Taking a Second Look at Placental-Derived Mesenchymal-Like Adherent Stromal Cell Therapy for Crohn Disease
Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed a new analysis of gene and protein signatures from patients treated in the company’s legacy clinical trials.
Poseida's Dual-Targeted Allogeneic CAR T Trial Cleared for B-Cell Malignancies
The company also recently entered into a collaboration with Roche and was invested in by Astellas.
CGTLive’s Weekly Rewind – August 25, 2023
Review top news and interview highlights from the week ending August 25, 2023.
BCMA-Directed CAR-T NXC-201 Gets FDA Orphan Drug Designation in Multiple Myeloma
Because of the lack of neurotoxicity seen in patients treated thus far, Nexcella believes NXC-201 may have potential to serve as an outpatient treatment.
Thomas Povsic, MD, PhD, on Addressing Unmet Needs in Refractory Angina With Gene Therapy
The interventional cardiologist and professor, Duke University School of Medicine, discussed how XC001 could improve outcomes for patients with refractory angina.
Arrowhead Seeks Clinical Trials for ALS, Muscular Dystrophy RNAi Therapies
Pending approval, ARO-DUX4 will be evaluated in patients with facioscapulohumeral muscular dystrophy and ARO-SOD1 will be evaluated in patients with amyotrophic lateral sclerosis.
Robert J. Hariri, MD, PhD, on Cord Blood Awareness Month 2023
The founder, chairman, and CEO of Celularity shared his view on the importance of cord blood-derived cells for the medical and clinical community.
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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CAR-M/Pembrolizumab Trial Doses First Patient With HER2-Overexpressing Solid Tumors
Initial data from the combination substudy are expected in the second half of 2023.
Gene Therapy Improves Grade 2/3 Xerostomia Symptoms
Participants treated both unilaterally or bilaterally reported clinically meaningful improvements on GRCQ and XQ scale scores.
Robert J. Hariri, MD, PhD, on the Importance of Placental-Derived Stem Cells in Cell Therapy
The founder, chairman, and CEO of Celularity discussed the advantages of cells derived from umbilical cord blood versus other sources of cells for cell therapies.
Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation
Earlier this year Bloomsbury Genetic Therapies met with the UK’s MHRA for a scientific advice meeting to discuss BGT-NPC.
Gaucher Disease Type 1 Gene Therapy Trial Proceeds to Dose Second Cohort
The company announced that it had completed dosing in the first cohort of the GALILEO-1 trial of FLT201.
Wugen’s NK Cell Therapy Trial Doses First Patient With AML
The company also reported that WU-NK-101 has received orphan drug designation for treating acute myeloid leukemia from the FDA.
Thomas Crawford, MD, on Bridging the Gap Between Screening and Treatment for Infants With SMA
The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the importance of connecting infants with spinal muscular atrophy to a therapy as soon as possible.
Ornithine Transcarbamylase Deficiency Gene Therapy Gets FDA Orphan Drug Designation
BGT-OTCD is being developed in a collaboration between Bloomsbury Genetic Therapies and University College London.
Hemophilia Gene Therapy Shared Decision Making Tool Needed, Providers Say
Surveyed clinicians and nurses from Rady Children's Hospital San Diego identified important factors and considerations needed in a potential SMD tool.
Artiva Biotherapeutics’ NK Cell Therapy AlloNK Cleared for Trial in Systemic Lupus Erythematosus
AlloNK will be evaluated as part of a combination therapy with anti-CD20 monoclonal antibody rituximab.
CGTLive’s Weekly Rewind – August 18, 2023
Review top news and interview highlights from the week ending August 18, 2023.
Richard Fessler, MD, on OPC1 Improving Quality of Life With Spinal Cord Injury
The professor of neurosurgery at Rush University Medical School discussed follow-up in the phase 1/2 trial of LCTOPC1 and further research to be done.
Neurogene’s Clinical Trial Seeks to Assess CLN5 Batten Disease Gene Therapy NGN-101
NGN-101 will be delivered to each participant via both intracerebroventricular and intravitreal routes on the same day in the phase 1/2 trial, with the therapy assessed for safety and efficacy.
No AdComm Meeting for Sickle Cell Therapy Lovo-Cel
Bluebird's gene-edited cell therapy has a PDUFA date of December 20, 2023.
ImmPACT Bio’s Systemic Lupus Erythematosus CAR-T Cleared for US Trial
IMPT-514 uses the same CAR-T construct as ImmPACT Bio’s IMPT-314, which is already under clinical evaluation for non-Hodgkin lymphoma.
David Boyer, MD, on Potential Advantages of Suprachoroidal Gene Therapy for Neovascular AMD
The senior partner at Retina Vitreous Associates Medical Group discussed new findings from the AAVIATE study of RGX-314.
Around the Helix: Cell and Gene Therapy Company Updates – August 16, 2023
MB-106 Shows Potential in Indolent Lymphoma
Mustang reported new data and responses from a multicenter phase 1 trial evaluating its CAR T-cell therapy in patients with NHL, FL, and WM.
Genprex Garners Orphan Drug Designation for Lung Cancer Immunogene Therapy Reqorsa
The new orphan drug designation follows a recently received fast track designation for Reqorsa in combination with Tecentriq for an extensive-stage small cell lung cancer indication.
Paul Harmatz, MD, on Challenges With Assessing Neurocognitive Outcomes
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges with clinical trials in neurocognitive diseases including MPS2.
FDA Lifts Partial Clinical Hold on Arcellx’s Trial for Multiple Myeloma CAR-T CART-ddBCMA
The partial clinical hold was originally placed in June 2023 in relation to the death of a patient participating in the trial.
