OpRegen Shows Sustained Efficacy in Treating Dry AMD
Three patients continue to show improvements in BCVA 3 years post-treatment.
Celyad Announces Positive Data and Updates for CAR T Candidates
The phase 1b KEYNOTE-B79 trial of CYAD-101 for mCRC is expected to initiate in Q4 2021.
Valoctocogene Roxaparvovec Efficacious in Treating Hemophilia A
Over 90% participants had an annualized bleeding rate of 0 or a lower bleed rate than baseline after week 4 after treatment.
Prime Medicine Takes Precise Approach to Gene Editing With “Find and Replace” Technology
The precision gene editing technology is designed to minimize off-target genetic alterations.
10 Gene and Cell Therapy Companies to Watch
With advancements happening at unprecedented rates, these are the cell and gene therapy companies and pipelines we’re keeping a close eye on.
Celularity Cements Focus on Placenta-Derived Cell Therapies
The clinical-stage biotech company recently announced the expansion of their phase 1 trial of CYNK-001 in patients with relapsed/refractory acute myeloid leukemia.
New Collaboration to Assess Stem Cell Therapy With Bispecific Antibody in AML
Janssen hopes that Vor’s stem cell therapy will allow their bispecific antibody to be better-tolerated.
Trends in Medical Research for Gene and Cell Therapies
The CEO of Avamab Pharma discussed how funding and research changes directions.
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.
