[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.] [@zhaoweiasu](/creator/twitter/zhaoweiasu) "AAV Gene Therapy Scoreboard (2025) 1Eye (Luxturna) ✅ Success. Local subretinal injection XXX XX vg/eye. Tiny immune-privileged space; minimal systemic exposure. 2Ear (OTOF) ✅ Early success. Local cochlear injection; low dose with minimal systemic exposure. 3Local brain (Huntingtons AMT-130) ✅ Encouraging. Direct infusion into striatum; bypasses the BBB. (Recent topline shows slowing of progression in high-dose cohort.) 4Systemic brain (Capsida CAP-002) ⚠ Trial paused after a patient death; investigation ongoing. IV delivery (1010 vg/kg) aims for brain-wide reach but broad systemic exposure is" [X Link](https://x.com/zhaoweiasu/status/1977466153665696230) [@zhaoweiasu](/creator/x/zhaoweiasu) 2025-10-12T20:07Z 6566 followers, 7704 engagements "AATD $WVE-006 vs $BEAM-302 ➡200 mg MAD (Q2W) vs XX mg single dose ➡Total AAT: XXXX vs XXXX M ➡M-AAT: XX% (peak) vs XX% (day 28) ➡Reduction in Z-AAT: -XXXX% (peak) vs -XX% (day 28) 🚨Conclusion: BEAM-302 is superior in every metric Note: Unlike WVE-006 BEAM-302 shows a clear dose response and has higher dose data coming (75mg and 60mg x 2) early next year" [X Link](https://x.com/zhaoweiasu/status/1963283436451602783) [@zhaoweiasu](/creator/x/zhaoweiasu) 2025-09-03T16:50Z 6559 followers, 9369 engagements "@yaireinhorn @CRISPRTX @CrisprSam Classical misleading statements by $CRSP. Base editing is not saturating at 12uM as $BEAM has not yet reported the 4th dose data. Prime editing was invented by David Liu's lab in 2019 and "SyNTase editing" is clearly copying it" [X Link](https://x.com/zhaoweiasu/status/1977783527480623558) [@zhaoweiasu](/creator/x/zhaoweiasu) 2025-10-13T17:08Z 6563 followers, 1221 engagements "$EDIT's bold move (different from PCSK9/ANGPTL3 knockdown) ➡ Dual guide RNA to delete regulatory elements in the 3' UTR ➡ Upregulates LDLR in the liver by 6X and drove XX% LDL-C lowering in animals (preclinical) ❓ Big question: in humans how low LDL-C should we goand whats the right target for lifelong risk reduction" [X Link](https://x.com/zhaoweiasu/status/1978595274768687122) [@zhaoweiasu](/creator/x/zhaoweiasu) 2025-10-15T22:54Z 6566 followers, 3130 engagements "Base editing corrects SERPINA1-E342K mutation for alpha-1 antitrypsin deficiency (AATD). "This landmark result in medicine represents the first clinical evidence of precise correction of a disease-causing mutation by rewriting the genetic code." -- John Evans CEO of Beam Therapeutics" [X Link](https://x.com/zhaoweiasu/status/1980431050741555424) [@zhaoweiasu](/creator/x/zhaoweiasu) 2025-10-21T00:28Z 6566 followers, 1483 engagements "@GeneInvesting People like @capital_usher and @crisprspace are clearly more bullish on $PRME than you. They have been supporting it all the time" [X Link](https://x.com/zhaoweiasu/status/1980795575428870390) [@zhaoweiasu](/creator/x/zhaoweiasu) 2025-10-22T00:37Z 6564 followers, XXX engagements "@GeneInvesting You cannot say you spend more time on BEAM and PRME than I do. In fact you already lost track on BEAM" [X Link](https://x.com/zhaoweiasu/status/1981090324681544142) [@zhaoweiasu](/creator/x/zhaoweiasu) 2025-10-22T20:08Z 6567 followers, XXX engagements
[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.]
@zhaoweiasu
"AAV Gene Therapy Scoreboard (2025) 1Eye (Luxturna) ✅ Success. Local subretinal injection XXX XX vg/eye. Tiny immune-privileged space; minimal systemic exposure. 2Ear (OTOF) ✅ Early success. Local cochlear injection; low dose with minimal systemic exposure. 3Local brain (Huntingtons AMT-130) ✅ Encouraging. Direct infusion into striatum; bypasses the BBB. (Recent topline shows slowing of progression in high-dose cohort.) 4Systemic brain (Capsida CAP-002) ⚠ Trial paused after a patient death; investigation ongoing. IV delivery (1010 vg/kg) aims for brain-wide reach but broad systemic exposure is"
X Link @zhaoweiasu 2025-10-12T20:07Z 6566 followers, 7704 engagements
"AATD $WVE-006 vs $BEAM-302 ➡200 mg MAD (Q2W) vs XX mg single dose ➡Total AAT: XXXX vs XXXX M ➡M-AAT: XX% (peak) vs XX% (day 28) ➡Reduction in Z-AAT: -XXXX% (peak) vs -XX% (day 28) 🚨Conclusion: BEAM-302 is superior in every metric Note: Unlike WVE-006 BEAM-302 shows a clear dose response and has higher dose data coming (75mg and 60mg x 2) early next year"
X Link @zhaoweiasu 2025-09-03T16:50Z 6559 followers, 9369 engagements
"@yaireinhorn @CRISPRTX @CrisprSam Classical misleading statements by $CRSP. Base editing is not saturating at 12uM as $BEAM has not yet reported the 4th dose data. Prime editing was invented by David Liu's lab in 2019 and "SyNTase editing" is clearly copying it"
X Link @zhaoweiasu 2025-10-13T17:08Z 6563 followers, 1221 engagements
"$EDIT's bold move (different from PCSK9/ANGPTL3 knockdown) ➡ Dual guide RNA to delete regulatory elements in the 3' UTR ➡ Upregulates LDLR in the liver by 6X and drove XX% LDL-C lowering in animals (preclinical) ❓ Big question: in humans how low LDL-C should we goand whats the right target for lifelong risk reduction"
X Link @zhaoweiasu 2025-10-15T22:54Z 6566 followers, 3130 engagements
"Base editing corrects SERPINA1-E342K mutation for alpha-1 antitrypsin deficiency (AATD). "This landmark result in medicine represents the first clinical evidence of precise correction of a disease-causing mutation by rewriting the genetic code." -- John Evans CEO of Beam Therapeutics"
X Link @zhaoweiasu 2025-10-21T00:28Z 6566 followers, 1483 engagements
"@GeneInvesting People like @capital_usher and @crisprspace are clearly more bullish on $PRME than you. They have been supporting it all the time"
X Link @zhaoweiasu 2025-10-22T00:37Z 6564 followers, XXX engagements
"@GeneInvesting You cannot say you spend more time on BEAM and PRME than I do. In fact you already lost track on BEAM"
X Link @zhaoweiasu 2025-10-22T20:08Z 6567 followers, XXX engagements
/creator/twitter::191281008/posts