Phase 1/2 Study of SIG-001 for Hemophilia A on Hold Following Serious Adverse Event
Study enrollment has been paused following the development of antibodies in the highest-dosed patient.
Searchable Genetic Database Links Variants to Human Health and Disease
Abbvie, Biogen, and Pfizer have collaborated to launch the free resource that hosts genetic sequencing data from hundreds of thousands of participants.
EB-101 Cell Therapy Continues to Show Efficacy in Recessive Dystrophic Epidermolysis Bullosa
The phase 3 VITAL study, which is currently enrolling, will evaluate the investigational cell therapy agent further.
Lumevoq Improves Visual Acuity in LHON Vision Loss
Investigators found that bilaterally treated participants experienced greater improvements in BCVA than unilaterally treated participants.
CAR-NK Cells Show Potential in Solid Tumor Immunotherapy
The engineered natural killer cells lack the off-target effects of typical CAR T-cell therapy and similarly lack any concerning safety signals.
Next-Generation CAR T-Cell Therapy, ALLO-605, Granted Fast Track Designation
The FDA’s designation follows positive data presented at the 2020 ASH meeting.
Key Themes From the 2021 ASGCT Meeting
The CEO of Avamab Pharma discussed how the field of gene therapy has shifted due to the COVID-19 pandemic.
Omidubicel Shows Efficacy Over Standard Umbilical Cord Blood Transplant in Hematologic Malignancies
Patients receiving omidubicel also spent less time in hospital following transplant.
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.
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.
Gene Therapy for Hemophilia B Efficacious, Well-Tolerated
uniQure announced positive results from their phase 3 HOPE-B study of etranacogene dezaparvovec.
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.
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.
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.
DA01 Dopaminergic Neurons and GDNF Gene Therapy Studies Underway for Parkinson Disease
Bayer provided an update on trials currently underway for the treatment of PD by its subsidiaries.
Gene Editing Trial Underway in Pediatric Methylmalonic Acidemia
The therapy utilizes LogicBio’s GeneRide technology to insert a corrective copy of the target gene via synthetic AAV vectors.
Duchenne Muscular Dystrophy Gene Therapy SGT-001 Continues to Show Efficacy, Safety Following Protocol Amendment
Participants in both the low- and high-dose cohorts showed improved functional outcomes at 1-year post-dose.
Biogen, Capsigen Partner on Novel AAV Capsid Development for Neurologic Disorders
The partnership will allow for the development of disease-specific AAV vectors that will serve Biogen's current gene therapy pipeline.
FAST Grant Enables Creation of Angelman Syndrome Hotline
Recent Foundation for Angelman Syndrome Therapeutics grants funded the hotline as well as work to develop cell line models for rarer genotypes of AS.
Investigating Stem Cell Therapy in TBI and Other Conditions
Bijan Nejadnik, MD, chief medical officer, SanBio, also thanked neurological experts that collaborated on the phase 2 STEMTRA trial.
SB623 Stem Cell Therapy to Regain Motor Function in TBI
Masahito Kawabori, MD, PhD, associate professor, Hokkaido University, discussed results of the phase 2 STEMTRA trial.
Brain Neurotherapy Bio Brings Clinical Programs for Parkinson and MSA to AskBio
Both therapeutic programs utilize AAD2-GDNF gene therapy targeted to brain structures vulnerable to Parkinson disease and multiple system atrophy.
SB623 Mesenchymal Stem Cell Implantation Efficacious and Well-Tolerated in TBI
There was no statistically significant difference between treatment-related adverse events in the SB623 stem cell and control groups.
SOD1 Variants Show Significant Association with ALS Risk
Researchers from the University of Turin also found that an NEFH variant showed a protective association with ALS.
Initial Results Prove Positive for Low-Dose AXO-AAV-GM1 in GM1 Gangliosidosis
These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.
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