Kyowa Kirin, Inc.

Ultragenyx Pharmaceutical appointed Eric Olson as Chief Business Officer and Executive Vice President effective September 22, 2025, following Thomas Kassberg's planned retirement after 14 years with the company.

UK-based Charm Therapeutics secured $80 million in Series B funding to advance its next-generation menin inhibitor for acute myeloid leukemia into Phase I clinical trials starting Q1 2026.

Kura Oncology's lead asset ziftomenib has advanced to FDA Priority Review for relapsed/refractory NPM1-mutant acute myeloid leukemia, with a PDUFA date of November 30, 2025.

Kyowa Kirin will present results from the Phase 3 ROCKET IGNITE trial of rocatinlimab, an investigational anti-OX40 monoclonal antibody for moderate-to-severe atopic dermatitis, at the EADV 2025 Annual Meeting in Paris.

Orchard Therapeutics has completed enrollment in the HURCULES registrational trial for OTL-203, an investigational hematopoietic stem cell gene therapy for MPS-IH (Hurler syndrome), nearly one year ahead of schedule.

Four innovative therapies are advancing through clinical trials to address critical unmet needs in MPS I treatment, particularly targeting central nervous system complications that current therapy Aldurazyme cannot reach.

The FDA has accepted Kura Oncology's new drug application for ziftomenib, an oral menin inhibitor for treating relapsed or refractory acute myeloid leukemia with NPM1 mutations.

• Lenmeldy (atidarsagene autotemcel), Orchard Therapeutics' hematopoietic stem cell gene therapy, demonstrated significant extension of life expectancy and preservation of motor and cognitive function in children with early-onset metachromatic leukodystrophy. • The New England Journal of Medicine publication details over 12 years of follow-up data with more than 250 patient-years of experience, showing durable efficacy in pre-symptomatic and early-symptomatic patients. • Orchard Therapeutics will present six studies at the upcoming American Society of Gene and Cell Therapy meeting, highlighting their expanding portfolio of hematopoietic stem cell gene therapies for rare neurometabolic diseases.

The global Adult T-cell Leukemia-Lymphoma (ATLL) market is projected to experience significant growth through 2035, driven by increasing HTLV-1 infections and advancements in immunotherapy technologies.

• Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) has become the first FDA-approved treatment for metachromatic leukodystrophy (MLD), a rare genetic disorder that causes rapid neurological deterioration in children. • At $4.25 million per one-time treatment, Lenmeldy now holds the title of the most expensive medicine in the United States, slightly higher than its European price of $3.7 million where it's marketed as Libmeldy. • Clinical trials showed remarkable results with all 37 treated children surviving to age six (compared to 58% in control groups), with 71% able to walk independently and 85% maintaining normal language and cognitive function.