Victoria Johnson

Myrtelle’s gene therapy previously demonstrated initial efficacy in the disease in February 2022.

New data presented at MDA 2022 showcased positive motor and bulbar function data.

Further results will be presented at the American Urological Association Annual Meeting, May 13-16, 2022.

The company is planning to initiate a phase 1 trial in the second half of 2022.

The therapy’s IND has been cleared and a first in-human trial is expected to begin later this year.

The first patient was dosed in the phase 1/2a trial earlier in March 2022.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

All patients in the first cohort exhibited increases in APOE2 CSF protein expression.

Patients with ulcerative or Crohn colitis showed improvements in clinical and endoscopic scores.

Both patients dosed experienced a stable increase in cerebrospinal fluid HexA activity.

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.

The decision is the latest action in a line of patent interferences over the revolutionary gene-editing technology.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The therapy was well-tolerated in 15 patients and no clinically relevant liver findings were observed.

Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.

Cory Nicholas, PhD, cofounder and chief executive officer, Neurona Therapeutics, discussed the company’s lead program in mesial temporal lobe epilepsy.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The trial design was informed by both patient and physician perspectives.

The pause follows a fatal serious adverse event in the first patient treated in the highest dose cohort.

The trial, sponsored by Mustang Bio, will continue to enroll patients and doses will be escalated to 300 million cells.

The pheNIX clinical hold follows an announcement that BioMarin’s PKU hold may last several quarters.

The therapy has been well-tolerated in the 15 patients treated so far.

The FDA cleared SBT-101's investigational new drug application earlier in February 2022.

The company also announced that the phase 3 study of val-rox has completed enrollment.

Interim data from the SPOTLIGHT study were presented at the ASCO GU Symposium.

Myrtelle’s therapy targets oligodendrocytes to deliver a functional copy of the ASPA gene.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.

Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.

AVROBIO presented data on their analytical pipeline and exploratory studies of molecular follow-up from their lentiviral cell therapy products.