Pearl Freier
@PearlF
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Founder of Cambridge BioPartners, Inc.
Cambridge, MA USA
Joined March 2009
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New Journal of Clinical Oncology
@JCO_ASCO
paper estimated potential savings of $661.8M for US taxpayer-funded Medicare if replace current Medicare Part D median formulary prices w/
@costplusdrugs
pricing for 7 generic oncology drugs. h/t
@RuchikaTalwarMD
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@nikillinit
Wow, and he's lucky his father is still alive but so scary that this is what it took to prove who the real person is
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@chrissyfarr
Also some women can’t breastfeed for medical reasons. The shaming should have stopped years ago.
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Here's
@ScottGottliebMD
WSJ piece with a helpful explanation on the differences in protection between masks. "A real medical-procedure mask will be cleared by the Food and Drug Administration and designated as offering one of three levels of protection."
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Baylor/
@PeterHotez
: because of SARS in 2003, we knew the spike protein was going to be the target. The important news from Pfizer & Moderna is that indeed that was the right target. All that happened because of 15 yrs of basic research. And then we had CEPI
#WVCWC
.
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CVS/Aetna/Caremark CEO has a book on leadership coming out next week. From one of the early reviews on Amazon: "You need a prior authorization to read this book....."
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Privately held healthcare software company Epic has said that 78% of all patients in US hospitals/&related practices use their medical records software. They announced GPT-4 partnership w/ Microsoft at
#HIMSS23
& plan to integrate generative AI into Epic's
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@KarlaValliere
@GMA
Does the writer of the
@GMA
story last August,
@yijiny
, know the update that now $CVS Caremark is denying access to the medication the baby has been on previously & needs immediately. cc
@RobinRoberts
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The level of revisionist history is stunning. I remember some of the Admiral's Sunday morning show interviews last yr & CNN should find clips where he said US was prepared, enough testing etc. Leaders need to be held accountable instead of blaming group out of office for 3 yrs.
Level of revisionist history going on tonight on CNN is stunning
Especially from Giroir -- who spent all last spring saying we had plenty of testing, which was plainly untrue
Now he says system was unprepared?
May be level with the American people last year when it mattered?
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This explains so much....this has to change. Check out the newsletter below for more information. Both debates so far this year were poorly run by the commission in the year of the most consequential US election since President Lincoln's era.
Frustrated by the debates?
Well here’s who is in charge...
The three co-chairs of the debate commission are all white, two of them are 81-years-old, and the other one is 71-years old.
Read more in my newsletter:
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@EricSchultz
Whether they respond or not, there’s still no accountability. No resignations. They can also accept gifts from billionaires and rule in their favor and it’s somehow not considered an ethics violation.
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@drkomanduri
@chrissyfarr
It also seems like more of these articles started come out when the infant formula shortage became a crisis. Many women were shamed for buying formula & blamed for the shortages.
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@michaelmina_lab
Decision by CA DPH also makes it more difficult for many US workers to get paid (or unpaid but not held against you by employer) sick days if rapid test is positive.
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How many US biotech (drug R&D) companies are led by a female CEO now? Reshma/Vertex, Yvonne/Alnylam, Jackie /Agios, Daphne/Puretech, Aoife/Synlogic, Ivana/Vigil, Rene/Xilio, Helen/Halozyme, Barbara/Tango, Liz/UroGen, Melanie/EQRx, Sharon/ITCI, Jennifer/MiNK, Zandy/MeiraGTx 1/n
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It's a good day so far for female biotech CEOs: Carmot Therapeutics CEO Heather Turner leads acquisition to Roche for $2.7B upfront & potentially up to $3.1B; Jo Viney-led Seismic Therapeutic announces $121M B round, & Sandra Glucksmann announced as new CEO of CNS biotech
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4% of drug discovery projects made it to Phase 1 clinical trials & only 8% of that 4% progressed through clinical trials to regulatory approval & drug launch—see slide below from Johnson & Johnson EVP Bill Hait's
#AACR24
presentation in San Diego today.
J&J’s William Hait highlighting the burden on pharma to launch products : 40 phase 1 ➡️ 3 BLAs! Hence $2B+ per drug launched.
