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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy
Melissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies, and the challenbges associated with them.
Immatics’ TCR-T Therapy Shows Good Phase 1 Safety and Survival Data, Phase 3 Plans Confirmed
Patients with melanoma treated with ACTengine IMA203 achieved high response rates, with progression-free survival of 6 months and duration of response beyond 1 year.
MDA 2025 as a Mixer for Strategies and Collaboration
Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.
Intellia’s CRISPR Gene Editing Therapy NTLA-2002 Appears to Eliminate Hereditary Angioedema Attacks in Some Patients Following One-Time Treatment
Participants in the trial received either a single dose of 25 mg of NTLA-2002, a single dose of 50 mg of NTLA-2002, or a placebo.
Expanding Frontotemporal Dementia Gene Therapy to Both GRN and C9orf72 Mutations
CGTLive®’s Weekly Rewind – October 25, 2024
Review top news and interview highlights from the week ending October 25, 2024.
Improving Newborn Screening for Spinal Muscular Atrophy
Crystal Proud, MD, the director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, spoke about the enhancements to newborn screening for the disease.
Ocugen Gets Green Light to Move Onto Phase 2 Portion of Trial Evaluating Stargardt Gene Therapy OCU410ST
The DSMB’s decision was made with reference to safety data from the GARDian trial’s phase 1 portion.
Sharon Hesterlee, PhD, on MDA 2025 as a Mixer for Strategies and Collaboration
The chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.
Sangamo Reaches Accord With FDA on Plans for Accelerated Approval Pathway for Fabry Disease Gene Therapy ST-920
The company noted that it expects to submit a BLA in the second half of next year.
Around the Helix: Cell and Gene Therapy Company Updates – October 23, 2024
Payer Challenges for Patient Access to Autologous Cell and Gene Therapies
Autologous cell and gene therapies show promise but face barriers such as high costs, limited access, and payer concerns over long-term effectiveness.
Working to Address Unmet Needs in Alzheimer Disease With Lomecel-B Cell Therapy
Joshua M. Hare, MD, the cofounder and chief science officer of Longeveron, discussed working to show potential benefits in a follow-up phase 3 trial.
REGENXBIO’s ABBV-RGX-314 Reduces Need for AntiVEGF Injections After Application in Fellow Eyes of Patients With Wet AMD
The data comes from a phase 2 substudy (NCT03999801) that treated the fellow eyes of patients who had previously been treated with ABBV-RGX-314.
Promising Data on Lomecel-B for Alzheimer Disease
Joshua M. Hare, MD, the cofounder and chief science officer of Longeveron, discussed updated data from the phase 2a CLEAR MIND study.
MeiraGTx’s Parkinson Disease Gene Therapy AAV-GAD Improves Motor Scores and Patient-Reported Quality of Life in Sham-Controlled Study
For the patients treated at the high dose, an average improvement of 18 points from baseline was observed in UPDRS Part 3 “off” medication score at 26 weeks posttreatment.
Using Programmable Genomic Integration to Make Edits Big and Small
John Finn, PhD, the chief scientific officer of Tome Biosciences, discussed the company’s pipeline for its new technology.
Tenaya Therapeutics Cleared to Move Onto Higher Dose Cohort in Trial of Hypertrophic Cardiomyopathy Gene Therapy TN-201
The company also announced protocol changes expanding eligibility for the MyPEAK-1 clinical trial.
CGTLive®’s Weekly Rewind – October 18, 2024
Review top news and interview highlights from the week ending October 18, 2024.
