CardiAMP Cell Therapy Yields 100% Response Rate in Heart Failure
The new, 2-year data is from a roll-in cohort of 10 patients presented at the HFSA 2022 meeting.
B-NHL Cell Therapy Cleared for Trials
Oncternal will soon initiate a phase 1/2 dose-escalation study of ONCT-808.
Gene Therapy Yields Improvements in Danon Disease
Rocket Pharma presented updated data from adult and pediatric patients at the 2022 HFSA meeting.
Solid Bio Adds Friedreich Ataxia to Pipeline With Aavanti Acquisition
Solid has also pivoted from developing SGT-001 to SGT-003 for treating Duchene muscular dystrophy.
Wet AMD Gene Therapy Reduces Anti-VEGF Treatment Burden
Participants in the AAVIATE trial had up to an 85% decrease in treatment burden 6 months after treatment with RGX-314.
Omidubicel Improves Survival in Hematologic Malignancies With HSCT
Disease-free survival was 56% in 105 participants over 3 years.
Modified Immune Cell Therapy Induces Kidney-Donor-Specific Immunosuppression
Patients treated with the highest dose of MIC-Lx had lymphocyte reactivity against third-party cells but not stimulatory donor blood cells.
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.
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.
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-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.
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.
Zevor-Cel Yields 100% ORR in R/R Multiple Myeloma
Eleven patients in the LUMMICAR-2 trial were evaluable for preliminary efficacy analyses.
Artiva to Evaluate CAR-NK Therapy in Solid Tumors
The FDA has cleared the investigational new drug application for AB-201.
IL13Rα2 Universal CAR T Therapy Shows Antitumor Activity in Recurrent Glioma
MT026 had an overall response rate of 83.3% in a first-in-human, investigator-initiated trial.
SNK01/Checkpoint Inhibitor Combo Yields Sustained Remission in Advanced SRC Sarcoma Case
The FDA authorized the natural killer cell therapy combo for compassionate use.
TCR T-Cell Therapy Shows Efficacy Across MAGE-A4+ Solid Tumors
ADP-A2M4CD8 will be evaluated in the phase 2 SURPASS-3 trial initiating in late 2022 or early 2023.
PD1 Knockout CAR T-Cell Therapy Well-Tolerated, Yields Durable Response in DLBCL
A phase 2 trial evaluated the therapy in patients with diffuse large B-cell lymphoma, B-acute lymphocytic leukemia, and follicular lymphoma.
CAR T/CARVac Therapy Yields Responses in Refractory Testicular Cancer
BNT211 showed encouraging responses in testicular cancer but less encouraging responses in ovarian cancer.
Ornithine Transcarbamylase Gene Editing Program Nabs Another FDA Designation
iECURE is planning to submit an IND for GTP-506 in mid-2023.
Astrocyte Cell Therapy Well-Tolerated in Amyotrophic Lateral Sclerosis
Postmortem analyses revealed cell survival and GDNF production in transplanted areas.
Sequential T-Cell Redirection Therapies Yield Deep, Durable Responses in R/R Multiple Myeloma
Patients receiving CAR T-cell therapy or bispecific antibody therapies as first salvage therapy had an ORR of 84%.
IND Halted for T-Cell Malignancy Multiplex-Edited Cell Therapy
The FDA has requested additional preclinical data from Beam Therapeutics on BEAM-201.
FDA Recognizes CRISPR Therapy for Treatment of Hereditary Angioedema
The in vivo genome-editing therapy is the first gene therapy to be evaluated in a clinical trial for preventing HAE attacks.
Regen Developing Novel CAR Approach for Solid Tumors
The company has filed a patent for the new approach, which is designed to combat T-cell exhaustion.
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