Kyverna’s CAR-T Gets RMAT Designation for Myasthenia Gravis
A patient with MG is also the first evaluated to reach 1 year of follow-up and has a continued durable immunomodulator-free response.
FDA Clears Biosyngen’s CAR-T Therapy BRG01 for Pivotal Trial in EBV-Positive Nasopharyngeal Carcinoma
BRG01 is the first cell therapy to move into a phase 2 clinical trial for relapsed/metastatic EBV-positive nasopharyngeal carcinoma in both the US and China.
Kiromic BioPharma’s NSCLC T-cell Therapy Deltacel Generates Favorable PFS Data in Interim Phase 1 Trial Results
For the 5 patients in long-term follow-up who have been evaluated, the average PFS is 4.8 months (range, 2-8 months).
William Chou, MD, on Targeting Progranulin With Gene Therapy for Frontotemporal Dementia
The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA.
FDA Lifts Clinical Hold on Trial for 4D Molecular Therapeutics’ Fabry Disease Gene Therapy 4D-310
In light of the FDA’s decision, 4DMT plans to continue enrolling patients in INGLAXA before the end of 2024.
As Gene Editing Technology Advances, Numerous Potential Modalities Emerge
Janice Chen, PhD, the cofounder and chief technology officer of Mammoth Biosciences, discussed the importance of diverse approaches to gene editing to address a variety of indications.
Tr1 Treg Therapy to be Evaluated in Trial for Crohn Disease
TRX103 is also being evaluated for the prevention of graft versus host disease (GvHD) in patients undergoing HLA-mismatched HSCT.
Alexandra Collin de l’Hortet, PhD, on Evaluating Epigenetic Gene Therapy for the Treatment of FSHD
The head of therapeutics at Epic Bio discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.
BioMarin Limits Geographic Scope of its Plans for Hemophilia A Gene Therapy
Going forward, the company is mainly focused on distributing the gene therapy in the United States, Germany, and Italy.
CGTLive®’s Weekly Rewind – August 9, 2024
Review top news and interview highlights from the week ending August 9, 2024.
David Dimmock, MBBS, on Accelerating Therapy Discovery and Approval With AI
The chief medical officer at Creyon Bio discussed future applications for the company’s AI-guided discovery platform.
Delivering Ultracompact CRISPR Systems With AAV
Lucas Harrington, PhD, the cofounder and chief scientific officer of Mammoth Biosciences, discussed the company’s mouse model research on treating hypertriglyceridemia.
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
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.