#Breaking
: The U.S. FDA approves the CRISPR/Cas9 gene-edited treatment, co-developed with Vertex Pharmaceuticals, for
#SickleCellDisease
. Learn more:
We have seen unprecedented innovation in biotech in the last 5 years (more than any other prior period). But we need to build real (and disciplined) businesses, not just spew out new platforms. Quality companies will come out even stronger!
#innovationthatmatters
I’m incredibly proud to share that we have completed the submission of our BLAs for the potential first-ever CRISPR-based therapy! Hope this milestone will be the first of many. Congrats to the team
@CRISPRTX
@VertexPharma
and thanks to our investigators and patients on the trial
We are pleased to announce that we’ve completed the submission of our Biologics License Applications (BLAs) to the U.S. FDA for our potential treatment for sickle cell disease and beta thalassemia. Learn more:
One of the most exciting weekends for
@CRISPRTX
at
@EHA_Hematology
2022 meeting in Vienna. Remarkable data in Sickle cell and TDT with CTX001, and new hope for patients with T-cell lymphomas with CTX130. Many thanks to the team, our partners, investigators & patients!
#EHA2022
I’m so proud of our teams for this exciting milestone of completing the first-ever global regulatory submission for a CRISPR-based therapy! This is a historic achievement and thank you to all who have contributed to make this possible.
@CRISPRTX
$CRSP
CRISPR Therapeutics and Vertex are pleased to share that we completed our regulatory submissions for exa-cel for sickle cell disease and beta thalassemia in the EU and the UK in December 2022.
Biomedical progress continues at an astounding pace. Off-the-shelf organs becoming a reality.
@CRISPRTX
we are developing off-the-shelf pancreatic islet cells from stem cells for Diabetes $CRSP
Thankful for the year we’ve had at CrisprTx. Lot of foundational progress and excited for 2022! I will be out there talking about the progress a lot more after staying relatively low-key this year. HNY!
Congratulations to Emmanuelle Charpentier and Jennifer Doudna for winning the 2020
#NobelPrize
for
#CRISPR
. No one more deserving than Emmanuelle and she continues to inspire us everyday
@CRISPRTX
as we work towards bringing this groundbreaking technology to patients!
Proud of the
@CRISPRTX
/
@VertexPharma
team today for the progress on CTX001, and thankful for the investigators and patients taking part in this groundbreaking research. A significant landmark, not just for us as a company but for the whole field
#hemoglobinopathies
#CRISPRCas9
$CRSP and
@VertexPharma
announce early positive safety and efficacy data from the first two patients treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 for severe hemoglobinopathies. Learn more here:
Incredible year
@CRISPRTX
. BLA filing, several clinical trials progressing, 3 new INDs/CTAs, state of the art manufacturing facility and new CRISPR-X group. Patient stories like this one below keep us inspired to do even more!
Despite rounds of chemotherapy and a stem cell transplant, nothing was working for these two cancer patients. Then, doctors tried something new: the CRISPR gene-editing technique.
NPR got exclusive access to tell their stories.
A major loss for all of us. Allan Hoffman (1932-2023) Univ_Washington passed away today. Allan was my academic brother (PhD '57 MIT) and great mentor. Devastated. He was such an inspiration to all of us. All in tears after we received the news about an hour ago. RIP.
Biotech lab employees are essential workers! We’ve had 190 brave researchers
@crisprtx
come into the lab every day for the last year to bring medicines to patients....we need to get them vaccines!
@MassBio
@MassGovernor
6 years since the Charpentier/Jinek publication elucidating CRISPR/Cas9 for gene editing...so much progress in such a short amount of time! CRISPR is going to continue to move much faster than previous tech disruptions...
Thank you
@adamfeuerstein
& bio-verse! Surprised to even be nominated given CRISPR is still in its early stages of growth. The vote of confidence is likely a reflection of the potential of the platform & the wonderful team
@CRISPRTX
, whom I have the joy of working with every day!
We are pleased to announce that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX110™ for the treatment of relapsed or refractory CD19+ B-cell malignancies. Learn more here:
We are honored to receive the Facility of the Year Award (
#FOYA
) in Innovation from the International Society for Pharmaceutical Engineering (
@ISPEorg
) for our flexible, digitally-enabled manufacturing facility in Framingham, MA. Learn more here:
Enjoyed my fireside chat with
@hannahkuchler
this morning on
#FTPharma
. CRISPR as a technology is not just going to transform medicine as we know it today, it is going to completely change the business model for Biopharma companies
@CRISPRTX
#CURES
Thank you
@BlackRock
for having $CRSP CEO
@CrisprSam
on The Bid podcast to discuss how we have advanced clinical trials for CRISPR-based medicines to treat serious diseases including
#SickleCell
,
#BetaThalassemia
and cancer.
