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
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.
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.
CGTLive’s Weekly Rewind – March 4, 2022
Review top news and interview highlights from the week ending March 4, 2022.
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.
Improving Rare Disease Awareness: Bruce Dezube, MD
The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.
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.
Determining Order of CAR T Therapy: Elias Jabbour, MD; Jae Park, MD
The oncologists from MD Anderson and Memorial Sloan Kettering Cancer Centers discuss sequencing CAR T-cell therapies and other key therapies in patients with B-cell acute lymphoblastic leukemia.
New Genetic Target May Hold Promise in Treating Blindness
The SARM1 gene is a key driver in the damage that ultimately leads to impaired vision.
Around the Helix: Cell and Gene Therapy Company Updates – March 2, 2022
Assessing Efficacy of Fibroblast Cell Therapy in MS: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.
A Positive Outlook for Rare Disease Research: Michael Singer, MD, PhD
The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.
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.
Cilta-Cel Approved for Relapsed/Refractory Multiple Myeloma
The CAR T-cell therapy from Janssen and Legend Biotech is the second to receive approval with an indication for treatment of multiple myeloma.
Improving Rare Disease Awareness: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.
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.
Developing Therapies for Rare Diseases: Kinnari Patel, PharmD
The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.
