TScan Set to Evaluate Multiplex TCR T Therapies for Solid Tumors
The FAD has granted primary IND clearance to the company’s T-Plex program and 2 initial TCR-T therapies.
Rett Syndrome Gene Therapy Cleared for Clinical Trial
NGN-401 is Neurogene’s second investigational gene therapy to enter clinical trials.
10 Cell and Gene Therapies to Watch in 2023
With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.
Late-Onset Pompe Disease Gene Therapy Cleared to Continue Trial
The FDA has lifted a clinical hold placed in the summer of 2022 due to a mild but medically significant case of peripheral sensory neuropathy.
Editas Sells off iNK Preclinical Cell Therapy Program, Gene-Editing Technologies to Shoreline
The company is reprioritizing to focus on its clinical stage programs, which ran into a number of setbacks in 2022.
Gene Therapy Improves Visual Acuity in MT-ND4 LHON Compared to Natural History
Investigators analyzed data from 3 phase 3 studies and a long-term follow-up study.
Hemgenix “Transformative” for Patients With Hemophilia B
Steven Pipe, MD, CS Mott Children’s Hospital, discussed the latest follow-up data from the HOPE-B study of the approved therapy, Hemgenix.
Neovascular AMD Gene Therapy Cleared for Trials
A phase 1 clinical trial of EXG102-031 is expected to initiate in the first quarter of 2023.
Advocating for Patients With Chronic Lymphocytic Leukemia
Brian Koffman, MDCM, DCFP, FCFP, DABFP, MSEd, Co-Founder, Executive Vice President, and Chief Medical Officer, CLL Society, discussed the role the society plays in the field.
Cell Therapy Development Advances in Hemorrhagic Trauma, Acute Ischemic Stroke
Athersys has made progress in enrollment with its MATRICS-1 and MASTERS-2 clinical trials.
Second Generation MUC1-CAR T Therapy Cleared for Solid Tumor Trial
huMNC2-CAR22 is designed to avoid off-tissue expression and reduce T-cell exhaustion.
Val-rox Meets End Points in Hemophilia A at 3 Years
The new analysis data was requested by the FDA ahead of the therapy’s March 31, 2023 PDUFA date.
Celularity’s MSC Platform Shows Clinical Benefit for Crohn Disease in Legacy Analyses
Phase 1/2 studies evaluated PDA001 in 50 participants with CD.
First Patient Dosed in Treg Cell Therapy Trial for Myelofibrosis
CKO804 is being evaluated as an add-on therapy to ruxolitinib in patients whose disease had a suboptimal response to ruxolitinib alone.
Intellia to Advance Development of ATTR Amyloidosis, Angioedema, AATD Gene Therapies
The company is kicking off 2023 by announcing planned advancements for several gene therapies in its pipeline.
Cellectis Shifts to In-House Manufacturing With UCART22
The BALLI-01 study, initiated in 2019, has now dosed its first patient with an in-house manufactured CAR T-cell therapy candidate.
Gene Therapies Advance in Danon Disease, CLN2 Batten Disease
Both REGENXBIO and Rocket Pharmaceuticals have announced trial updates in their gene therapy programs for rare diseases.
Solid Tumor CAR T Collaboration Cleared for Trials
Invectys and CTMC, a joint venture between MD Anderson Cancer Center and National Resilience, announced the therapy and collaboration in June 2022.
Ide-cel Shows Efficacy in Early Relapsed Multiple Myeloma
Patients had an ORR of 83.8% with a median DOR of 15.7 months in data presented at the ASH 2022 meeting.
Running With Scissors: The FDA Moves to Tame Gene Therapy Drug Development
With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.
B-VEC Continues to Show Durable Wound Healing in Dystrophic Epidermolysis Bullosa
Half of wounds treated with the gene therapy had complete healing at both months 3 and 6 compared to 7% of placebo-treated wounds.
GPRC5D-Targeted CAR T-Cell Therapy Shows Promising Efficacy After BCMA Therapy in R/R MM
New data from a phase 1/2 trial of BMS-986393 were presented at the 64th Annual ASH Meeting.
SPK-8011 Yields Durable FVIII Expression in Hemophilia A
Annualized bleed rate and FVIII infusion rates significantly decreased after infusion with the gene therapy.
Gene Therapy for Sickle Cell Disease Cost Effective in Reducing Health Disparities
A DCE analysis weighed gender-, age-, and disease-severity-specific annual costs as opposed to traditional cost-effectiveness models.
Neoantigen Reactive T Cell Therapy Shows Early Signs of Efficacy in NSCLC and Melanoma
Achilles Therapeutics presented updated data from CHIRON and THETIS at the 2022 ESMO immuno-oncology congress.
Stem Cell Gene Therapy Show Clinical Improvements in Gaucher Type 1 and 3
AVROBIO plans to initiate a global phase 2/3 trial of AVR-RD-02 in GD3 in the second half of 2023.
BioCardia Follows up CardiAMP With CardiALLO Clearance for Heart Failure
The allogeneic mesenchymal stem cell therapy trial will enroll patients ineligible for treatment with the autologous CardiAMP.
Genome-Edited Cell Therapy Reduces VOEs in Sickle Cell Disease
The first 2 participants dosed are free of vaso-occlusive events as of 5 and 1.5 months of follow-up after treatment with EDIT-301.
Leveraging Cell Therapy Advances for Treating Hodgkin Lymphoma, Other Cancers
Ivan Horak, MD, discussed Tessa’s cell therapy platforms and the ACTION clinical trial.
Neural Cell Therapy Reduce Seizures in MTLE
Updated data were presented at the AES annual meeting in Nashville.
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