AMT Receives a Third of €3.3M EU Grant for Clinical Development of Gene Therapy - Gilde Healthcare

AMT Receives a Third of €3.3M EU Grant for Clinical Development of Gene Therapy

February 1, 2011

Amsterdam Molecular Therapeutics (AMT) will receive €1.1 million (about $1.5 million) of a €3.3 million (approximately $4.5 million) grant being awarded by the European Union 7th Framework Program (FP7) to the AIPGENE consortium, which is working to develop a gene therapy product for treating acute intermittent porphyria (AIP).

As part of the consortium AMT has commercialization rights to the gene therapy product, AMT-021, and projects patient enrolment into a clinical trial could start in 2012.  It says the grant will cover development costs of the product to completion of Phase I/II trials.

AMT-021 is designed to deliver a normal version of the porphobilinogen deaminase (PBDG)  liver protein gene that is mutated in AIP. AMT says its studies have already shown treatment using AMT-021 results in normalization of the PBGD protein in an animal model of AIP as well as prevents the occurrence of attacks and significantly ameliorates the neuropathy that otherwise occurs in untreated animals. The firm in addition notes that work by FIMA (Fundación para la Investigatión Médica Aplicada), which is leading the multinational AIPGENE consortium, has demonstrated that gene expression in the liver will persist for over a year using AAV-mediated delivery methods similar to that used for AMT-021.

Gilde Healthcare company Tagworks Pharmaceuticals Announces FDA Clearance of IND Application and Initiation of Phase 1 Clinical Trial for TGW101

TGW101 is a next-generation, first-in-class ADC targeting TAG-72 with an MMAE payload, developed based on Tagworks proprietary Click-to-Release linker chemistry; TGW101 is the first bioorthogonal, in vivo click chemistry-activated ADC, which enables superior control of...
April 22, 2025

Gilde Healthcare company SynOx Therapeutics announces Board Chair transition to align with advancing Regulatory and Commercialization Strategy

SynOx Therapeutics Limited (“SynOx”), a late-stage clinical biopharmaceutical company developing of emactuzumab for Tenosynovial Giant Cell Tumours (TGCT), today announced the execution of a planned and orderly transition of its board chair position. Philip Astley-Sparke...
April 1, 2025

Gilde Healthcare company SpliceBio announces first patient dosed in Phase 1/2 ASTRA study of SB-007, a Dual-AAV Gene Therapy for Stargardt Disease

SB-007 addresses the root cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations SB-007 is the first dual AAV gene therapy in clinical development for Stargardt disease   SpliceBio,...
March 13, 2025