Infantile GM1 Gangliosidosis Gene Therapy Demonstrates Safety and Dose-dependent Biomarker Changes
Patients who received the high dose of PBGM01 showed an increase in β-Gal activity in the CSF of 4.7 to 5.2 times baseline.
Gene Therapy Yields Neurodevelopmental Improvements in Children With MPSI
Most treated participants were within 2 SDs of normative mean in acquiring cognition, expressive language and fine motor skills.
MPSII Gene Therapy Shows Some Signs of Durable Efficacy
Interim data from the phase 1/2 CAMPSIITE trial were presented at WORLDSymposium 2023.
Samiah Al-Zaidy, MD, on Gene Therapy for Infantile GM1 Gangliosidosis
Al-Zaidy, the vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the results of the phase 1/2 Imagine-01 clinical trial of PBGM01.
CGTLive’s Weekly Rewind – February 24, 2023
Review top news and interview highlights from the week ending February 24, 2023.
Arsa-cel Shows Favorable Risk/Benefit Profile in up to 11 Years of Follow-up Data
Long term data from 2 clinical trials and expanded access programs were presented at WORLDSymposium 2023.
HSC Gene Therapy Demonstrates Improvements in Type 3 Gaucher Disease Case Study
The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.
Graphite Bio Drops Lead Sickle Cell Gene-Edited Cell Therapy
The announcement of a “strategic realignment” comes less than 2 months after the company voluntarily paused the CEDAR clinical trial due to a serious, nula-cel-related adverse event.
Gaucher Disease HSC Gene Therapy Demonstrates Efficacy and Safety in Phase 1/2 Trial
Two patients treated with AVR-RD-02 showed clinically meaningful reductions in liver size.
Increased Motor Development Seen in First 2 Patients With Infantile Krabbe Treated With FBX-101
FBX-101 showed promising efficacy and has been well-tolerated so far in the RESKUE trial.
Around the Helix: Cell and Gene Therapy Company Updates – February 22, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Everett Meyer, MD, PhD, on Reducing GvHD in Leukemia Treatment
The assistant professor of medicine and pediatrics at Stanford University discussed updated data from 2 studies of Orca-T cell therapy.
Etranacogene Dezaparvovec, Marketed as Hemgenix, Approved in EU for Hemophilia B
The gene therapy was approved in the US in the same indication in November 2022.
Brian Van Tine, MD, PhD, on Bringing Cell Therapy Into Synovial Sarcoma Treatment
The professor medicine and pediatrics at Washington University in St. Louis overviewed data from the SPEARHEAD-1 trial of afami-cel.
NMI Bladder Cancer Gene Therapy Shows Promising Safety and Efficacy in Phase 1 Trial
By 3 months, 12 of 17 patients treated with a full cycle of EG-70 achieved a complete response.
Bhagirathbhai Dholaria, MBBS, on Administering Axi-Cel in the Outpatient Setting for LBCL
The assistant professor of medicine at Vanderbilt University Medical Center discussed results from a retrospective analysis presented at the 2023 Tandem Meetings.
ctDNA Assay Panel Predicts CAR-T Response in Multiple Myeloma
It was found that ctDNA levels at 28 days post-treatment were significantly higher for patients who ultimately progressed by 90 days post-treatment.
Surbhi Sidana, MD, on Assessing Ide-cel in Patients With R/R Multiple Myeloma and Renal Impairment
The assistant professor of medicine, bone marrow transplantation and cellular therapy, Stanford, discussed favorable data from a real-world experience study.
Christa Krupski, DO, MPH, on Reinfusion of Tisagenlecleucel in B-ALL
The hematologist/oncologist at Cincinnati Children’s discussed unfavorable outcomes with tisa-cel reinfusion in patients with B-cell acute lymphocytic leukemia.
Ide-cel Demonstrates PFR and ORR Benefit Over Standard of Care in Triple-Class-Exposed R/R Multiple Myeloma
Median PFS was 13.3 months in the ide-cel arm, compared to 4.4 months for the SOC arm.
Madeleine Powys, MBBS, on Delivering Libmeldy to Children With Metachromatic Leukodystrophy
The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the limited distribution model of arsa-cel in Europe.
CD22-CAR Shows Promise in LBCL After CD19 Relapse
Patients with large B-cell lymphoma had an ORR of 68% and a CR rate of 53%.
Gene Therapy Yields 100% 1-Year Survival in LAD-1
No adverse events related to RP-L201 have been reported to date.
Bispecific CAR-T Shows Promising Efficacy in Mantle Cell Lymphoma
Among the 14 patients with relapsed/refractory mantle cell lymphoma who were treated with LV20.19 CAR in the trial, the overall response at 28 days post-treatment was 100%.
Hematopoietic Cell-based Gene Therapy Shows Durable Response in Fanconi Anemia
Long-term hematologic stability has been observed in at least 6 out of 10 patients with at least 12 months of follow-up.
Nirav Shah, MD, on New Data on Zamto-cel in DLBCL, Mantle Cell Lymphoma
The associate professor of medicine at Medical College of Wisconsin discussed trials assessing the dual, CD19/CD20-targeting CAR T therapy.
Tabelecleucel Demonstrates Efficacy, Safety in Phase 3 Trial for EBV-positive Post-transplant Lymphoproliferative Disease
Among 43 patients who had EBV+ PTLD following allogeneic hematopoietic cell transplant or solid organ transplant, the overall response rate was 51.2%.
Pyruvate Kinase Deficiency Gene-Edited Cell Therapy Sustains Hemoglobin Normalization
Neither patient treated with RP-L301 required RBC transfusions at any point post-engraftment.
Matthew Gornet, MD, on Developing Alternatives to Surgery in Lumbar Degenerative Disc Disease
The spine surgeon from The Orthopedic Center of St. Louis discussed unmet needs in the lumbar DDD population.
Cell Therapy Decreases Viral Load in Patients With BK Viremia After Kidney Transplant
Antiviral responses increased until peaking at week 24.
