uniQure's Mesial Temporal Lobe Epilepsy Gene Therapy AMT-260 Cleared for US Trial
In light of the IND clearance, uniQure announced its intention to carry out a phase 1/2a clinical trial, with screening of potential participants with refractory MTLE anticipated to begin in the last 3 months of 2023.
Nusinersen Show Benefits in Other SMA Populations
New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.
Pamela Garzone, PhD, on Exploring Unknowns in CAR-T for Solid Tumors
The chief development officer of Anixa Biosciences discussed the company’s trial of a CAR-T in ovarian cancer that is looking at the effect of regional administration and lymphodepletion on efficacy.
Axi-Cel Expands LBCL Indication in China
The CAR T-cell therapy, marketed as Yikaida in China, has been approved for second-line treatment for patients with refractory/relapsed large B-cell lymphoma.
Around the Helix: Cell and Gene Therapy Company Updates – September 6, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Wave Life Sciences Submits CTA for Alpha-1 Antitrypsin Deficiency RNA Editing Therapy WVE-006
The therapy is expected to be able to treat both the lung and liver manifestations of AATD.
Fiona Freeman, PhD, on Investigating miRNA-29b in Osteosarcoma Models
The assistant professor at University College Dublin discussed challenges and different approaches to using microRNA in preclinical models of osteosarcoma.
Arsa-cel Continues to Show Benefit Up To 12 Years of Follow-up in Metachromatic Leukodystrophy
The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.
Pamela Garzone, PhD, on Tackling Challenges of Treating Solid Tumors With CAR-T
The chief development officer of Anixa Biosciences discussed how the company is tackling 2 major challenges presented by solid tumors in its ovarian cancer clinical trial.
Q&A with ACGT Research Fellow Stephen Gottschalk, MD
Stephen Gottschalk, MD, is using grant funding from ACGT to develop CAR T-cell therapy to treat children with sarcomas. He spoke about the challenges of treating sarcomas, the need for new therapies, and the importance of ACGT.
Alliance for Cancer Gene Therapy Awards Grant for Brain Cancer Research Project
ACGT recently awarded a half-million dollar grant to brain cancer researcher Juan Fueyo, MD, who is developing an oncolytic virus therapy to treat the brain cancer glioblastoma.
Efforts to Advance Cell and Gene Therapies for Children With Cancer
September is Childhood Cancer Awareness Month, serving as a chance to assess what progress has been made in advancing cell and gene therapy research for children with cancer.
Kyverna Makes Progress Evaluating Lupus Nephritis Cell Therapy in US, Germany
Phase 1/2 trials have been cleared in Germany and dosed first patients in the US.
CGTLive’s Weekly Rewind – September 1, 2023
Review top news and interview highlights from the week ending September 1, 2023.
CRISPR-Cas9 Edited Therapy OTQ923 Displays Early Promise for Sickle Cell Disease
In a small sample of patients, the CRISPR–Cas9–edited CD34+ hematopoietic stem- and progenitor-cell therapy showed expected safety while inducing red cell fetal hemoglobin.
Genprex Takes a Three-Pronged Approach to Assessing Reqorsa for Lung Cancer
Acclaim-1, Acclaim-2, and Acclaim-3 will evaluate Reqorsa in combination with osimertinib, pembrolizumab, and atezolizumab, respectively.
Abeona’s Sending Off RDEB Cell Therapy For BLA Submission
In recent data from the phase 3 VITAL trial, EB-101 decreased pain and improved wound healing.
UC Davis Eye Center Administers Wet AMD Gene Therapy ABBV-RGX-314 to Participant in Phase 3 Study
It was the first time the Eye Center treated a patient with a gene therapy.
Design Therapeutics' DT-216 Displays Positive Phase 1 Results in Friedreich Ataxia
Data from a multiple-ascending dose study suggest the investigational agent developed by Design Therapeutics was generally well tolerated with dose-related increases in frataxin levels.
Around the Helix: Cell and Gene Therapy Company Updates – August 30, 2023
Investing in Long-Term Studies May Provide a Better Picture of Cell Therapy Efficacy
Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed what the company learned from relitigating results of legacy trials for a mesenchymal stem cell therapy in Crohn disease.
TSHA-102 Gets FDA Fast Track Designation in Rett Syndrome
TSHA-102 has previously received orphan drug and rare pediatric disease designations from the agency and is being investigated in the REVEAL phase 1/2 trial (NCT05606614).
X-Linked Retinoschisis Gene Therapy Trial Doses First Patient
ATSN-201 utilizes Atsena’s novel spreading capsid AAV.SPR, which is expected to spread laterally and allow transduction of the gene therapy in the central retina.
Investigational AAV8 Gene Therapy GNT0003 Shows Safety in Crigler–Najjar Syndrome
In a small study, administration of the Généthon therapy encoding UGT1A1 resulted in no serious adverse events. It is also being evaluated in a larger pivotal trial.
FDA Approves Bristol Myers Squibb’s Reblozyl for Expanded Indication in Myelodysplastic Syndromes
The FDA’s decision was based on data from the randomized, open-label phase 3 COMMANDS clinical trial.
BlueRock Therapeutics' Parkinson Disease Cell Therapy Bemdaneprocel Shows Initial Efficacy in Phase 1 Trial
In light of the promising results, BlueRock is preparing for a phase 2 trial.
Allogeneic Natural Killer Cell Trial in Solid Tumors Doses First Patient
NKGen Biotech’s trial for SNK02 was initiated following the clearance of an investigational new drug application by the FDA in October 2022.
Robert J. Hariri, MD, PhD, on the Unique Potential of Chimeric Therapy
The founder, chairman, and CEO of Celularity shared his view on the concept of “chimeric vigor” and the potential benefits arising from it in the cell therapy field.
Taking a Second Look at Placental-Derived Mesenchymal-Like Adherent Stromal Cell Therapy for Crohn Disease
Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed a new analysis of gene and protein signatures from patients treated in the company’s legacy clinical trials.
Poseida's Dual-Targeted Allogeneic CAR T Trial Cleared for B-Cell Malignancies
The company also recently entered into a collaboration with Roche and was invested in by Astellas.
2 Clarke Drive Cranbury, NJ 08512