastric Cancer Awareness Month, held annually in November, we took a look back at the past few months' news in cell therapy for gastric cancer indications.
Patient With Sickle Cell Disease Dies From Complications Related to Busulfan-Conditioning Following Treatment With Beam’s Base-Editing HSC Therapy BEAM-101
The patient died of respiratory failure 4 months after receiving BEAM-101.
FDA Approves Obe-Cel for Adults With R/R B-Cell Precursor Acute Lymphoblastic Leukemia
The CAR-T therapy was approved based on the pivotal phase 1b/2 FELIX clinical trial (NCT04404660), which showed good rates of overall complete remission and median duration of remission.
CGTLive®’s Weekly Rewind – November 8, 2024
Review top news and interview highlights from the week ending November 8, 2024.
NTLA-2002 Gene Editing Reduces Angioedema Attacks in Hereditary Angioedema
Intellia’s investigational CRISPR-Cas9 gene-editing therapy NTLA-2002 reduced monthly attacks by more than 70% in both tested dose arms.
FDA Removes Clinical Holds Previously Placed on 3 of CARsgen's Oncology Cell Therapies
The clinical holds on the 3 products were originally placed by the FDA in December 2023 based on findings from an inspection of CARsgen’s manufacturing facility.
Lung Cancer Awareness Month 2024: Looking Back at Progress for Cell and Gene Therapy
In observance of Lung Cancer Awareness Month, held annually in November, we took a look back at the past few months' news in cell and gene therapy for lung cancer indications.
Around the Helix: Cell and Gene Therapy Company Updates – November 6, 2024
HuidaGene Therapeutics’ CRISPR/Cas13 RNA-Editing Therapy HG202 Gets Greenlight for US Trial in Age-Related Macular Degeneration
According to HuidaGene, it is the first CRISPR/Cas13Y RNA-targeting therapy to receive clearance for a clinical trial from the FDA or any other regulatory agency.
Gene Therapy for APOE4 Homozygous Alzheimer Disease
R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.
Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research
The noninvasive gene therapy exceeded the 30% efficacy threshold for normalizing GCase activity across all doses.
Noninflammatory Neurotoxicity Events in Patients Receiving CAR-T
Caroline Diorio, MD, FRCPC, FAAP, an attending physician at the Cancer Center at Children's Hospital of Philadelphia, discussed a study she coauthored that was recently published in Blood.
Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves Visual Acuity in Randomized Controlled Clinical Trial
The randomized, controlled clinical trial treated patients with either a high dose of MCO-010, a low dose of MCO-010, or a sham–control.
Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease
LX1001's safety profile was characterized as well-tolerated with no amyloid-related imaging abnormalities reported.
FDA Activity Recap: October 2024 Features IND Clearances and Company Alignment on Plans in FDA Meetings
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
RiboX's Circular RNA Therapy RXRG001 Cleared for Phase 1/2a Trial in Radiation-Induced Xerostomia and Hyposalivation
RiboX stated that this is the first IND clearance the FDA has granted for any circular RNA therapy.
Caroline Diorio, MD, FRCPC, FAAP, on Noninflammatory Neurotoxicity Events in Patients Receiving CAR-T
The attending physician at the Cancer Center at Children's Hospital of Philadelphia discussed a study she coauthored that was recently published in Blood.
CGTLive®’s Weekly Rewind – November 1, 2024
Review top news and interview highlights from the week ending November 1, 2024.
Data Roundup: October 2024 Features Update for TCR-Based Autologous Cell Therapy in Melanoma, the First Clinical Demonstration of Therapeutic RNA Editing in Humans, and More
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Abeona Therapeutics Resubmits BLA to FDA for Epidermolysis Bullosa Gene Therapy Pz-Cel
The company made the resubmission after reaching an accord with the FDA in a Type A meeting with the agency that took place in August 2024.
R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, on Gene Therapy for APOE4 Homozygous Alzheimer Disease
The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.
Targeting Progranulin With Gene Therapy for Frontotemporal Dementia
William Chou, MD, president and chief executive officer of Passage Bio, discussed feedback from a recent Type C meeting with the FDA.
Around the Helix: Cell and Gene Therapy Company Updates – October 30, 2024
BridgeBio Pharma’s Gene Therapy BBP-812 Effects Progressive Improvement in Motor Function in Patients With Canavan Disease
Furthermore, the majority of patients treated with the gene therapy showed improved myelination as measured by T-2-weighted MRI.
