KIR-CAR T-Cell Therapy Recognized for Mesothelioma Treatment
Verismo plans to initiate a phase 1, first-in-human trial of SynKIR-110 in the first quarter of 2023.
Sarepta’s DMD Gene Therapy Goes In Front of FDA With BLA Submission
SRP-9001 has shown safety and efficacy across multiple studies compared to controls, with additional efficacy data expected in 2024 from the EMBARK trial.
Cellenkos to Try T Regulatory Cell Approach in ALS
A phase 1 and phase 1b study of CK0803 will soon be initiated following the FDA’s IND clearance.
Allogeneic CAR T Recognized for Cutaneous T-Cell Lymphomas
CTX-130 is being investigated in phase 1 studies for relapsed/refractory T or B-cell malignancies and renal cell carcinoma.
Around the Helix: Cell and Gene Therapy Company Updates – September 28, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Leaping the Next Hurdle in CAR T-Cell Therapies
Phil Cyr, Senior Vice President, Precision Value & Health, discussed challenges to tackle with CAR T-cell therapies.
Vertex Continues Gene Therapy Momentum in Blood Disorders With Exa-Cel Submission
Bluebird bio’s ZYNTEGLO was the first gene therapy to be approved in the space in August 2022.
Repeated Injections of ALS Gene Therapy Well-Tolerated
Although efficacy and HGF expression were assessed as outcome measures, comparisons were not able to be made between placebo and Engensis groups.
DMD Cell Therapy Shows Promise in Early Clinical Trial Data
The 3 patients demonstrated a mean significant improvement of up to 200% of baseline in motor unit potential.
Optimizing CAR T Therapies for Gastric Cancer
Hong Ma, senior vice president, clinical development, cancer immunotherapy, CARSgen, discussed data from a phase 1 trial of CT041.
University of Miami Researcher Awarded $3.5 Million for Research on Gene Therapy for Usher Syndrome
Xue Zhong Liu, MD, PhD, and research partner Zheng-Yi Chen, PhD, will investigate gene editing and CRISPR/Cas9 systems to potentially treat Usher syndrome.
First Patient Dosed in Limb-Girdle Muscular Dystrophy Gene Therapy Trial
The 2-stage, multicenter clinical trial will recruit approximately 39 patients who have been diagnosed with LGMDR9.
Deyaa Adib, MD, on Safety Advantages of TAC T-Cell Therapy in Solid Tumors
The chief medical officer of Triumvira Immunologics discussed how TAC01-HER2 could address unmet needs in treating solid tumors.
First-in-Human Trial to Assess KIR-CAR T-Cell Therapy in MSLN+ Solid Tumors
Verismo plans to begin enrollment in the STAR-101 trial in the first quarter of 2023.
Target for Polycystic Kidney Disease Gene Therapy Might Lie in MicroRNA Binding Sites
Elimination of the miR-17 binding site on PKD1 and PKD2 mRNA alleviated cyst growth in preclinical models.
Jeffrey Miller, MD, on Emerging Research With Natural Killer Cells
The deputy director of the Masonic Cancer Center discussed trends of presentations from ESMO 2022.
HIV Gene Editing Clinical Trial Treats First Patient
The patient is expected to qualify for analytical treatment interruption of background anti-retroviral therapy.
CGTLive’s Weekly Rewind – September 23, 2022
Review top news and interview highlights from the week ending September 23, 2022.
Pfizer, Sangamo Reopen Phase 3 Study of Hemophilia A Gene Therapy
The FDA previously lifted its clinical hold of the AFFINE study in May 2022.
Decibel Puts Forth Congenital Hearing Loss Gene Therapy for Clinical Trials
The company previously presented positive preclinical data at the 2021 ASGCT meeting.
Roberto Giugliani, MD, PhD, on Reducing GAGs in MPS Type 2 With Gene Therapy
The professor of genetics at Federal University of Rio Grande do Sul discussed updated data from the CAMPSIITE trial.
Zevor-Cel Yields 100% ORR in R/R Multiple Myeloma
Eleven patients in the LUMMICAR-2 trial were evaluable for preliminary efficacy analyses.
Deyaa Adib, MD, on Evaluating HER2 TAC T-Cell Therapy in Solid Tumors
The chief medical officer of Triumvira Immunologics discussed updates from the TACTIC-2 study presented at ESMO Congress 2022.
Around the Helix: Cell and Gene Therapy Company Updates – September 21, 2022
Artiva to Evaluate CAR-NK Therapy in Solid Tumors
The FDA has cleared the investigational new drug application for AB-201.
Jeffrey Miller, MD, on Engaging NK Cells in Solid Tumors
The deputy director of the Masonic Cancer Center discussed the preclinical findings of his study on a TriKE engager and NK cells.
Cilta-Cel Yields High MRD-Negativity Rates in Multiple Myeloma After Prior Anti-BCMA Treatment
Seven of 10 evaluable patients achieved minimal residual disease negativity after cilta-cel treatment.
Streamlining Stem Cells to Treat Macular Degeneration
Scientist have proposed several methods for converting stem cells into RPE, but there is still a gap in our knowledge of how cells respond to these stimuli over time.
Eli-Cel Approved for CALD, Marking Second Gene Therapy Win for bluebird bio
The approval follows unanimous support of eli-cel's benefits in CALD from an FDA AdComm meeting and an approval for beti-cel in beta-thalassemia.
Intellia Nets Positive Data Across In Vivo CRISPR Therapies for ATTR Amyloidosis Cardiomyopathy, Angioedema
Administration of NTLA-2001 led to rapid and deep reductions in serum TTR by day 28.
