Around the Helix: Cell and Gene Therapy Company Updates – May 3, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Bart P. Leroy, MD, PhD, on Improving Light Sensitivity, Quality of Life With Luxturna
The head of the Department of Ophthalmology at the Center for Medical Genetics, Ghent University Hospital, discussed the main takeaways of a post-marketing study on the gene therapy.
X-Linked Retinoschisis Gene Therapy Cleared for First-in-Human Trial
Atsena anticipates initiating the Lighthouse study of ATSN-201 in mid-2023.
Jay Barth, MD, on Ascidian Therapeutics' Developments in Gene Therapy
CGTLive's sister publication spoke with Barth, the chief medical officer at Ascidian, about the company’s attempts to develop a new method of administration for gene therapy at ARVO 2023.
Liso-cel Meets Primary End Point in Follicular and Mantle Cell Lymphoma Trials
Bristol Myers Squibb noted that in addition to meeting the ORR end point, both trials also met a key secondary CR rate end point.
Steven Katz, MD, on Tackling TME Immunosuppression, Intratumoral Pressure
The chief medical officer of Trisalus Life Sciences discussed the company’s PEDD system and SD-101.
Non–Small Cell Lung Cancer Cell Therapy Receives FDA IND Clearance
Kiromic BioPharma expects to initiate a phase 1 clinical trial for Deltacel within Q2 2023.
T-cell Therapy Prevents Multiple Virus Infections after Allo-HSCT
Updated 52-week follow-up data were presented at the EBMT meeting.
James Lim, PhD, on the Challenges Posed by the Solid Tumor Microenvironment
The cofounder and chief scientific officer of Xcell Biosciences discussed the limitations of the methods currently used for modeling in preclinical cell therapy research for solid tumors.
Structural Changes Support Gene Therapy's Effect on Geographic Atrophy
New data from imaging analyses on RG6501 were presented at the ARVO 2023 Annual Meeting.
Luxturna Demonstrates Improvements on Multiple Vision Outcome Measures in Real-World Setting
The study found significant improvements in BCVA, in contrast to several other studies evaluating Luxturna.
Advanced Retinitis Pigmentosa Optogenetic Therapy Demonstrates Vision Function Improvements
The data, presented at ARVO's 2023 conference, also showed a favorable safety profile.
CGTLive’s Weekly Rewind – April 28, 2023
Review top news and interview highlights from the week ending April 28, 2023.
Arnaud Lacoste, PhD, on AURN001‘s Immunomodulatory Properties
The chief scientific officer of Aurion Biotech discussed the properties of the cell therapy for corneal dystrophies, which was recently approved in Japan.
CRISPR/Cas9 Therapy Shows Some Visual Improvements in LCA10
GenSight Biologics’ Lumevoq Demonstrates Clinically Meaningful Improvements in Early Access Programs
The new data builds upon positive long-term follow-up data from clinical trials.
Bart P. Leroy, MD, PhD, on Continuing to Verify Luxturna’s Efficacy
The head of the Department of Ophthalmology at the Center for Medical Genetics, Ghent University Hospital, discussed the results of a post-marketing study presented at ARVO’s 2023 conference.
Frank Borriello, MD, PhD, on Promising Activity of SUPLEXA-101 Across Cancers
The scientific cofounder and chief executive officer of Alloplex Biotherapeutics discussed early data from a phase 1 trial of the PBMC-derived therapy.
MT-ND4 LHON Gene Therapy Continues to Yield Vision Improvements
The safety profile of lenadogene nolparvovec at 5-years post treatment was similar to 2-year data.
Around the Helix: Cell and Gene Therapy Company Updates – April 26, 2023
Rachel Lynn, PhD, on Reprogramming Cell Therapies to Combat Exhaustion
The associate director of research at Lyell Immunopharma discussed the company’s programs and newest technologies aimed at solid tumors.
Zinaida Good, PhD, on Exploring CAR-T Expansion Through Lineage Tracing
The instructor at Stanford Institutes of Medicine discussed the key takeaways of her research into the factors that affect CAR-T expansion.
LCA1 Gene Therapy Improves Sensitivity, BCVA at Highest Dose
No serious TEAEs have been related to ATSN-101 with data up to 6 months after treatment.
Arnaud Lacoste, PhD, on Addressing Unmet Needs in Corneal Dystrophies
The chief scientific officer of Aurion Biotech discussed the investigational cell therapy AURN001.
PDE6A-associated Retinitis Pigmentosa Gene Therapy May Cause Loss of Vision in Some Patients
STZ eyetrial’s treatment rAAV8.hPDE6A was otherwise deemed well-tolerated.
bluebird bio Submits BLA for Sickle Cell Gene-Edited Cell Therapy, Lovo-cel
The BLA submission comes after a series of delays which have pushed lovo-cel behind Vertex’s exa-cel in the race to approval.
Luxturna’s Safety and Efficacy Profile Supported by Retrospective Data in Patients With RPE65-Associated Inherited Retinal Dystrophy
The commercially available gene therapy, developed by Spark Therapeutics, was evaluated in multiple studies presented at ARVO’s 2023 conference.
Spark Therapeutics’ Luxturna Improves Light Sensitivity in Postmarketing Study of Inherited Retinal Dystrophy
The new positive data on the AAV based gene therapy builds upon real-world data presented at ARVO’s conference in 2022.
Leticia Tordesillas, PhD, on the Potential of γδ CAR T-cells to Treat Bone Metastatic Prostate Cancer
The research scientist at Moffitt Cancer Center discussed the results of the preclinical study she presented at the 2023 AACR annual meeting and avenues for further research.
Therapeutic Solutions International Seeks to Expand JadiCell Treatment to Broader ARDS Population
JadiCell is already being evaluated for the treatment of COVID-19 associated ARDS in a phase 3 trial.
