U.S. FDA cleared our IND for PM577a, our investigational in vivo Prime Editing therapy for H1069Q-mutated Wilson disease.
With our NZ CTA, this establishes a global Phase 1/2 program & positions us to advance PM577a.
More https://t.co/SbGYoCGgHo
#PrimeMedicine#Wilsondisease
U.S. FDA cleared our IND for PM577a, our investigational in vivo Prime Editing therapy for H1069Q-mutated Wilson disease.
With our NZ CTA, this establishes a global Phase 1/2 program & positions us to advance PM577a.
More https://t.co/SbGYoCGgHo
#PrimeMedicine#Wilsondisease
Stealing IP and trade secrets from the biotech platform companies is nothing new.
What makes it even more nefarious and lethal to the pioneers is when it further leverages structural cost advantages such as in #China.
If you e.g. support $BOLD as it rips off $prme (struggling to raise even a fraction of what boundless greed gets thrown after) and $beam, it's all I need to know about what the social contract really means to you: you make money, everybody else loses it.
Experts in gene editing, n=1 medicine development, in vivo delivery, rare diseases, and cell & animal models of genetic disorders have founded the Center for Therapeutic Genetics (CTG), a non-profit effort involving @broadinstitute@BostonChildrens@jacksonlab, as well as others from industry, academia, and patient advocacy organizations, to bring the promise of programmable medicines including base and prime editors to patients suffering from rare genetic diseases. This article from @ginakolata@nytimes frames the compelling need: https://t.co/OyrYCZtWIk
Official announcement: https://t.co/MwJCUarQnX
My read on the Beam-Prime arbitration:
This is clearly a very positive outcome for Prime Medicine. For Beam, it is slightly negative, but the real short-term impact is probably small.
The original 2019 Beam-Prime agreement was made when prime editing had just been invented. The basic split was: Beam had exclusive rights to use prime editing for transition mutations, the kind of edits base editing can already do, plus sickle cell disease. Prime had rights to other mutations. In return, Prime got important help from Beam in its founding, plus manufacturing and delivery related licenses and know-how.
But neither side could fully foresee how prime editing would evolve. Later PE4/PE5-style ideas made it common to install nearby silent edits to evade mismatch repair and boost efficiency.
That is why Prime’s AATD program became an interesting edge case. The disease mutation, E342K, is a transition mutation. But to get higher efficiency, Prime’s design appears to install nearby silent non-transition edits. So technically, it is not transition-only.
The arbitration ruled that PM647 is within Prime’s field and that Prime did not breach the agreement.
The practical consequence is that Prime likely has much more room to pursue transition-mutation diseases than previously assumed, if the actual prime-editing design includes non-transition silent edits, which it may want to do anyway for efficiency.
Another consequence: Prime will have to defend its prime-editing IP position against other companies using similar prime-editing approaches. More freedom also means more responsibility.
For Beam, this is not ideal. But I do not think it changes the AATD race much in the short term. Beam is already well ahead clinically with BEAM-302, and the data so far look very strong. Other companies, including Prime, can still enter AATD, but it will be hard to prove better patient outcomes than BEAM-302 unless BEAM-302’s longer-term profile weakens.
Also, Beam is not actively advancing any disclosed prime-editing program right now. So practically speaking, freeing Prime to use prime editing more broadly may be better than leaving it stuck in legal uncertainty.
If Beam later wants to use prime editing, it should still have rights for transition edits and possibly transition-only silent MMR-evasion edits. If it wants broader non-transition coverage, or wants to install non-transition silent edits in a prime-editing design, my understanding is that it would need to pay additional fees.
So my bottom line:
Prime gets a major win and removes a big overhang.
Beam gets a slightly negative outcome, but the near-term commercial impact is limited.
In AATD, Beam remains clearly ahead clinically, and competitors will need to prove they can match or beat an already very strong BEAM-302 profile in actual patients.
$BEAM $PRME
We announced a positive resolution of our arbitration with Beam Therapeutics, confirming our right to develop and commercialize PM647, our investigational Prime Editing program for AATD, a disease with no approved disease-modifying or curative treatments.
We announced a positive resolution of our arbitration with Beam Therapeutics, confirming our right to develop and commercialize PM647, our investigational Prime Editing program for AATD, a disease with no approved disease-modifying or curative treatments.
$PRME 🔥 👀 at some point we will see a gene editing bull market. We cannot cede this to China. Reminder: prime editing is the leading and most advance gene editing platform and Prime Medicine owns the IP.
Prime editing has continuously improved since @davidrliu's lab introduced it in 2019. 3 new studies from his lab advance prime editing systems, addressing key bottlenecks by increasing the efficiency and improving its potency when delivered into the body. https://t.co/r9qYb2eaXb
@EliKorey The bump was driven by $PRME getting added to the Russell 2000, not platform/regulatory news. With upcoming $PRME catalysts and industry momentum, seems short sighted to me.
Proud to have sponsored the CGDAA Clinicians & Coffee session at the #IDF National Conference on June 26.
Great to support connection and collaboration in rare immune disease care.
#RareDisease#Immunology
Join us at #BIO2026 in San Diego!
Matthew Hawryluk, PhD, Prime Medicine’s CBO, will speak on:
Unlocking the Uncommon: Accelerating CGTs for Rare Diseases
Tuesday, June 23
1:45–2:45 PM
Room 32AB
#CellAndGeneTherapy#RareDisease#Biotech
Another important milestone: FDA has granted RMAT designation to PM359 for p47phox-deficient CGD — based on Phase 1/2 data published in NEJM. PM359 now holds RMAT + Fast Track + Orphan Drug + Rare Pediatric Disease Designations. #GeneEditing#RareDisease#FDA#PrimeEditing
Another important milestone: FDA has granted RMAT designation to PM359 for p47phox-deficient CGD — based on Phase 1/2 data published in NEJM. PM359 now holds RMAT + Fast Track + Orphan Drug + Rare Pediatric Disease Designations. #GeneEditing#RareDisease#FDA#PrimeEditing