Fidanacogene Elaparvovec Well-Tolerated in Hemophilia B
No concerning effects to liver health were observed in the phase 1/2a study.
FDA Lifts Clinical Hold on Novartis SMA Gene Therapy
The company announced plans to initiate the STEER study of intrathecal OAV-101 for older patients with SMA type 2.
Hemophilia B Gene Therapy AMT-060 Shows Durable Effect
Phase 3 studies are continuing in the enhanced Padua FIX variant AMT-061 for hemophilia B.
Gene Therapies for Batten Disease, ALS Receive FDA Designations
The designations allow for expedited drug development and review of the investigational gene therapies for CLN2 Batten disease and SOD1 ALS.
MRI Data Supports Gene Therapy for Sanfilippo Syndrome Type A
Abeona Therapeutics is seeking a path towards BLA filing based on the promising clinical data.
Skysona Gene Therapy Approved in EU for Early CALD
Bluebird Bio plans to submit its biologics license application in the US by mid 2021.
Valoctocogene Roxaparvovec’s Treatment Durability in Hemophilia A in Question
A drop in endogenous Factor VIII expression was observed from treatment to 5-year follow-up despite continued demonstration of efficacy.
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.