We are excited to bolster our manufacturing capabilities and competency! Thanks to
@MassGovernor
and his team for the encouragement and support.
@CRISPRTX
Today $CRSP announced that we are building a new cell therapy manufacturing facility in Framingham, Massachusetts for clinical and commercial production of our investigational cell therapy product candidates. Learn more here:
"We're very passionate about bringing these therapies to patients around the world."
@CRISPRTX
CEO
@CrisprSam
discusses how advancements in cell and gene therapy innovation will shape the future of medicine across the globe.
Learn more:
Exciting day at
#EBMT23
for
@CRISPRTX
. Two Presidential symposia talks - one on Exa-cel by Dr. Locatelli and one on CTX110 by Dr. McGuirk. Kudos to the team and thanks to our investigators
As we get into proxy season, let's begin the annual charade of
@issesg
ISS and Glass Lewis
@GlassLewis
ratings, just so companies can hire them to "fix" ESG issues. Apparently developing curative medicine does not qualify as social good per these ratings agencies
@CRISPRTX
We capped off an exciting week beginning with our ASH presentation in
#Sicklecell
and
#Thalassemia
with a company townhall featuring our scientific founder Emmanuelle Charpentier!
@manue_lab
. It was great to hear from her (& see what the prize actually looks like)!
I am so proud of this recognition (this survey was completely outside-in)! I am honored to be leading such a wonderful group of people, all dedicated to the cause of making transformative medicines!
@CRISPRTX
We’re proud to be recognized as the
#1
employer in
@biospace
's 2022
#BestPlacestoWork
report, among top biopharma employers with <1,000 employees. This survey spotlights companies that life sciences professionals consider top choice employers. Read more:
Exciting day for us
@crisprtx
and our partners
@VertexPharma
with our data presentation at
#EHA25
. We are grateful to our investigators and patients in the study and proud of the team in getting to this milestone.
#Breaking
: Today, $CRSP and
@VertexPharma
announced new clinical data and progress for CTX001™ in severe hemoglobinopathies at the
#EHA25Virtual
Congress. Learn more here:
Today,
@CRISPRTX
and
@ViaCyte
announced that the first patient has been dosed in the Phase 1 clinical trial of VCTX210 for the treatment of type 1 diabetes (T1D). Read more here:
Kudos to our in vivo team! We are going to keep charging ahead with both ex vivo and in vivo gene editing approaches in a number of different indications. Also pleased to strengthen partnership with Vertex
@CRISPRTX
$CRSP $VRTX
@VertexPharma
We’re excited to expand our collaboration with
@VertexPharma
to develop best-in-class gene-edited therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1. Learn more here: $CRSP
What will 2020 bring for medicine and science? - STAT. Excited for what CRISPR will bring and other advances in diagnostics, digital health and most importantly, global public health!
It’s hard to articulate how excited I am to be joining
@CRISPRTX
. I’m looking forward to learning from everyone on the team and hoping to contribute meaningfully to the company’s work.
Great trip to Riyadh. Spoke at
#FII2019
and visited King Faisal hospital with Drs. Alshanqeeti, Alzahrani and Ahmed, a leading site for Sickle cell and Cancer care in Asia. Nearly 60k suffer from Sickle cell disease in KSA
@FIIKSA
Based on our experience with the RMAT and CTX130 IND, I want to thank and compliment the FDA for their proactive leadership and support in the middle of this pandemic
@US_FDA
We are pleased to announce that CTX001™ has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. FDA for the treatment of severe hemoglobinopathies. Learn more here:
We are pleased to invite Maria Fardis Ph.D., MBA to join our Board of Directors. Her extensive leadership in scaling companies and bringing novel therapies to patients will be invaluable as we continue to advance our pipeline and platform. Learn more here:
Happy to be a sponsor for the
#NAACPConvention
in Boston. We are proud to be developing a medicine
@CRISPRTX
for Sickle cell - a disease that has been ignored for far too long
We’re pleased to announce that Dr. Phuong Khanh (P.K.) Morrow, M.D., FACP, is joining us as Chief Medical Officer. Read more about her appointment here:
Economists are missing the fact that inflation is bimodal right now. Traditional measures don’t reflect reality. We should all be giving/donating these holidays!
