A2 Bio’s Screening Study Identifies Eligible Patients for Treatment With Tmod CAR-T
The BASECAMP-1 screening study has now identified 28 patients whose solid tumors are positive for HLA-A*02 LOH.
Setting the Stage for Engineered B-Cell Therapies in Cancer and Autoimmune Disease
David Rawlings, MD, the director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute, discussed findings from several preclinical studies that could help push engineered B-cell therapies towards clinical trials.
Thomas McCauley, PhD, on Continuing Evaluations on OTX-2002 for MYC-Expressing Cancers
The chief scientific officer at Omega Therapeutics discussed milestones Omega Therapeutics is looking forward to in the next year.
Tenaya's Phase 1b MyPeak-1 Trial is Testing the Waters in Gene Therapy for Hypertrophic Cardiomyopathy
TN-201 uses an AAV vector-based approach to deliver a functional copy of the disease-targeted MYBPC3 gene to cardiomyocytes.
T-Cell Engagers Show Potential in HPV16+ and WT1+ Cancers
Patients with HNSCC had a median overall survival (OS) of 20.8 months and a 12-month OS rate of 59%.
University of Manchester Launches MPS II Gene Therapy Trial, Picking Up Where AVROBIO Left Off
UoM was previously collaborating on the development of AVR-RD-05 with AVROBIO, but regained the license to the HSC gene therapy following the company’s cessation of development on all programs.
Ahmad Masri, MD, MS, on the Potential of Cell Therapy in Light Chain Amyloidosis
For World Amyloidosis Day, the cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University discussed how Nexcella’s NXC-201 could help address unmet needs.
mRNA/LNP CAR-M Shows Promise for Treating Solid Tumors in Preclinical Research
In terms of safety, it was noted that repeated administration of CAR mRNA/LNP was well-tolerated by mice.
CGTLive’s Weekly Rewind – November 3, 2023
Review top news and interview highlights from the week ending Novmber 3, 2023.
The Future of Dermatology? B-VEC's Impact in DEB 6 Months Post Approval
CGTLive spoke to experts in the field about their experiences and impressions of B-VEC almost half a year after its approval for dystrophic epidermolysis bullosa.
Orchard Therapeutics’ Gene Therapy OTL-203 Stabilizes or Improves Sensory Symptoms in Patients With MPS-IH
Hearing function was found to be normal for 4 of the patients (50%) at their most recent follow-up.
Stella Vnook, PharmD, on Improving Cell Therapy Delivery to Solid Tumors With Core Shell Spherification
The chief executive officer of Likarda discussed the company’s CSS technology and how it may reduce off-tissue effects.
Limb-Girdle Muscular Dystrophy Gene Therapy Trial Set to Enroll Second Cohort Based on Initial Positive Results
The DSMB has cleared Atamyo to go forward with a planned dose of ATA-100 for the second cohort that is 3 times higher than the dose used for the first cohort.
Around the Helix: Cell and Gene Therapy Company Updates – November 1, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CLDN6-Targeted CAR Shows Some Signs of Activity in Solid Tumors
CARVac RNA vaccine may amplify the persistence of the BNT211 CAR T-cell therapy.
FDA AdComm Favorable Towards Vertex and CRISPR’s Sickle Cell Gene-Edited Cell Therapy, but Recommends Postmarketing Research
The members of the AdComm were generally of the opinion that the off-target analyses conducted by Vertex were reasonably robust.
CRISPR Therapy Well Tolerated, Detectable in First 3 Participants With HIV
Excision plans to escalate the trial of EBT-101 to the second dose level in the fourth quarter of 2023.
ELEVIDYS Study Fails Primary End Point in DMD, Secondary End Points Show Statistically Significant Benefit
Despite the announcement, CEO Doug Ingram shared that the company is seeking a wider approval for delandistrogene moxeparvovec-rokl regardless of age or ambulatory status.
Auditory Improvements Seen in First Child Treated With OTOF Hearing Loss Gene Therapy
Other efforts to develop a gene therapy for OTOF congenital hearing loss include programs from Sensorion and Eli Lilly.
Maria Pia Morelli, MD, PhD, on Considerations for Cell Therapy in GI Cancers
The assistant professor at MD Anderson Cancer Center discussed factors to consider when trying to treat patients with gastrointestinal cancers with cell therapy.
Considerations for Relapsed/Refractory Follicular Lymphoma CAR T-Cell Therapies and Bispecific Antibodies
Patient preferences should be considered when selecting the optimal treatment regimen for patients with relapsed/refractory follicular lymphoma, as both CD19-directed CAR T-cell therapies and CD20-targeted bispecific antibodies can be efficacious.
FDA Staff Call for Further Study Considerations for Exa-Cel Ahead of Decision in Sickle Cell
The therapy’s PDUFA date is scheduled for December 8, 2023, and an Advisory Committee is set to meet on October 31, 2023, to discuss the treatment’s potential approval for sickle cell disease.
Brett Kopelan, MA, on the Future Treatment of Epidermolysis Bullosa With Advanced Therapeutics
In observance of Epidermolysis Bullosa Awareness Week, the executive director of debra of America discussed how a combination of advanced and topical approaches may represent the future of EB treatment.
Tenaya’s ARV Cardiomyopathy Gene Therapy TN-401 Cleared for US Trial
In light of the IND clearance, Tenaya now intends to go forward with plans for a phase 1b clinical trial (RIDGE-1) that will seek to recruit up to 15 adult patients.
Islet Cell Pouch Implantation Shows Promise in Type 1 Diabetes
One patient had their Cell Pouch removed after the donor islet cells were found to be contaminated with Candida albicans yeast.
CGTLive’s Weekly Rewind – October 27, 2023
Review top news and interview highlights from the week ending October 27, 2023.
Caribou's Allogeneic CAR-T CB-012 Cleared for US Trial in R/R AML
In light of the IND clearance, the company is planning to carry out plans for a phase 1 clinical trial (AMpLify) for adult patients with r/r AML.
Mustang Bio’s CAR/Oncolytic Virus Combo Cleared for Glioblastoma/Astrocytoma Trial
The combo, termed MB-109, consists of the MB-108 oncolytic virus and MB-101 CAR T-cell therapy.
Ahmad Masri, MD, MS, on the Potential of Gene Therapy in ATTR Amyloidosis
In observance of World Amyloidosis Day, the cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University discussed the changing landscape of care in this field.
Gaucher Disease Type 1 Gene Therapy Shows Promising Initial Data
The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.
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