Advancing Hope #89: Four Studies Investigating Potential Therapies for People with FTD Caused by GRN Genetic Mutations

Advancing Hope: Four Studies Seeking Potential Therapies for People with FTD Caused by GRN Genetic Mutations

Participate in FTD Research

AFTD does not “endorse” or “approve” any study or take funding to publicize research opportunities. We provide information and encourage you to consider whether this study seeking participants is a good fit for you.

This week we are highlighting four studies investigating potential therapies for people with FTD caused by GRN genetic mutations. Variants in the GRN gene lead to reduced levels of progranulin, a protein that is involved in cell survival and regulating inflammation. Decreased progranulin levels are associated with the development of FTD, although the exact relationship is not fully understood today.

  • AL001  The company Alector is testing AL001, a potential therapy to increase progranulin levels. For more information: AL001 – INFRONT-3
  • PR006  The company Prevail is researching the safety and effectiveness of a one-time gene therapy, PR006, to examine if it successfully corrects or modifies the effects of the variant. For more information: PR006
  • PBFT02 – The company Passage Bio is researching the safety and effectiveness of a one-time gene therapy, PBFT02, to increase progranulin levels in people in FTD with GRN variants. For more information: PBFT02
  • DNL593 – The company Denali is researching the safety and effectiveness of DNL593, an investigational treatment for people with FTD with GRN variants. For more information: DNL593

Learn how to get involved in these studies by visiting the Studies Seeking Participants page on our website.

 

Click here to access the full issue of Help & Hope #89.

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