Bluebird Bio Slashes Workforce as It Awaits Key FDA Gene Therapy Decisions
The biotechnology company previously pulled out of Europe after encountering payor challenges for their gene therapies.
Cell Therapy Well-Tolerated in Severe COVID-19
The same therapy by Capricor Therapeutics has demonstrated efficacy in Duchenne muscular dystrophy.
Preclinical Data Show Promise for Adrenomyeloneuropathy Gene Therapy
The latest data build on previous research that showed on-target effects analyzed across multiple measurements and a favorable safety profile.
FDA Grants ASC618 Fast Track Designation for Hemophilia A
The gene therapy ASC618 has received European and US designations and opinions that could help expedite development for hemophilia A.
FDA Approves Second-Line Axicabtagene Ciloleucel for LBCL
The FDA has approved second-line axicabtagene ciloleucel as a treatment for adult patients with large B-cell lymphoma following frontline chemoimmunotherapy.
CGTLive’s Weekly Rewind – April 1, 2022
Review top news and interview highlights from the week ending April 1, 2022.
B-VEC Gene Therapy Heals Recessive Dystrophic Epidermolysis Bullosa Wounds
Positive interim results from a phase 3 study presented at AAD 2022 bolster recently published phase 2 data.
Around the Helix: Cell and Gene Therapy Company Updates – March 30, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Renal Cell Carcinoma CAR T-Cell Therapy Fast Tracked
Allogene’s clinical hold on their AlloCAR T candidates was previously lifted in January 2022.
CAR T-Cell Expansion Indicative of Response in B-cell Lymphoma
Research presented at the EBMT meeting suggests cell expansion may be a good biomarker of both response and outcomes.
CGTLive’s Weekly Rewind – March 25, 2022
Review top news and interview highlights from the week ending March 25, 2022.
Liso-cel Shows Survival Benefit in R/R Large B-Cell Lymphoma
A prespecified interim analysis from the phase 3 TRANSFORM study showed an event-free survival benefit with the second-line therapy.
Dual Receptor Modular CAR T-Cell Therapy Shows Preclinical Efficacy
A2 Biotherapeutics is developing CEA- and MSLN- targeted Tmod CAR T-cell therapies.
Improving Speed and Fidelity of CRISPR/Cas9: Kenneth Johnson, PhD, and David Taylor, PhD
The professors and researchers from The University of Texas at Austin discussed new findings of Cas9 structure and mechanisms.
Around the Helix: Cell and Gene Therapy Company Updates – March 23, 2022
CAR T-Cell Therapy Promising in R/R Chronic Lymphocytic Leukemia
Seven patients from the trial continued to be followed-up as of the data cut-off date.
Combining RNA and CAR T-Cell Therapy for Myasthenia Gravis
Michael Singer, MD, PhD, cofounder and chief scientific officer of Cartesian Therapeutics, discussed the company’s lead candidate, Descartes-08.
Lineage Cell Therapeutics to Develop Cell Therapy for Hearing Loss
The company’s lead cell therapy candidate, OpRegen, has demonstrated efficacy in dry AMD.
Val-Rox Gene Therapy Restores Factor VIII Production and Reduces Bleeding in Hemophilia A
Complete results from the phase 3 GENEr8 study were published in the New England Journal of Medicine.
Research Needs in Pompe Disease: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed unmet needs that remain in Pompe disease.
Huntington Disease Gene Therapy AMT-130 Completes Trial Enrollment
A sister study of AMT-130 in Europe dosed its first patients in February.
Gene Therapy for Rare Diseases: Latest Updates
Kinnari Patel, PharmD, president and chief operating officer of Rocket Pharmaceuticals, discussed Rocket Pharma’s programs.
SQZ Biotech Awarded $2 Million to Develop Parkinson Disease Cell Therapy
The company recently received IND clearance for a solid tumor cell therapy.
Neutralizing Antibody Versus Total Antibody Assays in Gene Therapy Development: Key Considerations for Assessing Immunogenicity
Viral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective.
Improving Diagnosis in Rare Diseases: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.
Adjuvant Olaparib Approved for BRCA+, High-Risk Early Breast Cancer
Olaparib is now the first early-line therapy targeting BRCA-mutations in breast cancer.
Improving Representation in Clinical Trials: James Beck, PhD
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.
T-Cell Therapy Gets Orphan Drug Designation for AML
Half the patients in the high-dose cohort of a phase 1a/2b trial achieved complete responses.
CGTLive’s Weekly Rewind – March 18, 2022
Review top news and interview highlights from the week ending March 18, 2022.
Addressing AAV Antibodies in Hemophilia B Gene Therapy: Wolfgang Miesbach, MD, PhD
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study.
