Early Phase Development

The FDA has granted Regenerative Medicine Advanced Therapy designation to Ocugen's OCU410, a one-time AAV gene therapy for geographic atrophy secondary to dry age-related macular degeneration, based on Phase 2 data showing a statistically significant reduction in lesion growth.

Paul Romness, chair, chief executive officer, and president of OS Therapies, discusses how advances in linker chemistry, payload design, and evolving regulatory approaches could shape the next generation of antibody–drug conjugates for oncology.

Takeda will use Boltz biomolecular AI models to support structure prediction, affinity estimation, and molecular design in preclinical research.

The company reported that all three participants in the 3 µg/kg dose cohort achieved a ≥25% SALT score reduction following single-dose subcutaneous administration, with dose escalation ongoing toward a recommended phase 2 dose.

Study startup delays persist due to multiple factors, with improved performance increasingly tied to aligning feasibility and activation planning with real-world site capacity, maintaining early momentum, and reducing operational friction through clearer coordination and enabling technologies.

LTZ Therapeutics’ $38 million financing supports phase 1 development of myeloid engager therapies targeting oncology and autoimmune diseases.

A disciplined approach is needed to address complexity, limited knowledge, lack of platform processes and standard analytics, and regulatory uncertainty.

Practical insights into early phase trial design that highlight important statistical considerations and recent developments shaping early phase research.

The launch of Excalipoint Therapeutics brings to the table multi-specific T-cell engager platforms that aim to overcome tumor microenvironment barriers and expand efficacy into solid tumors and immunology indications.

The company is targeting the $3 billion pancreatic cancer market with its lead proenzyme therapy candidate, PRP, which is supported by strong preclinical tumor inhibition data and for which a PK assay will be developed and validated.

Under the collaboration, Merck and Mayo Clinic will integrate multimodal clinical and genomic datasets with AI models to enhance target biology insights and translational decision-making.

Under an exclusive license, the joint venture aims to advance HCW11-006 into Phase I for solid tumors, validating TRBC-derived immunotherapy in a global development strategy.

Stoke has initiated a Phase I trial of STK-002, advancing antisense protein restoration as a potential strategy for genetic optic neuropathies.

Operational missteps, not flawed science, remain a leading cause of drug development delays, as fragmented systems, uneven technology adoption, and transactional sponsor–CRO relationships widen the “clinical execution gap.”

VectorY Therapeutics advances ALS R&D with a vectorized antibody strategy that targets TDP-43 pathology to enable sustained CNS exposure and biomarker-driven evaluation.

The biological reagents provider is moving to a 5000-square-foot site in Cambridge Technopark to accommodate the company’s growth.

Scientists at MIT and other institutions have discovered compounds that activate a defense pathway inside host cells that could be used as antiviral drugs.

Aviva Capital Partners and developer Socius are investing £1 billion in a 12-acre site to be located at the London Cancer Hub next to The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust’s Sutton site.

CAR T-cell therapies, TILs, and other advanced therapies are pushing progress in the treatment of cancers.

Hurdles exist across all aspects and phases of drug development and manufacturing.

Treatment for cancer continues to drive immense innovation in the drug development pipeline, with advanced cell therapies, vaccines, and ADCs taking center stage.

Platform processes and effective risk assessments help overcome time and cost challenges.

The authors have reviewed applications of novel technologies in the major stages of biosimilars development: process development, pharmacology, toxicology, and clinical trials, with an emphasis on recent regulatory requirements.

With the Proteologix acquisition, Johnson & Johnson gains two bispecific antibody early phase assets for immune-mediated diseases.

Broken String Biosciences and the Francis Crick Institute will collaborate on research into how genome stability impacts ALS.