Denali

DNL126, Denali Therapeutics' brain-penetrating enzyme replacement therapy, corrected signs of Sanfilippo syndrome type A in a mouse model.

Intravenous administration showed a dose-dependent increase of DNL126 in the brain, compared with mice without human TfR. The liver also showed a significant reduction in heparan sulfate with DNL126. [website](https://patients.sanguinebio.com/sanfilippo-syndrome-study/), the natural history study will require seven visits over the course of about two years. Because the SGSH enzyme is found in lysosomes, the cellular compartments responsible for breaking down and recycling molecules, heparan sulfate buildup disrupts lysosomal function. Participants will be compensated up to a total of $1,400. The study is expected to enroll 20 participants, ages 4 months to 13 years, with at least 10 patients younger than age 4. This is the most common type, and a more severe form, with As a result, this sugar builds up to toxic levels inside cells, causing damage and inflammation, especially in brain tissue. Now, the biopharmaceutical company plans to seek U.S. Once there, DNL126 may help break down heparan sulfate and address Sanfilippo’s cognitive, behavioral, and physical symptoms. In the DNL126 preclinical study, healthy mice were engineered to produce a portion of the human TfR. [Sanfilippo syndrome type A](https://sanfilipponews.com/types-of-sanfilippo-syndrome/) in a mouse model, according to its developer, [Denali Therapeutics](https://www.denalitherapeutics.com/).