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STAT+: Pennsylvania Dutch country, tired of waiting for biopharma, builds drug company of its own

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Based on a feed excerpt; full article content has not been acquired. Source evidence retrieved 27/10/2026, 08:36:25 UTC.

Briefings

Researchers in Lancaster, Pennsylvania successfully treated a 79-pound calf with a gene therapy for maple syrup urine disease (MSUD), proving the technology works. The treatment was administered to a 5-year-old heifer named Petunia, who was the second of three cows in the study. Despite the success, none of the clinic's patients received the treatment, and the specific gene therapy remains unapproved for clinical trials.

Key points

• Researchers in Lancaster, Pennsylvania successfully treated a 79-pound calf with a gene therapy for maple syrup urine disease (MSUD), proving the technology works.
• The treatment was administered to a 5-year-old heifer named Petunia, who was the second of three cows in the study.

Context

This finding addresses a critical need for a one-time fix for families in the Mennonite community in Lancaster, Pennsylvania, who suffer from MSUD. The study involved three researchers and one cow, with the goal of offering a simple, one-time solution to a life-threatening condition.

Limitations

The evidence is limited to a single cow treated in a clinical setting, not a clinical trial. The specific gene therapy used remains unapproved for clinical trials, and the specific gene therapy used remains unapproved for clinical trials.

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