MCO-010

Question 1

Nanoscope Therapeutics is focused in optogenetics, a gene therapy approach where an exogenous gene encoding light-sensitive proteins is introduced into cells, enabling them to respond to light.

Answer 1

TRUE

Question 2

The two diseases that Nanoscope Therapeutics is focusing on are cancer and diabetes.

Answer 2

FALSE

Question 3

Retinitis pigmentosa is an inherited disease that predominantly affects the retinal ganglion
cells.

Answer 3

FALSE

Question 4

Cone degeneration in retinitis pigmentosa usually follows rod damage, resulting in progressive loss of central vision and colour perception.

Answer 4

TRUE

Question 5

The available therapies for retinitis pigmentosa are retinal prosthesis, gene therapy and stem cells therapy.

Answer 5

FALSE

Question 6

Opsin genes are divided into three distinct superfamilies: microbial opsins (type I), animal opsins (type II) and vegetal opsins (type III).

Answer 6

FALSE

Question 7

Channelrhodopsin-2 (ChR2) is a microbial opsin widely used in optogenetic research.

Answer 7

TRUE

Question 8

MCO-010 is administered through a one-time intravitreal injection that, apart from the AAV2, it also contains pronase E to enhance delivery efficiency to bipolar cells in the retina.

Answer 8

TRUE

Question 9

MCO-010 gene therapy uses a viral vector to integrate the transgene into the patient’s genome for long-term expression in retinal cells.

Answer 9

FALSE

Question 10

The MCO-010 gene therapy requires the use of external light amplifiers to restore vision in Retinitis Pigmentosa patients.

Answer 10

FALSE

Question 11

The all-trans retinal (ATR) chromophore in MCO-010 absorbs light and triggers the conformational change required to activate the opsins.

Answer 11

TRUE

Question 12

The optogenetic technology used by Nanoscope does not rely on specific genetic defects, allowing a larger patient population to be treated.

Answer 12

TRUE

Question 13

Chrimson opsin’s activation range goes from 400 nm (blue light) to 550 nm (green light).

Answer 13

FALSE

Question 14

MCO-010 therapy requires special glasses to amplify the light and activate the implanted opsins.

Answer 14

FALSE

Question 15

Nanoscope’s STARLIGHT and RESTORE clinical trials are designed to treat Stargardt’s disease and retinitis pigmentosa, respectively.

Answer 15

FALSE

Question 16

MCO-010 gene therapy is designed to restore vision in patients with retinitis pigmentosa by activating bipolar cells.

Answer 16

TRUE

Question 17

Retinal prosthetic devices such as the Argus II fully restore vision in patients with retinitis pigmentosa.

Answer 17

FALSE

Question 18

Nanoscope Therapeutics uses a combination of animal and microbial opsins in its optogenetic technology.

Answer 18

FALSE

Question 19

CatCh opsin is a modified variant of channelrhodopsin-2, optimised to be sensible to lower light intensity.

Answer 19

TRUE

Question 20

MCO-010 uses an adeno-associated virus type 3 (AAV3) vector to insert its transgene into retinal bipolar cells.

Answer 20

FALSE