The BioPharm Brief: Gene Therapy, ADCs, Big Pharma Alliances

Welcome to The BioPharm Brief, your daily news update from BioPharmInternational.com, where we break down the most important developments shaping drug development, regulatory strategy, and biopharma partnerships.

Today’s headlines span rare disease gene therapy, targeted oncology innovation, and one of the largest early-stage collaboration deals announced this year.

AskBio has dosed the first participant in its Phase 1/2 PROGRESS-GT LOPD clinical trial evaluating AB-1009, an adeno-associated virus, or AAV-based gene therapy for late-onset Pompe disease. The investigational therapy is designed to address the underlying genetic cause of Pompe disease by enabling sustained production of the deficient GAA enzyme. The study will enroll approximately 12 adult participants in the United States and will assess safety, tolerability, and early efficacy signals. Current standard of care relies on enzyme replacement therapy, which requires ongoing infusions and may become less effective over time in some patients, highlighting the need for longer-acting treatment approaches.

In oncology, Servier announced that the US Food and Drug Administration has granted Breakthrough Therapy designation to emiltatug ledadotin, an investigational antibody-drug conjugate targeting B7-H4 for the treatment of adenoid cystic carcinoma, or ACC. ACC is a rare and aggressive cancer most commonly originating in the salivary glands, and there are currently no approved systemic therapies for advanced disease. Early Phase 1 data showed antitumor activity across multiple tumor types with a manageable safety profile. The Breakthrough Therapy designation is intended to accelerate the development and regulatory review of therapies that may offer meaningful advantages over existing treatment options.

And in a major industry development, Bristol Myers Squibb and Hengrui Pharma announced a collaboration potentially worth up to $15.2 billion. The agreement covers 13 early-stage programs across oncology, hematology, and immunology, and includes joint development and licensing arrangements designed to advance a broad pipeline of investigational assets. The partnership reflects continued expansion of cross-border deals, particularly between large global pharmaceutical companies and China-based innovators, as companies look to strengthen early-stage pipelines and diversify therapeutic portfolios.

Taken together, today’s updates reflect three defining trends in biopharma: the push toward potentially durable gene therapies in rare disease, increasing use of targeted biologics in hard-to-treat cancers, and continued globalization of early-stage drug development through large strategic partnerships.

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Summary of key developments

• AskBio initiated dosing in a Phase 1/2 study of AB-1009, an AAV gene therapy for late-onset Pompe disease.
  
• Servier received FDA Breakthrough Therapy designation for emiltatug ledadotin in adenoid cystic carcinoma.
  
• Bristol Myers Squibb and Hengrui launched a $15.2B collaboration spanning 13 programs in oncology, hematology, and immunology.