Meaningful Activity Improvements Seen in SMA After Nusinersen
Individual items on RULM revealed meaningful improvements in participants from the SHINE and CHERISH studies.
Giant Axonal Neuropathy Gene Therapy Well-Tolerated With Some Clinical Benefit
New data from a single-site, first-in-human study were presented at the 2023 MDA Conference.
Nusinersen Well-Tolerated After Zolgensma in SMA
Data from the phase 4 RESPOND trial were presented at the 2023 MDA Conference.
Bringing Solid Tumors into the Cell Therapy Space
Alison Betof Warner, MD, PhD, assistant attending physician, Memorial Sloan Kettering Cancer Center, discussed the solid tumor session she chaired at the 2023 Tandem Meetings.
No Time to Pause: Companies Abandon Lagging Cell Therapy Programs
As investigational cell therapies influx the clinical trial landscape, especially for oncologic indications, some companies are ceding the race to their competitors.
Celyad Announces Complete Data from Discontinued CAR in Patients With AML, MDS, MM
The company is back to the drawing board after discontinuing its last clinical-stage program at the end of 2022.
LHON-ND4 Gene Therapy Trial Completes Enrollment
NR082 is being evaluated in a phase 1/2/3 clinical trial in patients with Leber hereditary optical neuropathy.
Patient With Parkinson Disease to Receive Compassionate Use of NK Cell Therapy
SNK01 is primarily being assessed in combination and as a monotherapy for treating solid tumors.
Sarepta May Have a Smooth Road to DMD Gene Therapy Approval
Competitors REGENXBIO and Capricor Therapeutics both have gene therapy candidates in clinical trials.
Gene-Editing Therapy for Hereditary Angioedema Cleared for US Phase 2 Sites
Intellia is also seeking an IND for phase 2 US sites of its other lead candidate, NTLA-2001 for ATTR amyloidosis.
IPSC Therapy Designed to Deliver Full-length Dystrophin Gets Orphan Drug Designation for DMD
The therapy demonstrated an ability to repair injured muscle in mice models of Duchenne.
Upcoming FDA Decisions for Rare Diseases in 2023
CGTLive takes a look at some upcoming FDA decisions for Rare Disease Day.
Gene Therapy Yields Neurodevelopmental Improvements in Children With MPSI
Most treated participants were within 2 SDs of normative mean in acquiring cognition, expressive language and fine motor skills.
MPSII Gene Therapy Shows Some Signs of Durable Efficacy
Interim data from the phase 1/2 CAMPSIITE trial were presented at WORLDSymposium 2023.
Arsa-cel Shows Favorable Risk/Benefit Profile in up to 11 Years of Follow-up Data
Long term data from 2 clinical trials and expanded access programs were presented at WORLDSymposium 2023.
Graphite Bio Drops Lead Sickle Cell Gene-Edited Cell Therapy
The announcement of a “strategic realignment” comes less than 2 months after the company voluntarily paused the CEDAR clinical trial due to a serious, nula-cel-related adverse event.
Increased Motor Development Seen in First 2 Patients With Infantile Krabbe Treated With FBX-101
FBX-101 showed promising efficacy and has been well-tolerated so far in the RESKUE trial.
Hemgenix Approved in EU for Hemophilia B
The gene therapy was approved in the US in the same indication in November 2022.
Gene Therapy Yields 100% 1-Year Survival in LAD-1
No adverse events related to RP-L201 have been reported to date.
Cell Therapy Decreases Viral Load in Patients With BK Viremia After Kidney Transplant
Antiviral responses increased until peaking at week 24.
HHS, CMS to Promote Model for Improved Access to Cell and Gene Therapies, Urge Accelerated Approval Trials to Completion
A third price model to be tested will limit the price of generic drugs for chronic conditions to $2 under Medicare plan D.
BCMA CAR T Therapy Recognized for R/R Multiple Myeloma
CT103A recently demonstrated safety in a phase 1 trial treating participants with NMOSD.
Gene Therapy Continues to be Well-Tolerated in Retinitis Pigmentosa
Encouraging efficacy signals have also been observed in treated participants.
NXC-201 Continues to Show Safety, Efficacy in Relapsed/Refractory Multiple Myeloma
The latest data set includes 22 newly evaluable patients with triple-refractory disease.
GPRC5D-CAR T-cell Therapy Shows Continued Benefit in R/R Multiple Myeloma
The POLARIS study adds to the growing body of evidence validating GPRC5D as a target for CAR T-cell therapy in R/R MM.
First Patient with B-cell Lymphoma Dosed With Allogeneic NK CAR T Therapy
The FDA cleared Century’s IND for CNTY-101 in August 2022.
Dual-Targeting, FasTCAR-T Therapy Cleared for Phase 1b/2 Trial in R/R Multiple Myeloma
GC012F recently showed efficacy in newly-diagnosed MM in investigator-initiated trials.
FDA Blocks 4D’s Gene Therapy Program for Fabry, Clears IND for Diabetic Macular Edema
The clinical hold comes a few weeks after the company announced it was stopping enrollment in the phase 1/2 clinical trial.
Gene Therapy Trial Doses First Patient With Wet AMD in China
The phase 1 clinical trial is the second of Frontera’s trials to begin dosing in 2023.
Remestemcel-L Cell Therapy Makes Comeback With Resubmitted BLA for Pediatric GvHD
Mesoblast is resubmitting its BLA with new CMC, survival, and mechanism of action data on the therapy and phase 3 trial.