Outstanding talk by Dr. Ewelina Morawa about CAR-T and CRISPR technology and its potential use in RCC therapy. Challenges in solid tumors. CD70 seems a good target in solid tumors and RCC. CTX130 to go to clinical trials.
#IKCS18
If you're a
@statnews
subscriber, watch video from last night's event on
#CRISPR
here! Featuring $CRS, $NTLA, $EDIT, Beam, KSQ and Synthego, plus Jeantine Lunshof and an amazing audience!
Thoroughly enjoyed the USAIC conf - one of the best meetings on a host of biopharma industry topics
#usaic2019
. Enjoyed having Jim Wilson, Dan Curran, David Lucchino, Dhaval Patel and Tim Yu on the Rare diseases panel that I moderated.
Congrats to the biopharma heroes of the year – Stephane, Albert, Ugur and others – who have achieved the impossible through sheer grit and determination. And much respect to Behzad, Tassos and other biotech CEOs for making 2020 such a productive year for the industry.
We’re excited to welcome Raju Prasad, Ph.D., as Chief Financial Officer of
@CRISPRTX
. Dr. Prasad brings in-depth knowledge of the cell and gene therapy space along with extensive experience in capital markets. Learn more:
Excited about the caliber of leaders we have been able to bring in over the last few months - John Leonard as head of in vivo, John Martin who was top official at the FDA and Julian Fowler to transform digital for us! $CRSP
@CRISPRTX
We are pleased to welcome John Martin as Head of Government Affairs, and Julian Fowler as Head of IT at CRISPR Therapeutics! John and Julian bring unique expertise to our expanding leadership team and company during this phase of our growth.
This is a disease that has been ignored for too long....we are 100% committed to Sickle cell disease and hope that our efforts bring a transformative cure for these patients....
On this
#WorldSickleCellDay
, we’re standing behind the people living with
#sicklecell
disease and their caregivers. We’re proud to work with
@VertexPharma
to advance new treatment options for those living with this devastating disease.
#WSCD2019
I’ve traveled the country to meet with state pension funds, treasurers, and corporations. It is *staggering* how much influence BlackRock & ISS have wielded over these institutions. They captured the system in red & blue states alike. Time to bring new alternatives to the table.
Today
@VertexPharma
opted in to co-develop and co-commercialize CTX001, an investigational gene-editing treatment. $CRSP is excited to partner with $VRTX to develop a potentially transformative therapy for sickle cell disease and beta thalassemia
People across 180 countries will get to see the unknown side of PM
@narendramodi
as he ventures into Indian wilderness to create awareness about animal conservation & environmental change. Catch Man Vs Wild with PM Modi
@DiscoveryIN
on August 12 @ 9 pm.
#PMModionDiscovery
$CRSP and ViaCyte announce strategic collaboration to develop gene-edited stem cell-derived therapy for diabetes, accelerating CRISPR's efforts in regenerative medicine. Learn more here
Many thanks to
@FTLiveHealth
for including our own
@Sam_S_Kulkarni
in a discussion today about delivering on the potential of advanced cell and gene therapies at the FT US Pharma and Biotech Summit.
#FTUSPharma
Distributed innovation with CRISPR is going to be the basis of a competitive advantage over other platforms. Publications from both coasts today on CRISPR for hearing loss and ALS
Okay, here are my prime editing takes. I'm mostly going to try to be a voice of reason here. Not because I'm hyper critical of the paper but because it's necessary. This field is full of mega hype and cool tech, but cool doesn't get us anywhere unless it works as advertised.
@CRISPRTX
is proud to be a founding member of the inspiring
@LS_Cares
! We were honored to join over 500 others in the Biotech community at the 2019 Impact Breakfast. We look forward to continued partnership to make a difference in the greater Boston community.
#LSCImpact
We were thrilled to host our 2019 Volunteer Day this week in partnership with
@LS_Cares
and
@c2cboston
, making over 80 blankets, 40 Happy Hope Bags and sorting donated clothing, impacting over 1,000 children in the Greater Boston area. Thanks to all of our employees!
Great thought leadership on future of CRISPR from our Scientific founder, Matt Porteus
@Stanford
at the Senate HELP committee hearing today $CRSP
#CRISPR
A lot of work by a lot of people led to opening this lung cancer adjuvant study. 3-arm randomization to SOC chemo vs SEQUENTIAL chemo/pembro vs CONCURRENT chemo/pembro. Now open!
Sad news! The Sumatran
#rhino
– the world’s smallest rhino – has become extinct in Malaysia after the last surviving individual died in captivity
#RacingExtinction