Mesenchymal Stem Cell Therapy IND Cleared for Crohn Disease Fistulas
Another MSC therapy being developed by Mesoblast demonstrated efficacy earlier in March.
Mesenchymal Stem Cell Therapy Improves Joint Function in Rheumatoid Arthritis
Significant decreases were seen in swollen and tender joint measures.
$12 Million Grant Awarded for Hemophilia A Gene Therapy Research
The grant, given to Indiana University School of Medicine, will fund 3 major projects to improve different aspects of hemophilia gene therapy.
Proposed FDA Gene Editing Guidance Emphasizes Long-Term Safety Evaluations
The draft guidance from the Center for Biologics Evaluation and Research outlines safety considerations throughout the clinical development program timeline.
Around the Helix: Cell and Gene Therapy Company Updates – March 16, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
2-year Data for Duchenne Muscular Dystrophy Gene Therapy Shows Durable Improvements
Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.
EMBARK Study Evaluates DMD Gene Therapy
Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.
Adrenomyeloneuropathy Gene Therapy Gets Orphan Drug Designation
The new designation follows an IND clearance and fast track review in February 2022.
First Patient Dosed in T-Cell Malignancy CAR T-Cell Therapy Trial
WU-CART-007 is the first clinical-stage allogeneic CAR therapy for T-cell malignancies in the US.
FDA Recognizes Canavan Disease Gene Therapy With Multiple Designations
Myrtelle’s gene therapy previously demonstrated initial efficacy in the disease in February 2022.
Presymptomatic SMA Treatment With Zolgensma Associated With Normal Development
New data presented at MDA 2022 showcased positive motor and bulbar function data.
Gene Therapy Potential in Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed research with AAV gene therapy in hemophilia.
Urovant Gene Therapy Yields Improvements in Overactive Bladder
Further results will be presented at the American Urological Association Annual Meeting, May 13-16, 2022.
Epstein-Barr Virus and Autoimmune Disease: Jakob Dupont, MD
The global head of research and development at Atara Biotherapeutics discussed the future of MS research.
New Therapies for Age-Related Macular Degeneration
Christina Y. Weng, MD, MBA, Baylor College of Medicine, Houston, discussed therapies for macular degeneration.
Selecta’s Methylmalonic Acidemia Gene Therapy Program Resumes
The company is planning to initiate a phase 1 trial in the second half of 2022.
CGTLive’s Weekly Rewind – March 11, 2022
Review top news and interview highlights from the week ending March 11, 2022.
Myasthenia Gravis Cell Therapy Granted Fast Track Designation
The therapy’s IND has been cleared and a first in-human trial is expected to begin later this year.
Performing In-Depth Analysis of Lentiviral Cell Therapies: Luca Biasco, PhD
The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.
Intellia’s CRISPR-Engineered Cell Therapy Receives Orphan Drug Designation for Acute Myeloid Leukemia
The first patient was dosed in the phase 1/2a trial earlier in March 2022.
Mesenchymal Stem Cell Therapy Reduces NfL in Progressive MS
Patients demonstrated a median reduction of 63.5% in CSF NfL levels 6 months after treatment.
Cautious Optimism for GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.
Around the Helix: Cell and Gene Therapy Company Updates – March 9, 2022
LEXEO Gene Therapy Decreases Alzheimer Disease Biomarkers
All patients in the first cohort exhibited increases in APOE2 CSF protein expression.
Global Awareness, Diagnosis a Priority for Rare Diseases: Michael Parini, JD
The chief executive officer and director of Freeline Therapeutics discussed the importance of Rare Disease Day.
Mesenchymal Stromal Cell Therapy Yields Improvements in Colitis
Patients with ulcerative or Crohn colitis showed improvements in clinical and endoscopic scores.
Advantages of Invariant Natural Killer T Cell Therapies
Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed the company’s iNKT cell platform.
First In-Human Gene Therapy for Tay Sachs Disease Demonstrates Feasibility
Both patients dosed experienced a stable increase in cerebrospinal fluid HexA activity.
Working to Find a Cure for Charcot-Marie-Tooth: Keith Fargo, PhD
The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission.
Bispecific Antibodies Effective After CAR T-Cell Relapse in Myeloma?
Oliver Van Oekelen, MD, MSc, resident and PhD student, Icahn School of Medicine, Mount Sinai, discussed findings from a retrospective study.
