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Significant decreases were seen in swollen and tender joint measures.

The grant, given to Indiana University School of Medicine, will fund 3 major projects to improve different aspects of hemophilia gene therapy.

The draft guidance from the Center for Biologics Evaluation and Research outlines safety considerations throughout the clinical development program timeline.

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

Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.

Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.

The new designation follows an IND clearance and fast track review in February 2022.

WU-CART-007 is the first clinical-stage allogeneic CAR therapy for T-cell malignancies in the US.

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

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed research with AAV gene therapy in hemophilia.

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

The global head of research and development at Atara Biotherapeutics discussed the future of MS research.

Christina Y. Weng, MD, MBA, Baylor College of Medicine, Houston, discussed therapies for macular degeneration.

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

Review top news and interview highlights from the week ending March 11, 2022.

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

The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.

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

Patients demonstrated a median reduction of 63.5% in CSF NfL levels 6 months after treatment.

The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.

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.

The chief executive officer and director of Freeline Therapeutics discussed the importance of Rare Disease Day.

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

Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed the company’s iNKT cell platform.

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

The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission.

Oliver Van Oekelen, MD, MSc, resident and PhD student, Icahn School of Medicine, Mount Sinai, discussed findings from a retrospective study.