Gene Therapy Trial for Stargardt Disease Reaches Full Enrollment
Nanoscope Therapeutics’ Phase 2 STARLIGHT open-label trial enrolled 6 subjects with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease.
Around the Helix: Cell and Gene Therapy Company Updates – September 14, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Gene Therapy for OTOF-Mediated Hearing Loss Gets Green Light to Enter the Clinic
In nonclinical studies, AK-OTOF was well-tolerated in both mice and non-human primates.
BioMarin to Investigate B-ALL Case in Val-Rox Gene Therapy Clinical Trial
The gene therapy received a conditional marketing approval in the European Union in August 2022.
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.
Oncolytic Virus T-VEC Demonstrates Long-Term Benefits in Melanoma
The 5-year relapse-free survival rate was 22.3% with T-VEC prior to surgery, compared with 15.2% for surgery alone.
TIL Therapy Superior to Ipilimumab for Progression-Free Survival in Unresectable Melanoma
Based on the findings, investigators are considering registration with the FDA and EMA.
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.
CGTLive’s Weekly Rewind – September 9, 2022
Review top news and interview highlights from the week ending September 9, 2022.
CAR T/CARVac Therapy Yields Responses in Refractory Testicular Cancer
BNT211 showed encouraging responses in testicular cancer but less encouraging responses in ovarian cancer.
Noopur Raje, MD, on bb21217 Data in R/R Multiple Myeloma
The director, Center for Multiple Myeloma, Massachusetts General Hospital, discussed data seen with bb21217 in R/R MM.
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%.
Around the Helix: Cell and Gene Therapy Company Updates – September 7, 2022
IND Halted for T-Cell Malignancy Multiplex-Edited Cell Therapy
The FDA has requested additional preclinical data from Beam Therapeutics on BEAM-201.
Tecartus Approved in Europe for R/R Acute Lymphoblastic Leukemia
Of 55 evaluable patients in ZUMA-3, 71% achieved complete remission (CR) or CR with incomplete hematological recovery (CRi).
Adam D. Cohen, MD, on Cilta-Cel for Lenalidomide-Refractory Multiple Myeloma
The director of myeloma immunotherapy at University of Pennsylvania discussed cilta-cel's efficacy in lenalidomide-refractory multiple myeloma.
Applying a Novel Gene Editing Approach to Chronic Hepatitis B
Precision Bio is taking a novel approach to gene editing to deploy a cure for patients with chronic hepatitis B.
Mark Forman, MD, PhD, on Genetic Testing and Further Research in Frontotemporal Dementia
The chief medical officer of Passage Bio discussed research needs in FTD.
CGTLive’s Weekly Rewind – September 2, 2022
Review top news and interview highlights from the week ending September 2, 2022.
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.
Phase 3 Heart Failure Cell Therapy Trial Receives Go-Ahead from DSMB
The risk-benefit assessment determined that there are no significant safety concerns.
Developing a Safer Allogeneic Cell Therapy for Ovarian Cancer
Michael Heffernan, chairman and chief executive officer, Avenge Bio, discussed AVB-001's lower systemic toxicity profile seen in preclinical studies.
Cardiovascular Mesenchymal Cell Therapy Fast Tracked, Soon to Be Patented
Lomecel-B is being evaluated in the phase 2 ELPISII study in children with hypoplastic left heart syndrome.
Around the Helix: Cell and Gene Therapy Company Updates – August 31, 2022
Sangamo’s Gene Therapy Promising for Fabry Disease
Updated data from the STAAR clinical trial were presented at the SSIEM Annual Meeting.
