CRISPR Gene Editing for Transthyretin Amyloidosis Safe, Effective
Interim data from a phase 1 study from Intellia Therapeutics and Regeneron Pharmaceuticals is the first to support in vivo CRISPR genome editing in humans.
Promising Outlook in Multiple Myeloma with Cilta-Cel: Philippe Moreau, MD
Philippe Moreau, MD, discussed the CARTITUDE-1 trial's promising results with ciltacabtagene autoleucel read out at the 2021 ASCO Annual Meeting.
Safety Profile of Cilta-Cel: Saad Z. Usmani, MD, MBA, FACP
The director of Clinical Research in Hematologic Malignancies at Levine Cancer Institute discussed adverse events in the CARTITUDE-1 trial.
Optogenetic Monotherapy for Retinitis Pigmentosa IND Approved, Phase 2b Study Enrolling
The announcement follows positive results from the phase 1/2a trial of MCO-010 for RP.
ATA188, a Cell Therapy for Progressive MS: Jakob Dupont, MD, MA
The head of global research and executive vice president of Atara Biotherapeutics discussed the development of ATA188.
Tackling Storage and Logistical Challenges in Cell Therapy
The chief executive officer of Gamida Cell discussed upcoming research on 2 of their investigational agents, GDA-201 and omidubicel, for hematological malignancies.
uniQure Expands Neurological Gene Therapy Pipeline With Epilepsy Program Acquisition
Corlieve Therapeutics’ lead program, AMT-260, previously demonstrated proof-of-concept in preclinical studies of temporal lobe epilepsy.
The Need for Novel Options in High-Risk Mantle Cell Lymphoma
Peter Riedell, MD, discussed the need for more research in mantle cell lymphoma and access to novel treatments, including CAR T-cell therapies.
Harnessing Gene Therapy for Osteoarthritis
Flexion Therapeutics believes that local administration of a gene therapy may not only improve pain management but potentially slow disease progression.
Gene Therapy for Hemophilia B Efficacious, Well-Tolerated
uniQure announced positive results from their phase 3 HOPE-B study of etranacogene dezaparvovec.
Presymptomatic Zolgensma Treatment Yields Age-Appropriate Development in SMA
Newly published data continues to demonstrate Zolgensma's efficacy in presymptomatic and symptomatic SMA Type 1.
Improving Bone Marrow Transplant With Omidubicel
The chief executive officer of Gamida Cell discussed the company's novel approach to cell proliferation.
New Collaboration to Evaluate Lower-Dose Gene Therapy Conditioning for Sickle Cell Disease
Jasper Therapeutics and Aruvant Sciences are studying the use of JSP191 used with ARU-1801 in patients with sickle cell disease.
Car T-Cell Therapy: The Future of Multiple Myeloma Treatment?
C. Ola Landgren, MD, PhD, discussed the role of CAR T-cell therapies in multiple myeloma.
The Potential of CAR T-Cell Therapy for Multiple Myeloma
James Hoffman, MD, discussed the significance of the approval of ide-cel for patients with multiple myeloma.
CAR T-Cell Therapy for Early-Relapsed Multiple Myeloma
Faith E. Davies, MD, discussed the potential of CAR T-cell therapies for the treatment of multiple myeloma.
Longer Follow-Up Shows Cilta-Cel Induces Deep, Durable Responses in Relapsed/Refractory MM
At the 18-month follow-up, the CAR T-cell therapy led to early, deep, and durable responses with a manageable safety profile in patients with relapsed/refractory multiple myeloma.
Broadening Gene Therapy's Reach With Novel Capsids
LogicBio's Mariana Nacht, PhD, shares details of the company's novel capsid discovery platform and gene editing technology.
Gene Therapies for ALS, Friedreich’s Ataxia the Focus of New CRISPR, Capsida Partnership
The news comes after CRISPR’s favorable data readouts in its sickle cell and beta thalassemia studies.
Mesenchymal Stem Cells Hold Promise for Osteoarthritis and More Chronic Diseases
Cynata Therapeutics' Ross Macdonald, PhD, discusses the company's pipeline and clinical-phase trials.
KTE-X19 Safe, Efficacious as Therapy for Pediatric B-Cell ALL in ZUMA-4
Results of the phase 1 portion of ZUMA-4 support continued investigation into the efficacy of KTE-X19 in pediatric patients with B-cell acute lymphoblastic leukemia.
Cilta-cel for the Treatment of Multiple Myeloma: Mounzer Agha, MD
The director of the Mario Lemieux Center for Blood Cancers at UPMC Hillman Cancer Center discussed findings from the CARTITUDE studies.
Targeting Human Retinal Progenitor Cell Injections for Retinitis Pigmentosa
Investigators find approach improved vision and visual fields in some patients.
Axi-cel Shows Efficacy, Durability, in Relapsed/Refractory Follicular Lymphoma
Henry Chi Hang Fung, MD, from the Fox Chase Cancer Center, discusses axi-cel's potential in relapsed/refractory FL.
Electrotransfection: A Different Approach to Plasmid Delivery
The chief scientific officer and chief medical officer at Eyevensys discuss the biotech company's pipeline and approach to gene therapy.
Timrepigene Emparvovec Fails to Demonstrate Efficacy in Choroideremia
Detailed data from the phase 3 trial, including safety findings, which were in line with previous studies, will be presented at a future scientific meeting.
StrataGraft Cellularized Scaffold Approved for Treatment of Deep Partial Thickness Burns
The allogeneic, cellularized scaffold product helps support autologous skin tissue regeneration in patients with thermal burns requiring surgical intervention.
Data Collected in Patients With DLBCL Reveal Real-World Trends for Use of Axi-Cel and Tisa-Cel
Real-world data regarding the use of tisagenlecleucel and axicabtagene ciloleucel CAR T-cell therapy are reported at 2021 EHA.
Working Toward a Cure in Follicular Lymphoma
A panel of lymphoma experts discussed 2 recently approved treatments that are providing hope to patients with relapsed or refractory follicular lymphoma.
Unraveling the Genetics of Glaucoma
Details enhance knowledge of biological pathways that contribute to disease pathogenesis.
