Last Week in Biomedicine | Jun 29–Jul 3 | Part 2 of 2: Baby Pompe Disease Trial, DLBCL Combo Win

Part 2 covers three more standout stories from the same week: a Phase 3 trial that gives babies with a devastating enzyme disease a fighting chance, a first-of-its-kind cell therapy approved to make bone-marrow transplants safer, and a new lymphoma combination that could give aggressive-blood-cancer patients another option after other therapies fail.

In Part 2 we cover:

4. Sanofi — Nexviazyme (avalglucosidase alfa) for infantile-onset Pompe disease (Phase 3)

5. Orca Bio — Tregzi (allogeneic regulatory T-cell immunotherapy) for adults with blood cancers undergoing matched-donor bone-marrow transplant (FDA approval)

6. Genmab / AbbVie — Epkinly (epcoritamab) plus lenalidomide for relapsed or refractory diffuse large B-cell lymphoma (Phase 3)

What could this mean for patients? The Sanofi Baby-COMET result is the most emotionally consequential story of the week. Infantile-onset Pompe disease is usually fatal in a baby's first year without treatment, and a Phase 3 trial hitting every primary and secondary endpoint means more infants may reach toddlerhood breathing on their own — an outcome that was unimaginable a generation ago.

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