NK Cell Therapy/Rituximab Combination Fast Tracked for B-cell Non-Hodgkin Lymphoma
AB-101 is also being investigated with the innate cell engager AFM13 for CD30 lymphomas.
Multi-characteristic Opsin Gene Therapy Gets Second Fast Track for Retinal Degenerative Diseases
MCO-010 is in phase 2 clinical trials for treating retinitis pigmentosa and Stargardt disease.
Allogeneic CAR T Cleared for B-cell Malignancy Trials
Sana Biotechnology also plans to submit another IND for the same indications for a CD22-targeted CAR T later in 2022.
Advanced Colorectal Cancer CAR T Cleared for Trial
The phase 1 portion of the trial assessing CNA3103 will start enrollment in Australia in the first half of 2023; the phase 2 portion will expand to the US.
Discogenic Progenitor Cell Therapy Shows Improvements in Lumbar Degenerative Disc Disease, Recognized by FDA
The DGX-A01 study met its primary safety and efficacy endpoints in its highest dose cohort of IDCT.
New Data Supports Breyanzi’s Expansion to Chronic Lymphocytic Leukemia
Breyanzi is currently approved for the second-line or later treatment of large B-cell lymphomas.
Lifileucel Creeps Closer to Clinic for Melanoma, NSCLC
Iovance is on track to complete its rolling BLA submission for lifileucel in the post-anti-PD1 melanoma indication in the first quarter of 2023.
Logic-gated CAR T Therapy Cleared for Lymphoma Trials
The CD19/CD20 dual-targeted CAR is currently under investigation in an investigator-initiated study in UCLA.
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.
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