Amanda Sani

Strategy consultant by day. Cell and gene therapy super fan by night. Opinions are my own. Don't get investment advice from your twitter feed.

[Amanda Sani @AmandaSani4]

Excited to announce my upcoming webinar with my colleague and mentor @VMittalPhD! Tune in to hear our thoughts on redosing in gene therapy and what that means for commercial strategy 📈 #GeneTherapy #BioMarin #hemophilia

[Health Advances @HealthAdvances]

The @US_FDA’s rejection of #Roctavian is an unexpected twist in the race for #genetherapy for #hemophilia. But can #BioMarin use this setback to redefine & optimize the commercial strategy for #Roctavian? Tune in for @HealthAdvances free webinar on 9/3... https://t.co/mh3oORvk2C

[Amanda Sani @AmandaSani4]

Here are my thoughts on the major questions and themes out of #Mesa19 Hats off to @alliancerm for another great conference in the books!
https://t.co/MDFulw5sb2 #celltherapy #genetherapy

[Amanda Sani @AmandaSani4]

@andyfunderburk @HealthAdvances Thanks Andrew!

[Amanda Sani @AmandaSani4]

@davidrliu and team continues to advance the science and technology enabling gene editing! Impressive work with potential to open up a vast number of diseases to targeting with editing-based approaches #Genetics

[nature @Nature]

A new gene-editing tool called prime editing allows for greater precision and control over DNA edits compared to the popular CRISPR-Cas9 system. https://t.co/jtKXbIFhIM

[Amanda Sani @AmandaSani4]

Heading down to San Diego for @alliancerm's Medicine's Meeting on the Mesa! Excited to hear the latest on:
•Allogeneic cell therapy advancements
•Multi-antigen targeting constructs
•And of course, novel outcomes-based payment models
#Mesa19 #CellTherapy

[Andrew Funderburk @andyfunderburk]

That's not what you usually see for pancreatic cancer - early data/small N but wow! #AACR19

[Amanda Sani @AmandaSani4]

Fascinating data courtesy of @AndyBiotech on improving blood cancer survival rates mapped to new therapy entrances. Hoping to see CAR-T therapies continue this trend!

[Andy Biotech @AndyBiotech]

Blood cancers are probably the best examples to demonstrate how #biotech innovations have made huge impact on patient lives.

5-Yr survival rate from 1970s to 2010s
CML: 22% -> 68%
NHL: 47% -> 73%
Myeloma: 25% -> 53%

[Amanda Sani @AmandaSani4]

My favorite fact from #JPM19 so far courtesy of Dan Faga from @spark_tx "Of the 75 patients who received doses of Luxturna this year, not one paid anything out of pocket." Now that's what I call access!

[Amanda Sani @AmandaSani4]

@logankugler @chrissyfarr @MattRosoff @Techmeme Respectfully disagree. This is news because she is the fourth public exec hire at an extremely high profile venture. The headline notes she's the first female exec but the article itself focuses on her background, experience, and the implications for the venture.

[Health Advances @HealthAdvances]

Senior Analyst, Amanda Sani, reflects on key takeaways & themes from the @alliancerm 2018 Meeting on the Mesa #Mesa18 #CellTherapy #GeneTherapy #RegenMed https://t.co/l7IGE5Hcou

[Jeff Marrazzo @jeffmarrazzo]

Exciting to see this early preclinical progress as we strive to one day bring a #genetherapy to patients living with Pompe disease.

[Amanda Sani @AmandaSani4]

$BOLD: New ASPIRO data looking good. All patients (except control) with >100% change from baseline in MIP score which is a respiratory functional measure. Exciting stuff!

[Amanda Sani @AmandaSani4]

Excuse the awful photo. Check out the @vinetiworks site for a much better one!

[Amanda Sani @AmandaSani4]

One of the coolest things about autologous cell tx (besides, ya know, survival benefit) is that companies like @vinetiworks make dashboards where HCPs/patients can follow their cells along the manufacturing process. Domino's pizza tracker but for medicine! #mesa18 #Celltherapy

[Amanda Sani @AmandaSani4]

From Matt Peterson ($BOLD): Expect an update on AT132 on Friday #Mesa18

[Amanda Sani @AmandaSani4]

Impressive Ph1/2 clinical data from $ABEO in RDEB patients. Excited to see if they can replicate it in Ph3. Check out those PROs! #Mesa18 #GeneTherapy

[Health Advances @HealthAdvances]

Innovative cell & gene therapies deserve innovative payment models. Our #RegenerativeMedicine team examines the potential benefits + risks of outcome-based contracting for Kymriah @Novartis, Yescarta @KitePharma @GileadSciences, & Luxturna @spark_tx: https://t.co/qUuafi2bzr

[Amanda Sani @AmandaSani4]

Another learning: No one is worse at silencing/turning off their cell phones than business people at a conference 🎵 #mesa18

[Amanda Sani @AmandaSani4]

Sandy Macrae's (@SangamoTx) prediction for the Hemophilia space 3 years from now: 3 on market gene therapies for Hem A, 2 for Hem B, and one gene editing product appropriate for use in young patients #mesa18 #GeneTherapy

[Amanda Sani @AmandaSani4]

Also noteworthy: "To date most of our Luxturna contracts have an outcomes component." #mesa18