NATIONWIDE CHILDREN'S HOSPITAL

Andelyn Biosciences Delivers Breakthrough Gene Therapy for Ultra-Rare NEDAMSS Disease in Record Time

10 days ago

• Andelyn Biosciences has successfully manufactured a novel viral vector gene therapy using its AAV Curator Platform to treat a baby with NEDAMSS, an ultra-rare neurodegenerative disorder with no known treatments. • The therapy was produced and released in just 10 months from development start and 14 months after diagnosis, establishing a new benchmark for rapid response in rare disease treatment development. • Eight-month-old Elly Krueger became the first child to receive IRF2BPL gene replacement therapy on April 3, 2025, with the FDA supporting expansion of this treatment to other affected children.

Fortress Biotech and Partex Form Strategic AI Partnership to Accelerate Drug Discovery and Development

2 months ago

• Fortress Biotech and Partex NV have announced a strategic collaboration leveraging Partex's proprietary AI platform to identify and evaluate biopharmaceutical compounds for potential acquisition or licensing. • The partnership aims to expedite target identification, indication expansion, and molecular profiling, ultimately accelerating the process of bringing innovative therapeutics to market more efficiently. • Fortress Biotech, with seven marketed products and over 20 programs in development, expects this AI-driven approach to enhance their business model of acquiring and advancing assets to create long-term shareholder value.

FDA Grants Priority Review to CUTX-101 for Menkes Disease, Offering Hope for Rare Pediatric Condition

5 months ago

• The FDA has accepted Sentynl Therapeutics' NDA for CUTX-101, granting priority review for the treatment of Menkes disease, a rare genetic disorder. • Clinical trials of CUTX-101 demonstrated an almost 80% reduction in mortality risk compared to untreated patients, significantly improving overall survival. • CUTX-101 has been granted multiple designations, including Breakthrough Therapy and Orphan Drug, highlighting its potential to address a critical unmet need. • Cyprium Therapeutics is eligible to receive up to $129 million in milestone payments and royalties, retaining ownership of a potential Priority Review Voucher.

Novartis' Intrathecal Zolgensma Shows Positive Phase III Results for SMA Type 2

5 months ago

• Novartis' intrathecal onasemnogene abeparvovec (OAV101 IT) met its primary endpoint in the Phase III STEER study for SMA Type 2. • The STEER trial demonstrated a statistically significant increase in motor function, as measured by HFMSE scores, in treatment-naïve patients. • OAV101 IT showed a favorable safety profile, with adverse events similar to the control arm, potentially expanding treatment options for SMA. • Novartis plans to submit the data to regulatory agencies in 2025, seeking approval to broaden the availability of this one-time gene therapy.

MAIA Biotechnology and BeiGene Collaborate to Evaluate THIO in Multiple Cancers

5 months ago

• MAIA Biotechnology and BeiGene enter a clinical supply agreement to assess THIO, combined with tislelizumab, in Phase 2 trials. • The trials will focus on hepatocellular carcinoma (HCC), small cell lung cancer (SCLC), and colorectal cancer (CRC). • Preclinical results showed THIO, combined with CPI, induced potent anti-tumor immune responses and complete responses in various cancer models. • MAIA retains global development and commercial rights to THIO, targeting accelerated FDA approvals for multiple cancer indications.

Fortress Biotech's Emrosi Receives FDA Approval for Rosacea Treatment

6 months ago

• The FDA approved Emrosi (minocycline hydrochloride extended-release capsules, 40mg) for treating inflammatory lesions of rosacea in adults, marking Fortress Biotech's first FDA approval. • Clinical data presented at the Fall Clinical Dermatology Conference showed Emrosi provides higher dermal concentration than doxycycline, potentially leading to clinically meaningful impact. • Fortress Biotech anticipates a PDUFA goal date of December 28, 2024, for cosibelimab, an anti-PD-L1 antibody, for treating metastatic or locally advanced cutaneous squamous cell carcinoma. • Journey Medical will launch Emrosi in late Q1 or early Q2 2025, offering a potential new treatment paradigm for millions suffering from rosacea.

Genetic Study Uncovers Key Survival Predictors in Black AML Patients

7 months ago

• A global study identified novel molecular predictors of survival in Black patients with acute myeloid leukemia (AML), highlighting ancestry-specific genetic factors. • Researchers found that incorporating these ancestry-specific mutations into risk stratification systems improved outcome predictions for Black AML patients. • The study revealed significant differences in genetic mutation frequencies and biological features between Black and white AML patients. • The findings underscore the need for increased diversity in clinical studies to improve AML care and understanding across all patient populations.

Elevidys Gene Therapy for Duchenne Shows Sustained Benefit in Long-Term Follow-Up

7 months ago

• Five-year follow-up data from Study 101 supports the role of delandistrogene moxeparvovec (Elevidys) in stabilizing or slowing Duchenne muscular dystrophy (DMD) progression. • Patients treated with Elevidys maintained 10-meter walk/run time over five years, showing a clinically meaningful difference compared to the external control cohort. • EMBARK trial data indicates Elevidys does not negatively impact cardiac measures over a 52-week treatment period, reinforcing its manageable safety profile. • A pre-specified global statistical test in the EMBARK trial indicated a functional treatment effect, despite not meeting the primary endpoint of change in NSAA score.

Novartis Presents New Data on Safety and Efficacy of Zolgensma in Older and Heavier Children with SMA

2 years ago

Novartis has unveiled new data from the SMART study, highlighting the safety and efficacy of Zolgensma, a one-time gene therapy for spinal muscular atrophy (SMA), in older and heavier children. The study shows that nearly all patients maintained or improved motor milestones after 52 weeks, supporting the therapy's use in a broader patient population.