September 29th 2023
Review top news and interview highlights from the week ending September 29, 2023.
September 27th 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
September 26th 2023
The phase 1/2 trial is opening its second phase of recruitment after being well-tolerated in the first 4 patients and demonstrating some vision improvements.
September 25th 2023
NFS-05 is an ophthalmic injection that uses an AAV vector to deliver the OPA1 gene to retinal ganglion cells.
September 20th 2023
Ocugen’s Gene-Agnostic Gene Therapy OCU400 Demonstrates Improvement or Stabilization in Retinitis Pigmentosa, Leber Congenital Amaurosis
Among 12 patients evaluable for efficacy, 10 patients experienced stabilization or improvement from baseline in BCVA in their treated eyes.
First Patient Dosed With LCA5 Gene Therapy
Opus Genetics plans to add a pediatric cohort to the phase 1/2 trial once safety is established in adults.
CGTLive’s Weekly Rewind – September 15, 2023
Review top news and interview highlights from the week ending September 15, 2023.
Around the Helix: Cell and Gene Therapy Company Updates – September 13, 2023
Novartis Drops Development of Geographic Atrophy Gene Therapy GT005
Novartis stated that the decision was made in light of a recommendation from an independent data monitoring committee.
Around the Helix: Cell and Gene Therapy Company Updates – September 6, 2023
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.
Around the Helix: Cell and Gene Therapy Company Updates – August 30, 2023
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.
Neurophth Therapeutics’ ND1-LHON Gene Therapy Trial Doses First Patient
The single-arm, open-label clinical trial will seek to enroll approximately 18 patients aged 18 to 75 years.
Around the Helix: Cell and Gene Therapy Company Updates – August 23, 2023
CGTLive’s Weekly Rewind – August 18, 2023
Review top news and interview highlights from the week ending August 18, 2023.
Around the Helix: Cell and Gene Therapy Company Updates – August 16, 2023
Around the Helix: Cell and Gene Therapy Company Updates – August 2, 2023
Around the Helix: Cell and Gene Therapy Company Updates – July 26, 2023
Synthetic Promoters May Help Reduce Off-Target Expression in AAV Gene Therapy
Magdalena Cichewicz, PhD, a scientist II at Senti Biosciences, discussed preclinical research she presented at ASGCT’s 2023 conference.
Around the Helix: Cell and Gene Therapy Company Updates – July 19, 2023
FDA Releases Guidance on CGT Manufacturing Changes, Good Cause for PMR Noncompliance
The guidance outlines recommendations for managing and reporting CGT manufacturing changes and PMR noncompliance.
Around the Helix: Cell and Gene Therapy Company Updates – July 12, 2023
AMD Gene Therapy Cleared for US Trial
A trial in China was initiated in December 2022 for patients with neovascular and wet age-related macular degeneration.
Gene Therapy Yields Vision Improvements in Patients With LHON
Data from patients treated in early access programs were presented at the 9th EAN Congress.
Around the Helix: Cell and Gene Therapy Company Updates – July 5, 2023
Around the Helix: Cell and Gene Therapy Company Updates – June 28, 2023
Around the Helix: Cell and Gene Therapy Company Updates – June 21, 2023
Around the Helix: Cell and Gene Therapy Company Updates – June 14, 2023
Investigating CRB1 Mutations in Retinitis Pigmentosa 12
Sang Yoon Moon, PhD, postdoctoral Research Associate, Lions Eye Institute, discussed research with CRISPRa in human fibroblast cultures.
Around the Helix: Cell and Gene Therapy Company Updates – June 7, 2023
Around the Helix: Cell and Gene Therapy Company Updates – May 31, 2023
Around the Helix: Cell and Gene Therapy Company Updates – May 24, 2023
CGTLive’s Weekly Rewind – May 19, 2023
Review top news and interview highlights from the week ending May 12, 2023.