#AACR24
“Path to Entrepreneurship” session
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Vertex Pharma's small molecule VX-659 for cystic fibrosis patients took three years from first synthesis till completion of phase 3 studies, said CEO Jeff Leiden in JPM presentation. He said that it may be a world record in this industry. $VRTX
#cysticfibrosis
#cf
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FDA approved 56 new/novel drugs so far in 2023 vs 37 in 2022. 31 of 56 new drugs approved are pills/small molecules (incl 1 radioactive diagnostic agent). 17 of the 56 are for oncology. From L.E.K.'s Daniel Neumeier's post:
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Genentech announced that interim CEO Ashley Magargee will be full-time CEO starting Jan 1. Genentech's 1st female CEO in its 47 year history. Ashley has been at Genentech/Roche since '04. Prior to becoming CEO, Ashley was Genentech's SVP & Head of Commercial Porfolio & SVP
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@OliverStacksMD
@nikillinit
My guess is that one reason they didn't do it earlier is because they didn't believe the real person because he was homeless & the thief had the documentation he needed to open the bank account there. The thief, on the surface, looked legit & even had a legit work
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Here's former Roivant CEO suggesting that school shootings & murders like Sandy Hook aren't real in the US
We live in a nation now where liars and frauds who run for president are allowed - without consequences- to suggest school shootings are orchestrated as part of a conspiracy to pass stronger gun laws.
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Ex Roche VP Kapil Dhingra was asked by Torreya's Mark Simon at last week's
@CancerProgress
conference about the increase in CRLs (complete response letter) from FDA to biopharma companies. Kapil: Yes, the FDA is getting tougher on a number of issues. Some of these are new 1/n
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The White House released a statement on the new treatments for sickle cell disease last week & didn't acknowledge any of the companies, researchers & investigators involved with these major achievements. They also didn't thank the patients who volunteered as participants in the
After the FDA approvals of 2 new groundbreaking cell-based gene therapies for sickle cell disease on 12/8, the White House released the following statement:
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As more biotech layoffs become publicly known, take note of how layoffs were handled & how the founding investors acted. Did scientists' receive any severance? What happened 2 health insurance? Any assistance w/ outplacement? Look 4 the founders that took care of their employees.
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If you're a scientist considering a job at a newly announced biotech, look past funding news & that CEO is an alum of a famous lab because numerous are. Look up how CEO's previous VC-backed startup treated employees & how operations wound down. If you know scientists who 1/n
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@jemelehill
I think the media has also mostly missed one of the biggest challenges for small business owners/restaurateurs is that the cost of healthcare & health insurance has risen dramatically over the last 10 yrs—as insurance increased profits while refusing to cover typical needs for
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Regeneron launched its 1st corporate VC fund & announced plans to invest $100M/yr for 5 yrs in biotech, devices, tools, & "enabling technologies." Regeneron Ventures is based in Greenwich, CT & general partners incl ex $REGN execs Michael Aberman & Jay Markowitz. Congrats 1/n
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If your medication is available through
@costplusdrugs
, stick with them & keep supporting Cost Plus Drugs. Many pharmacy counters across the country (46 states & DC) now accept the Team Cuban Card for the discount on your medication
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Ex Stanford president had hired 1 of Elizabeth Holmes' ex attorneys to try to block publication of Theo's articles in Stanford's independent newspaper--according to LA Times Q&A w/ Stanford undergraduate student journalist Theo Baker by
@corinnepurtill
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Police in Hopkinton, RI twice denied a resident's application to buy firearms due to a cyberstalking charge, his mental health history, & previous arrests. After his lawyer cleared the way, he bought guns & killed his prev abused wife. cc
@shannonrwatts
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One of ex Roivant CEO's ideas is to rename the US if he gets elected, the United States of Vant. Each state will be a vant too. Massavant, Connectivant, Rhode Islavant
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Mark Cuban's
@costplusdrugs
launched Thurs selling >100 generic drugs directly to consumers. They set up its own registered pharmaceutical wholesaler & bought drugs directly from manufacturers with no PBMs in middle to jack up prices. Cash pay only . 1/n
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Roche is planning to launch its antibody test in May. CEO Severin Schwan said they scrutinized other antibody tests in developing their own & commented on others' reliability: "It’s a disaster. These tests are not worth anything, or have very little use, "
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Vertex CEO Jeff Leiden on their cystic fibrosis drug Trikafta: "It was less than 4 yrs from the synthesis of that compound in a laboratory in San Diego to FDA approval. I have never seen another example of that in my 30 or 40 yrs in the industry." comments during
#JPM20
last week
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@MikeIsaac
also what if they did send a thank you email but to a hiring manager who may not have mentioned it or what if the thank you email went in someone's spam folder
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Vertex co-founder & ex CEO Josh Boger ('89-09) was 1 of the interviewees on a recent Planet Money episode. He was a chemist at Merck before a VC called him & he left to start Vertex. "I was making 25 to 50 compounds a yr by hand, 1 at a time. We were told
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@mmitchell_ai
The two female board members (Tasha & Helen) were involved in ousting the female interim CEO (Mira) at some point this past weekend when they hired the new male CEO on Sunday
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3 PBMs/prescription drug insurance companies (CVS Caremark. Optum/United & Express Scripts/Cigna) control >70% of all drug prescriptions in the US. FTC said today the PBMs haven't been cooperating w/ their investigation & it's been 2 yrs ... how much more evidence does FTC need?
