Clinical Research Focus. 44th Edition

NIH Scientists Advance Gene-Editing Approach for Late-Onset Tay-Sachs

NIH researchers have successfully used gene-editing to boost enzyme activity and ease symptoms of late-onset Tay-Sachs in human cells and mice. This breakthrough could pave the way for future therapies targeting Tay-Sachs and other lysosomal storage disorders.

Learn more

Milestone Achieved: Cromos Pharma Earns ISO 9001:2015 Certification When compliance becomes a competitive advantage

We are proud to announce that Cromos Pharma has earned ISO 9001:2015 certification — a milestone that underscores our commitment to excellence in clinical research quality management. By achieving this certification, we reaffirm our commitment to delivering clinical trials that are faster, more reliable, and consistent worldwide.

Learn more

FDA Grants First-Ever Waiver of Clinical Efficacy Studies for Monoclonal Antibody Biosimilars

Professor Sarfaraz K. Niazi has secured a historic FDA decision to approve a monoclonal antibody biosimilar without requiring clinical efficacy studies. This breakthrough is set to cut development costs by over 90%, speed up market entry, and significantly improve global patient access to affordable biologic medicines.

Learn more

From Tariff-Free to Targeted: How the EU–US Trade Deal Puts Pharma in the Crosshairs

The July 2025 EU–US trade agreement brought relief to many industries by averting a 30% U.S. duty on European goods. But for pharmaceuticals, the deal marked the end of a long-standing safe harbor. A new ceiling of 15% tariffs on EU exports, including branded medicines, has upended decades of tariff-free transatlantic trade and thrust the sector into uncharted territory.

Learn more

Global Oncology Trials, Local Relevance: Why the FDA Just Raised the Bar

The FDA’s rejection of Genentech’s Columvi BLA underscores a critical shift: global oncology trials must generate data that are directly relevant to U.S. patients. Our latest publication explores what went wrong in the STARGLO trial and outlines practical strategies for sponsors and CROs to design globally inclusive, regulator-ready studies.

Learn more

FDA Approves First Immunotherapy for Recurrent Respiratory Papillomatosis

The FDA has approved Papzimeos (zopapogene imadenovec-drba), the first immunotherapy for recurrent respiratory papillomatosis (RRP). This breakthrough offers patients an alternative to repeated surgeries, with over half achieving sustained responses in trials.

Learn more

Akeso & Summit’s Ivonescimab Becomes First PD-1xVEGF to Show OS Benefit in Lung Cancer

In the phase 3 HARMONi-A trial, Akeso and Summit’s bispecific antibody ivonescimab achieved a statistically significant overall survival improvement in EGFR-mutated NSCLC. This marks the first time a PD-1xVEGF therapy has extended patient lives, reinforcing its breakthrough potential in global oncology.

Learn more

Regeneron’s siRNA Drug Hits Phase 3 in Myasthenia Gravis, Eyes FDA Filing

Regeneron has reported positive phase 3 results for cemdisiran in generalized myasthenia gravis (gMG), meeting its primary endpoint and outperforming placebo. The siRNA therapy, licensed from Alnylam, now positions Regeneron to pursue an FDA filing in early 2026, adding momentum in the competitive neuromuscular space.

Learn more

Lilly’s Oral GLP-1 Orforglipron Meets Phase 3 Goals in Obesity and Diabetes, Global Filings Set for 2025

Eli Lilly announced that its once-daily oral GLP-1, orforglipron, achieved all primary and secondary endpoints in the phase 3 ATTAIN-2 trial. Patients with obesity and type 2 diabetes lost an average of 10.5% body weight and saw significant A1C reductions. With these results, Lilly is moving forward with global regulatory submissions in 2025, aiming for broad approval as the first scalable oral GLP-1 obesity therapy.

Learn more