AMT-130

Question 1

Huntington’s disease is an autosomal dominant disorder.

Answer 1

TRUE

Question 2

All patients of HD show the same course of disease, characterized by involuntary choreatic movements and behavioural and cognitive disturbances.

Answer 2

FALSE

Question 3

In HD, there is a direct correlation between the length of the CAG repeats and the age of onset: the longer the repeats, the later the patient has the first symptoms.

Answer 3

FALSE

Question 4

Exon 2 HTT protein is highly pathogenic and very aggregation-prone.

Answer 4

FALSE

Question 5

In HD, degeneration occurs primarily in neurons in the putamen and caudate, as well as the cerebral cortex.

Answer 5

TRUE

Question 6

HEMGENIX, developed by uniQure and CSL Behring, is the world’s first gene therapy for haemophilia B and won the 2023 Prix Galien USA Award.

Answer 6

TRUE

Question 7

Glybera was the first approved gene therapy in the world and was developed by uniQure for treating Huntington’s disease.

Answer 7

FALSE

Question 8

AMT-130, uniQure’s gene therapy candidate for Huntington’s disease, utilizes LinQURE technology to silence the Huntingtin gene.

Answer 8

FALSE

Question 9

uniQure is an innovative biotechnology company based in Amsterdam, Netherlands, focused on gene therapy

Answer 9

TRUE

Question 10

mRNA silencing involves an antisense RNA binding to the target mRNA to inhibit protein translation.

Answer 10

TRUE

Question 11

Adeno-associated viruses (AAVs) have low immunogenicity and do not integrate into the host genome, reducing cancer risks.

Answer 11

TRUE

Question 12

AAV vectors can replicate independently without the need for helper viruses like adenoviruses.

Answer 12

FALSE

Question 13

SNP-based strategies have universal applicability for all Huntington’s disease patients.

Answer 13

FALSE

Question 14

AMT-130 is designed to silence the mRNA encoded by both the mutant and wild-type huntingtin gene.

Answer 14

FALSE

Question 15

AMT-130 reverses existing neuronal damage caused by Huntington’s disease.

Answer 15

FALSE

Question 16

Once infused, AMT-130 diffuses into the cortex and other HD-affected brain regions.

Answer 16

TRUE

Question 17

Baculovirus/Sf9 production is more commonly employed during the commercial manufacturing of adeno-associated viruses (AAVs) due to its scalability and cost efficiency.

Answer 17

TRUE

Question 18

The transient transfection system for adeno-associated viruses (AAVs) production is known for its poor full/empty ratio and high cost of goods, despite having a good yield and a short timeline for development.

Answer 18

TRUE

Question 19

The cerebrospinal fluid (CSF) neurofilament light chain (NfL) is released when neurons are damaged and serves as an important biomarker for neurodegeneration in Huntington’s disease (HD). It has been used as a measure during AMT-130 clinical trials.

Answer 19

TRUE

Question 20

Adenovirus are the most prevalent delivery vehicle in clinical gene therapy research

Answer 20

FALSE