Around the Helix: Cell and Gene Therapy Company Updates – March 9, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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
Patient-Informed Study to Evaluate Hunter Syndrome Gene Therapy
The trial design was informed by both patient and physician perspectives.
Enrollment Pauses in Mesothelioma CAR T-Cell Therapy Trial
The pause follows a fatal serious adverse event in the first patient treated in the highest dose cohort.
CAR T-Cell Therapy Demonstrates Feasibility in Resistant Prostate Cancer
The trial, sponsored by Mustang Bio, will continue to enroll patients and doses will be escalated to 300 million cells.
Second Phenylketonuria Gene Therapy Study Put on Clinical Hold
The pheNIX clinical hold follows an announcement that BioMarin’s PKU hold may last several quarters.
Gene Therapy for Diabetic Retinopathy Yields Continued Improvements
The therapy has been well-tolerated in the 15 patients treated so far.
Adrenomyeloneuropathy Gene Therapy Granted Fast Track Review
The FDA cleared SBT-101's investigational new drug application earlier in February 2022.
BioMarin’s PKU Gene Therapy On Hold Pending New Studies
The company also announced that the phase 3 study of val-rox has completed enrollment.
Memory T Cell CAR T Therapy Showcases Anti-Tumor Activity in Resistant Prostate Cancer
Interim data from the SPOTLIGHT study were presented at the ASCO GU Symposium.
Gene Therapy Shows Potential in Canavan Disease
Myrtelle’s therapy targets oligodendrocytes to deliver a functional copy of the ASPA gene.
Around the Helix: Cell and Gene Therapy Company Updates - February 16, 2022
Lysogene Gene Therapies Show Promise in GM1 Gangliosidosis and MPSIIIA
Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.
First In-Human Trial to Assess Krabbe Disease Gene Therapy
Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.
Molecular Analysis Elucidates Cell Therapy Destiny and Distribution
AVROBIO presented data on their analytical pipeline and exploratory studies of molecular follow-up from their lentiviral cell therapy products.
Gene Therapy Stabilizes Disease Progression in GM1 Gangliosidosis
Positive results from a phase 1/2 study of AXO-AAV-GM1 were presented at WORLDSymposium.
CNGB3 Achromatopsia Gene Therapy Improves Visual Sensitivity
Clinical development will be stopped on the CNGA3 achromatopsia program as no clinical improvements were seen.
Around the Helix: Cell and Gene Therapy Company Updates - February 9, 2022
Gene Therapies for Mucopolysaccharidosis Show Safety, Efficacy
Interim data from trials of RGX-121 and RGX-111 were presented at WORLDSymposium 2022.
Study to Evaluate Gene-Edited Cell Therapy for MPSII
The LV-HSCGT therapy has demonstrated efficacy in preclinical studies in mouse models.
Beti-Cel Yields Durable Transfusion Independence in β-Thalassemia
The gene therapy is currently under FDA review for the treatment of β-thalassemia across genotypes.