Excited to share full data for BEAM-301 in GSD1a, covering the first two dose cohorts
** Importantly, 2/3 patients at the 2nd dose level achieved maximum fasting duration and no longer require corn starch supplementation after a single dose of BEAM-301
globenewswire.com/news-relea…
Exciting updates on gene editing support from the U.S. Government!
We didn’t plan this… but just a few hours after our conference call outlining Beam’s vision to deliver increasingly customized genetic correction therapies to patients, even more news hit
In sickle cell disease, CMS CMMI is already working on behalf of 33 states to standardize access and reimbursement to these critical therapies
And now they want to expand this model to support other one-time genetic medicine products = great news
This press release from HHS brings it all together:
hhs.gov/press-room/fda-cms-a…
Thank you to @SecKennedy@DrOz@FDA_KyleD CBER Director Karim Mikhail, and especially to all the rare disease patients, families, and advocates who champion more progress for genetic diseases
Excited to share full data for BEAM-301 in GSD1a, covering the first two dose cohorts
** Importantly, 2/3 patients at the 2nd dose level achieved maximum fasting duration and no longer require corn starch supplementation after a single dose of BEAM-301
globenewswire.com/news-relea…
The predictability of base editing makes this work:
Different mutations
Same target gene
Same disease pathway
Same mechanism (base editing)
Same LNP delivery
Same in vivo effects (normalizing phenylalanine levels)
Putting it all together –
the predictability of base editing,
internal manufacturing for dose production,
and FDA rules that can accelerate our platform –
we see an important potential opportunity to treat many more patients with genetic correction
Great interview by @statnews with @moderna_tx CEO Stephane Bancel.
I’ve had this feeling before for RNAi: “It was always clear to me that mRNA was going to work for nothing, or a lot of things.”
Moderna's CEO Stéphane Bancel on what's next after the recent success in cancer vaccine, and how that impacts the company's business strategy. trib.al/fn9nyhn
Wonderful in-depth story here from Bloomberg @business about Beam’s efforts to provide a one-time genetic correction for Alpha-1 Antitrypsin Deficiency (BEAM-302)
Amazingly, author Gary Sernovitz is himself an Alpha-1 patient, who is learning about this genetic disease and the emerging options in development to treat it
I really enjoyed getting to know Gary, who brought so much curiosity and perspective to researching this story
His inspiring conclusion, finding himself recently diagnosed with a genetic condition that now sits at the frontier of a new kind of medicine:
“I find myself in a cohort who worry about our liver and lungs… and who are the ideal pioneers for a mind-boggling medical advance. I suspect many are like me, eager to put our body to the test and accept our unasked-for role in the history of gene editing. We have good luck in our bad luck.”
(subscription required)
bloomberg.com/news/features/…