March 17th 2023
Intergalactic Therapeutics’ IG-002 utilizes a proprietary non-viral delivery method.
March 15th 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
March 14th 2023
GenSight Biologics reported that 73% of patients treated bilaterally with Lumevoq improved at least +15 ETDRS letters relative to their worst recorded BCVA.
March 11th 2023
Serious adverse events of reduced visual acuity and eye inflammation were observed in 2 clinical trials.
March 8th 2023
LHON-ND4 Gene Therapy Trial Completes Enrollment
NR082 is being evaluated in a phase 1/2/3 clinical trial in patients with Leber hereditary optical neuropathy.
Around the Helix: Cell and Gene Therapy Company Updates – March 1, 2023
Around the Helix: Cell and Gene Therapy Company Updates – February 22, 2023
Around the Helix: Cell and Gene Therapy Company Updates – February 15, 2023
HHS, CMS to Promote Model for Improved Access to Cell and Gene Therapies, Urge Accelerated Approval Trials to Completion
A third price model to be tested will limit the price of generic drugs for chronic conditions to $2 under Medicare plan D.
Wet AMD Gene Therapy Trial Demonstrates Consistent Safety and Efficacy With New Manufacturing Process
No serious adverse events deemed related to RGX-314 occurred in the treated patients.
Gene Therapy Continues to be Well-Tolerated in Retinitis Pigmentosa
Encouraging efficacy signals have also been observed in treated participants.
CGTLive’s Weekly Rewind – February 10, 2023
Review top news and interview highlights from the week ending February 10, 2023.
Around the Helix: Cell and Gene Therapy Company Updates – February 8, 2023
Preclinical AMD Gene Therapy Shows Potential for Other Retinal Diseases
The new findings are consistent with previous research, in which ophNdi1 demonstrated benefit in AMD models.
IND for Gene Replacement Therapy for Inherited Retinal Dystrophy Cleared by FDA
The company is developing a one-time, non-AAV2 gene replacement therapy to restore, treat, and prevent blindness of patients with RPE65 mutation-associated retinopathies.
CGTLive’s Weekly Rewind – February 3, 2023
Review top news and interview highlights from the week ending February 3, 2023.
FDA Blocks 4D’s Gene Therapy Program for Fabry, Clears IND for Diabetic Macular Edema
The clinical hold comes a few weeks after the company announced it was stopping enrollment in the phase 1/2 clinical trial.
Gene Therapy Trial Doses First Patient With Wet AMD in China
The phase 1 clinical trial is the second of Frontera’s trials to begin dosing in 2023.
Around the Helix: Cell and Gene Therapy Company Updates – February 1, 2023
Multi-characteristic Opsin Gene Therapy Gets Second Fast Track for Retinal Degenerative Diseases
MCO-010 is in phase 2 clinical trials for treating retinitis pigmentosa and Stargardt disease.
Around the Helix: Cell and Gene Therapy Company Updates – January 25, 2023
10 Cell and Gene Therapies to Watch in 2023
With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.
Gene Therapy Improves Visual Acuity in MT-ND4 LHON Compared to Natural History
Investigators analyzed data from 3 phase 3 studies and a long-term follow-up study.
Around the Helix: Cell and Gene Therapy Company Updates – January 18, 2023
CAR-T for NMOSD Demonstrates Manageable Safety in Phase 1 Trial
It was previously announced that CT103A received FDA clearance of its investigational new drug application for the treatment of relapsed/refractory (r/r) multiple myeloma.
Neovascular AMD Gene Therapy Cleared for Trials
A phase 1 clinical trial of EXG102-031 is expected to initiate in the first quarter of 2023.
Around the Helix: Cell and Gene Therapy Company Updates – January 11, 2023
Leber Congenital Amaurosis-2 Gene Therapy Trial Doses First Patient
The company expects to provide initial data from the trial in the second half of this year.
Around the Helix: Cell and Gene Therapy Company Updates – January 4, 2023
2022 Year in Review: Top News and Insights in Cell Therapy
Review some of our most-viewed coverage of advancements in cell therapies, including study data and clinical trial updates.
2022 Year in Review: Top News and Insights in Gene Therapy
Review some of our most-viewed coverage of advancements in gene therapies, including study data and clinical trial updates.
2022 Year in Review: Top Conversations on Cell and Gene Therapy
Review some of our most-viewed interviews with clinicians, researchers, and biotech companies about expanding applications of cell and gene therapies.
IND Cleared for Leber Hereditary Optic Neuropathy Gene Therapy
NFS-02 previously received orphan drug designation from the FDA in January of this year.
Around the Helix: Cell and Gene Therapy Company Updates – December 21, 2022