AMP BGTC Funding Opportunities

Clinical Opportunities

Clinical trial proposals directed to one of the 14 bespoke indications in the table below can be submitted in response to the BGTC’s clinical trials RFP for final consideration for the BGTC’s clinical program (see RFP below). Applications are due by 11:59 PM EDT on October 31, 2022 and an informational webinar will be held on August 30, 2022. We anticipate completing the final selection process in February 2023 and selecting 5-6 diseases to move forward with clinical trials. BGTC highly encourages collaboration between Investigators working in these disease areas, including collaboration with NIH Investigators, and engagement with relevant patient groups in the application process. Read the full announcement here.

Clinical Trial RFP
Appendix 1, BGTC Clinical Trial Proposal Submission Form

Appendix 2, Phase 1/2 IND Clinical Trial Protocol Template
Appendix 3, Budget worksheet COMING SOON
Appendix 4, Informed Consent Template

The BGTC is hosting an informational webinar for the Clinical Trials RFPs on August 30, 2022 from 11:30 AM - 1 PM. Learn more here.

Other Resources

Click here for a description of the disease nomination review process that was followed to determine eligibility.

Click here for a resource page designed to assist patient groups in their advocacy journey.

Disease Name (pseudonym) Affected Gene BGTC Classification
Congenital Hereditary Endothelial Dystrophy (CHED) SLC4A11 Eye / Cornea
MPS VI corneal disease ARSB Eye / Cornea
Leber Congenital Amaurosis 16 (LCA16) KCNJ13 Eye / Retina
Retinitis pigmentosa (RP) - CNGB1 CNGB1 Eye / Retina
NPHP5-RD NPHP5 Eye / Retina
Charcot Marie Tooth disease type 4J (CMT4J) FIG4 Neurological
Multiple Sulfatase Deficiency SUMF1 Neurological
Spastic Paraplegia, type 47 (SPG47) AP4B1 Neurological
Spastic paraplegia 50 (SPG50) AP4M1 Neurological
Barth Syndrome TAZ Cardiac
PGM1-Congentital Disorder of Glycosylation (CDG) (PGM1 deficiency) PGM1 Inborn error of metabolism
Propionic Acidemia PCCB Inborn error of metabolism
Fibrodysplasia Ossificans Progressiva (FOP) ACVR1 Orthopedic
Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) GALNS Orthopedic

Table 1. Rare diseases and disorders received in the nomination process that were selected by the BGTC Steering Committee as candidates for further applications.

 

AAV Biology Research Opportunities

BGTC has reissued new AAV biology research opportunities. View and download the RFPs below. Proposals are due by 11:59 PM EDT, August 28, 2022.

AAV Biology Gene Expression RFP
AAV Biology Vector Production RFP
Appendix 1, BGTC Proposal Submission Form and Response Template
 

The BGTC is held an informational webinar for the AAV Biology RFPs. Click here for a recording of the webinar. FAQs will be posted soon.