Pathway

Step 1

Tertiary
care center

Step 2

Application for
Treatment is submitted
to n-Lorem

Physicians

Patients

Institutions

Step 3

ATTC reviews
application

Step 4

n-Lorem reviews and makes a decision

Step 5

Patient consents and genome sequencing begins

Step 6

ASO discovery starts

Step 7

Natural history
collection begins

Step 8

ASO development and manufacturing begins

Step 12

Patient begins
treatment

Step 11

Step 10

Institution review board and master service agreements

Sterile fill and finish (Occurs in tandem with Step 11)

Step 9

IND preparation and
filing begins

Duration:
(N/A)

Duration:
(Ongoing from discovery to dosing)

Duration:
(3 months)

Duration:
(2 months)

Duration:
(1-3 months)

Duration:
(2 months)

Initiation to treatment:
(Steps 6-12)
15-18 months on avg.

Duration:
(7 months)

Duration:
(1-2 months)

Duration:
(N/A)

Duration:
(1-3 months)

Patient
Pathway to
Treatment