Rett Syndrome Gene Therapy Adds RMAT Designation to the List
NGN-401 is being evaluated in an early phase clinical trial which has been selected for the FDA’s START Pilot Program.
Long-Term Axi-Cel Data Shows Durable Outcomes, Elucidates Drivers of Nonrelapse Mortality
Elderly patients were at higher risk of infections and subsequent malignant neoplasms.
FDA Activity Recap: July 2024 Features Agency Program Announcements, a BLA Acceptance, Trial Design Feedback, and More
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
Ocugen’s Retinitis Pigmentosa Gene Therapy Available in EAP Ahead of BLA Submission
Participants are being dosed with OCU400 in the ongoing phase 3 liMeliGhT trial.
Around the Helix: Cell and Gene Therapy Company Updates – August 7, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Joshua M. Hare, MD, on Working to Address Unmet Needs in Alzheimer Disease With Lomecel-B Cell Therapy
The cofounder and chief science officer of Longeveron discussed working to show potential benefits in a follow-up phase 3 trial.
Indapta to Evaluate g-NK Therapy in Trial for Progressive Multiple Sclerosis
The trial evaluating the safety and tolerability of IDP-023 will be led by Stanford and UCSF.
John Finn, PhD, on Using Programmable Genomic Integration to Make Edits Big and Small
The chief scientific officer of Tome Biosciences also discussed the company’s pipeline for its new technology.
FDA Modifies CAR-T Risk Strategies to Lower Healthcare Burden
A recent study also recommended a shorter, more flexible monitoring period post-CAR–T therapy.
David Dimmock, MBBS, on a Promising Case Study of Ultra-Rare, AI-Guided, ASO Development
The chief medical officer at Creyon Bio discussed findings from a treated patient with a TNP02 missense mutation.
Estrella Immunopharma’s Trial for CD19-Targeted T-cell Therapy EB103 in B-Cell Lymphomas Doses First Patient
The open-label, multicenter phase 1/2 STARLIGHT-1 study takes the form of a dose escalation trial with a 3+3 design.
Pivotal Bria-IMT Trial Hopes to Support BLA for Advanced Metastatic Breast Cancer
BriaCell initiated the pivotal phase 3 BRIA-ABC trial in October 2023.
Bringing Autologous Natural Killer Cell Therapy to Parkinson Disease
Paul Y. Song, MD, the chairman and chief executive officer of NKGen, discussed discussed the new clinical study design for evaluating cell therapy SNK01 in PD.
Ixo-Vec Granted RMAT Designation for Wet AMD
At the selected phase 2 dose, 76% of patients were injection free, with maintained visual acuity and fluid control.
William Chou, MD, on Expanding Frontotemporal Dementia Gene Therapy to Both GRN and C9orf72 Mutations
The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA.
mRNA Therapy/Anti-PD-L1 Therapy Combo Meets Primary Endpoint in ORR Over Historical Control
BioNTech is evaluating BNT111 in a phase 2 trial, primary analysis data from which will be presented in a future medical conference.
Galapagos Pauses Enrollment in Multiple Myeloma CAR-T Trial After Parkinsonism Case
The company has filed a protocol amendment with the EMA and anticipates resuming recruitment imminently.
CGTLive®’s Weekly Rewind – August 2, 2024
Review top news and interview highlights from the week ending August 2, 2024.
FDA Approves Adaptimmune’s TCR T-cell Therapy Afami-cel for Synovial Sarcoma
It is the first engineered T-cell therapy to be approved by the FDA for a solid tumor indication.
Obsidian’s TIL Therapy Fast Tracked for Metastatic Melanoma
OBX-115 recently demonstrated a 50% ORR at doses over 30 × 109 cells at 29.5 weeks after infusion.
Scott Jeffers, PhD, on The Importance of Precise Reproducibility of AAVs
The chief technology officer at GenSight Biologics discussed using Verdot’s FlexiPro system to produce Lumevoq gene therapy.
New Study Recommends Shorter, Flexible Monitoring After CAR T-Cell Therapy
There were no new cases of CRS past 2 weeks after infusion and non-relapse mortality was driven by infection in follow-up.
Umoja Biopharma's In Situ CAR-T Therapy Cleared for US Trial in Hematologic Malignancies
The trial will be open to patients with relapsed/refractory large-B-cell lymphoma and chronic lymphocytic leukemia.
Michael Severino on In Vivo Gene Editing With RNA Gene Writers
The chief executive officer of Tessera Therapeutics discussed ongoing research into developing its platform for sickle cell disease, CAR T-cell therapy, and more.
Carisma’s CAR-Monocyte Fast Tracked for HER2-Overexpressing Solid Tumors
CT-0525 is being evaluated in a phase 1 clinical trial currently enrolling participants.
Around the Helix: Cell and Gene Therapy Company Updates – July 31, 2024
Autologous Natural Killer Cells Hold Potential as Novel Treatment for Parkinson Disease
Paul Y. Song, MD, the chairman and chief executive officer of NKGen, discussed the mechanism behind the company’s NK cell therapy SNK01 and promising early results in patients with PD.
Chris Wright, MD, PhD, on Anelloviruses, a Potential Alternative to AAV for Gene Therapy
The chief medical officer and head of translational research at Ring Therapeutics discussed research presented at ASGCT 2024.
Pfizer Drops Development of DMD Gene Therapy Fordadistrogene Movaparvovec
The program’s cancellation follows a previous announcement that its phase 3 trial had missed its primary end point.
saRNA Therapy Gets Rare Pediatric Disease Designation for DMD
Ractigen is also evaluating saRNA therapies for ALS and SMA.
