Dual Receptor Modular CAR T-Cell Therapy Shows Preclinical Efficacy
A2 Biotherapeutics is developing CEA- and MSLN- targeted Tmod CAR T-cell therapies.
Around the Helix: Cell and Gene Therapy Company Updates – March 23, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Lineage Cell Therapeutics to Develop Cell Therapy for Hearing Loss
The company’s lead cell therapy candidate, OpRegen, has demonstrated efficacy in dry AMD.
Val-Rox Gene Therapy Restores Factor VIII Production and Reduces Bleeding in Hemophilia A
Complete results from the phase 3 GENEr8 study were published in the New England Journal of Medicine.
Huntington Disease Gene Therapy AMT-130 Completes Trial Enrollment
A sister study of AMT-130 in Europe dosed its first patients in February.
SQZ Biotech Awarded $2 Million to Develop Parkinson Disease Cell Therapy
The company recently received IND clearance for a solid tumor cell therapy.
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.
Around the Helix: Cell and Gene Therapy Company Updates – March 16, 2022
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.
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.
Urovant Gene Therapy Yields Improvements in Overactive Bladder
Further results will be presented at the American Urological Association Annual Meeting, May 13-16, 2022.
Selecta’s Methylmalonic Acidemia Gene Therapy Program Resumes
The company is planning to initiate a phase 1 trial in the second half of 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.
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.
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.
Mesenchymal Stromal Cell Therapy Yields Improvements in Colitis
Patients with ulcerative or Crohn colitis showed improvements in clinical and endoscopic scores.
First In-Human Gene Therapy for Tay Sachs Disease Demonstrates Feasibility
Both patients dosed experienced a stable increase in cerebrospinal fluid HexA activity.
New Findings May Help Bolster CRISPR/Cas9 Accuracy
David Taylor, PhD, an assistant professor in the department of molecular biosciences at UT Austin, discussed the Cas9 research conducted between 2 labs at UT Austin.
CRISPR Patents Decision Favors Harvard and MIT’s Broad Institute
The decision is the latest action in a line of patent interferences over the revolutionary gene-editing technology.
Around the Helix: Cell and Gene Therapy Company Updates – March 2, 2022
Latest Results in ATTR Amyloidosis CRISPR Trial Bolsters Potential of In Vivo Gene Editing
The therapy was well-tolerated in 15 patients and no clinically relevant liver findings were observed.
Advancing Treatments for Rare Diseases Requires a Team Approach
Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.
Exploring Cell Therapy for the Treatment of Drug-Resistant Epilepsy
Cory Nicholas, PhD, cofounder and chief executive officer, Neurona Therapeutics, discussed the company’s lead program in mesial temporal lobe epilepsy.
Around the Helix: Cell and Gene Therapy Company Updates - February 23, 2022