VP of Research @CureDuchenne, Partner at CureDuchenne Ventures. #Duchenne #Neuroscience, #Neuromuscular, #RareDisease #VenturePhilanthrophy
Boston
Joined March 2018
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Do you love a beautiful story of community, friendship, love, and the resilience of the human spirit, told via an award-winning documentary?
🎥 Watch The Remarkable Life of #Ibelin on #Netflix, the story of Mats Steen, a young man with Duchenne who found community in #worldofwarcraft 🙏 Thanks to the Steen family & @Blizzard_Ent for supporting #CureDuchenne with the #RevenPack fundraiser! 💙
cureduchenne.org/all-news/wo…
Lianna Orlando retweeted
Adopt Reven & Reven’s Comfy Carrier to support CureDuchenne today!
Learn more: blizz.ly/4eXJScG
Lianna Orlando retweeted
JOIN THE WEBINAR: #FDA Decision on #Elevidys Explained
Date & TimeJun 25, 2024 3:00 PM in Pacific Time / 6:00 PM in Eastern Time: REG: us02web.zoom.us/webinar/regi…
It's been a pleasure working w/ @MDAorg & @ParentProjectMD, in support of a study from @barrybyrne1 testing if a FDA-approved drug from @argenxglobal can lower pre-existing antibodies to AAV & enable more individuals with #Duchenne to have access to AAV-delivered #genetherapies
We're thrilled to work together to address one of the most pressing challenges in Duchenne: pre-existing antibodies to AAV, limiting who can be treated and re-dosed.
@MDAorg @ParentProjectMD
cureduchenne.org/press-relea…
Lianna Orlando retweeted
We join the #BGTC in observing #RareDiseaseDay 2023 – together, we will accelerate and streamline therapies for rare diseases.
#BGTC is proud to observe #RareDiseaseDay 2023 with “Taylor’s Story.” Watch the full film highlighting the patient perspective and underscoring our commitment to accelerate therapies for ultra-rare diseases: fnih.org/our-programs/AMP/BG…
#RareDisease #RDDNIH #ShowYourStripes
Lianna Orlando retweeted
CureDuchenne Ventures is pleased to share the news of our latest investment.
hC Bioscience, Inc.’s experimental therapy aims to correct premature stop codon mutations in Duchenne to restore expression of full-length dystrophin.
cureduchenne.org/general/hcb…
I am proud to represent CureDuchenne in this collaborative effort, so that more rare diseases can benefit from safe and effective gene therapies
Lianna Orlando retweeted
CureDuchenne provided initial seed funding to Myosana in 2020. We welcome new investors as Myosana closes its latest round of funding to support further development of their non-viral gene therapy technology.
cureduchenne.org/research/cu…
I’m proud to represent @CureDuchenne in this important #GeneTherapy effort. A lot of work completed in one year, and many exciting developments ahead!
AMP® BGTC is celebrating its one-year anniversary! Learn more about this groundbreaking gene therapy effort, what the consortium has accomplished in year one, and what’s in store for year two: fnih.org/our-programs/AMP/BG…
This milestone in #Duchenne muscular dystrophy has been decades in the making. Congrats to @Sarepta $SRPT and thank you to everyone who has worked on #GeneTherapy over the years to help get us here!
NEWS: SRPT today announced it has completed the BLA submission seeking accelerated approval for SRP-9001 to treat ambulant individuals with #Duchenne. SRP-9001 is an investigational gene therapy being developed in partnership with Roche. Read the release: bit.ly/3UOWvNl
Lianna Orlando retweeted
We are growing our cardiovascular team and looking for roles at all levels! If you’re passionate about CV research and development, reach out to recruiting@edgewisetx.com to learn more. edgewisetx.com/careers
Lianna Orlando retweeted
Today we announced exciting #DMD results – PGN-EDO51 achieved the highest level of oligonucleotide delivery and exon 51 skipping following a single dose in humans. We will host a corresponding conference call and a live webcast at 8:00 a.m. ET. Read more: bit.ly/3rdXkBX
Our latest Community Bulletin provides an update on our intention to seek an accelerated approval from the U.S. FDA for SRP-9001 to treat individuals with Duchenne muscular dystrophy. Read the bulletin: bit.ly/3JgggZ9
Pfizer gets go-ahead to re-start Phase 3 #GeneTherapy trial in #Duchenne muscular dystrophy. US sites opening soon. $PFE pfizer.com/news/press-releas…
Lianna Orlando retweeted
@Sarepta announced promising results from Part 2 of Study 102, an ongoing DMD clinical trial of investigational gene therapy SRP-9001. cureduchenne.org/partner-new…
Lianna Orlando retweeted
Beam has entered a new collaboration with Pfizer for in vivo base editing! $300M upfront, >$1B milestones, 3 new targets (one each in liver, muscle, and CNS), plus a 35% WW co-co option on any one program at the end of P1/2:
globenewswire.com/news-relea…
CureDuchenne is happy to share REGENXBIO will be launching a novel gene therapy trial in Duchenne during the first half of 2022. The trial will be enrolling ambulatory individuals with Duchenne, aged 4 to 11 years old. bit.ly/3pXqdm7
Congratulations to @EdgewiseTx for their positive topline results from their Phase 1b In #Becker Muscular Dystrophy #BMD. #Duchenne #DMD study to start later this year. investors.edgewisetx.com/new…