정상인

컴퓨터공학전공 / 중도보수 / 이성적토론

[다율잉(DaYule) @m72068958]

$CRSP $VRTX
CTX112 delivers a robust safety profile:

No dose-limiting toxicities (DLT), GvHD, or severe infections (≥Grade 3)
Manageable side effects: Low, mild rates of CRS & ICANS

[다율잉(DaYule) @m72068958]

$CRSP $VRTX
Manufacturing efficiency & scalability:

Higher & consistent CAR-T cell production vs CTX110
Allogeneic therapy offers greater scalability & lower production costs

[다율잉(DaYule) @m72068958]

$CRSP $VRTX
CTX112 combines efficacy, durability, safety, & efficiency, marking a critical step forward in B-cell malignancy treatment. A promising future for CAR-T therapy!

[다율잉(DaYule) @m72068958]

Casgevy ATCs have increased from 31 to 33.

$CRSP $VRTX

[다율잉(DaYule) @m72068958]

Casgevy ATCs in the United States have increased from 33 to 34.

$CRSP $VRTX

[다율잉(DaYule) @m72068958]

Casgevy ATCs in the United States have increased from 34 to 36. $CRSP $VRTX

[다율잉(DaYule) @m72068958]

1.
- CTX112 is the current workhorse.
- In the future, as in vivo editing technology becomes simpler, this will become the new flagship.
- Particularly keeping an eye on type 1 diabetes and trying not to lose sight of it.
#CRSP #VRTX $CRSP $VRTX $NTLA $PRME

[다율잉(DaYule) @m72068958]

2. - Casgevy's first revenue patient was from the Middle East. #CRSP #VRTX $CRSP $VRTX $NTLA $PRME

[다율잉(DaYule) @m72068958]

3.
- Half of ATC targets activated.
- Expected to accelerate as progress accelerates.
#CRSP #VRTX $CRSP $VRTX $NTLA $PRME

[다율잉(DaYule) @m72068958]

4.
- Patient numbers are growing exponentially.
- Most patients whose cells have been collected are not abandoning treatment.
#CRSP #VRTX $CRSP $VRTX $NTLA $PRME

[다율잉(DaYule) @m72068958]

5.
- Moderator asks about the risk of death from busulfan.
- More severe patients than expected, but gentler conditioning could triple marketability.
- Can demonstrate this through database.
#CRSP #VRTX $CRSP $VRTX $NTLA $PRME

[다율잉(DaYule) @m72068958]

5-2: Many patients have received transplants, and busulfan is effective and stable.
Vertex and CRISPR are collaborating to optimize conditioning, and a better agent can be substituted if Kasgebi’s success continues. #CRSP #VRTX $CRSP $VRTX $NTLA $PRME

[다율잉(DaYule) @m72068958]

#CRSP #VRTX $CRSP $VRTX $NTLA $PRME
6. Operating on a revenue-sharing model rather than royalties.
In 8-10 years, as sickle cell and thalassemia transition to in vivo editing, the revenue share is expected to reach 50/50.

[다율잉(DaYule) @m72068958]

#CRSP #VRTX $CRSP $VRTX $NTLA $PRME
7. Moderator inquired about the response rate differences between CTX112 and CTX110.
CTX110's ORR data is based on optimized doses, while CTX112 is used for more severe cases, including elderly patients.

[다율잉(DaYule) @m72068958]

#CRSP #VRTX $CRSP $VRTX $NTLA $PRME
7-2: For lymphoma specifically, CTX112 shows a higher CR rate and may surpass autologous CAR-T therapies.
Durability results are being monitored.

[다율잉(DaYule) @m72068958]

#CRSP #VRTX $CRSP $VRTX $NTLA $PRME
8. Moderator asked if the patient population could be adjusted after dosing is finalized.
Regulatory bodies limit autologous CAR-T to just 25% of eligible patients due to treatment accessibility constraints.

[다율잉(DaYule) @m72068958]

8-2: Autologous CAR-T therapies like Yescarta and Breyanzi have reached market saturation.
Allogeneic CAR-T may prove superior to autologous, providing potential market expansion.
#CRSP #VRTX $CRSP $VRTX $NTLA $PRME

[다율잉(DaYule) @m72068958]

9. Addressed concerns about cytokine release syndrome (grade 2) and immune cell suppression (grade 1).
Cytokine activity is seen as a positive efficacy indicator.
#CRSP #VRTX $CRSP $VRTX $NTLA $PRME

[다율잉(DaYule) @m72068958]

9-1 #CRSP #VRTX $CRSP $VRTX $NTLA $PRME
The incidence of grade 2 or higher cytokine release is over 10% in autologous CAR-T, while allogeneic CAR-T shows greater safety.

[다율잉(DaYule) @m72068958]

10. Discussed editing limitations and cell fatigue with CTX112.
Cell memory remains robust, allowing for multiple edits with high efficiency using an RNA-based, single cassette approach.
#CRSP #VRTX $CRSP $VRTX $NTLA $PRME