"Every day we are forced to fill prescriptions for $20, $50, $100, $200+ under our purchase price,” the company stated on Facebook. “Every year the contracts offered by the PBMs get worse and 2024 is the worst yet.”
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Cambridge, MA officials are urging all residents (incl companies) to wear masks again indoors as covid cases r rising. They said 42% of new cases r breakthru cases among fully vaccinated. 67% of residents r fully vaccinated as of 7/15; 73% received 1 dose:
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A biotech's journey in 6-7 yrs. Launched w/$65M A & single cell genomics platform in '18. Fierce 15 winner in '19. Laid off 75% of staff 8/23. Cambridge, MA-based Celsius Tx was down to 1 person to see thru Phase 1 IBD clinical trial before deal 2b acquired by AbbVie for $250M
What a ride. We launched
@celsiustx
in 2018…co rode the biotech rollercoaster…eventually down to one person to see thru their Phase 1 IBD trial for its TREM1-focused drug candidate…and now
@abbvie
has swooped in and picked them up. Kudos to
@tkassum
for seeing it through.
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Mark Cuban was asked by
@halletecco
what most shocking moments were over 3-4 yrs of starting new online pharmacy 4 generic drugs cheaper $ than insurance co-pays. He said seeing how entrenched PBMs are. He said PBMs' “cover charge” on insulin went from 3% of the price to now 50%
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Tonight's 60 Minutes piece on ongoing drug shortages at US hospitals—most days, nearly 300 essential drugs can be in short supply. Incl glucose for NICU babies. A $5 chemotherapy drug is tough to get even though needed for cocktail w/ 80-90% cure rate
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I still can't believe the FTC did this. What happens to the clinical trials for MZE00 (glycogen synthase 1 (GYS1) inhibitor program) for Pompe disease now? Who will pay for the clinical trials for this rare patient population now?
Wow, FTC is intervening in a small biotech collab w/ a French pharma that could potentially help patients living w/ Pompe disease. Biotech companies need collabs w/large cos & the $ to develop new drugs:
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Several biotech startups that announced >$100M rounds sought strategic alternatives or laid off most of their employees less than a yr later. it's not surprising anymore. Some AI startups are going through this too. If a tech company's corporate VC was one of the biotech's lead
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One of the most prominent CEOs in the founder-led biotech CEO community is short seller Scorpion Capital's latest project: "If Bernie Madoff and Elizabeth Holmes had a secret love child, it might resemble the scam we expose tomorrow at 730am."
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Christopher & Dana ReeveFoundation
@ReeveFoundation
announced they've hired their 1st Chief Scientific Officer- rare disease expert Ethan Perlstein
@eperlste
. They're also raising $50M fund 2 invest in spinal cord injury cos . by
@RebeccaDRobbins
Congrats!
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"Medicinal chemist—heal thyself!”: Bristol Myers Squibb senior principal scientist
@MoslinRyan
living w/psoriasis helped discover BMS' psoriasis drug Sotyktu. 12 yrs of research. 1 of the pill's inventors is now a patient on the drug. FDA‐approved 9/22 1/n
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I saw today when reading one of the Biogen stories that's there's a biotech company in Newton MA called Soufflé Therapeutics. I don't know anything else about the company but is this a new biotech naming trend? Quiche Therapeutics, Macaron Biotech, Escargot Therapeutics :)
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The closed down Lord & Taylor stores in Natick, Burlington & Braintree, MA are being converted into life sciences facilities. The Neiman Marcus store at Natick Mall is going to be converted into lab space since the sale.
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Pfizer licensed their Covid oral drug, known as brand name Paxlovid, to The Medicines Patent Pool= enabled 35 generic manufacturers 2 sign deals w/MPP to produce low-cost & generic versions in combos w/ritonavir 4 supply in 95 low & middle income countries
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@GergelyOrosz
Seems surprising that Quora's CEO, 1 of the 4 board members, thought yesterday's actions were a good idea
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Derek Lowe wrote a column on AI & Xaira Therapeutics & substantial bet their team & investors are making "that AI techniques can somehow transform drug discovery." He explained why he believes other AI for drug disc companies haven't been successful yet:
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A biotech company with 3 FDA-approved cell-based gene therapies only has cash until Spring ‘24 according to below
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Research from University of Cape Town in South Africa: "Saliva swabs are the preferred sample for Omicron detection"
Important preprint.
“Saliva swabs are the preferred sample for Omicron detection”
TL;dr:
for Delta variant, nasal swabs 100% positive percent agreement. Saliva 71%
**for Omicron variant, nasal swabs 86% positive percent agreement. Saliva 100%**
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Biotech & healthcare investors, whose funds manage $68B, said latest proposed Senate bill would cause 235 companies to lose funding. They estimated this impacts 800K jobs in US. 87 CA companies, 58 in MA, 25 in NY. Some listed for cuts incl founder-led biotechs. See pgs 21-31 1/n
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An update on world's 1st CAR-T cell therapy for a child (teen) with lupus: "1 year after the treatment, I feel as good as I did before my diagnosis, except for a few colds." 15 yr old received CAR-T cells at Universitätsklinikum Erlangen in Germany in '23.
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Regeneron VP & Chief Data Officer
@jgreid
was asked by
@talkingbiotech
’s
@kevinfolta
if he had to recommend something to other scientific teams that were pursuing greater digitization or data augmentation, what would that be? Jeff: I would say that 1 thing that frustrates 1/n
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The White House is hoping 2 get $8B in funding for sequel to Operation Warp Speed—Project Covid Shield would be for next generation Covid vaccines which might finally get clinical data on whether nasal vaccines will be better at reducing Covid transmission
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Their belief is that covid-19 vaccine booster will be needed in the fall: 6-8 months post-vaccination (1st generation) especially since masks are off and back in the office. This is their opinion. FDA & CDC aren't in agreement yet but stay tuned for more data.
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"Biotechs are equipping CAR-T cells to destroy B cells — not to treat blood cancers, but to take on multiple sclerosis & a raft of autoimmune disorders," from
@NatureBiotech
story that incl 1st clinical
@Kyverna_Tx
& MS breakthru published in March $KYTX
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"My goal is not to make a trillion-dollar company,” healthcare VC
@rtnarch
told WSJ's
@GZuckerman
. “It’s to profoundly restructure a reactive broken industry into a curative industry that has profound impact on humans." (gift link)
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Boehringer announced a new chronic liver disease partnership w/Oxford, UK-based biotech &
@ycombinator
alum
@OchreBio
. $35M upfront & near-term payments & up to $1B if milestones met. CEO is
@Jack_O_Meara
, CSO
@QuinWills
,
@jackcastle
is Head of Corporate Development. Congrats!
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"Before the vote, Peters said he opposed the legislation on the grounds that it would stifle future investment in drug development. He cited a Sept. 8 letter in which >400 biotechnology investors argued that the bill...""
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Here's more discussion between CBS News
@margbrennan
&
@ScottGottliebMD
in 2nd 5/21 video on drug shortage crisis: "The reimbursement for these drugs under government programs has been driven down very low, something above the marginal cost of
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BioCentury's
@StephenPHansen
on pod: "60% of Nasdaq-listed biotechs are now under $200 million" but also oppty he said in this market 2 find homeruns w/reminder that at 1 point Genmab traded below $200M & now is a $25B company. Pharmacyclics traded at $1 & later sold for $21B
"Nearly half of all Nasdaq-listed biotechs have less than 18 months of cash. A little more than 20% of biotechs on Nasdaq are trading below a buck," said
@jeff_cranmer
on
@BioCentury
podcast yesterday.
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Congrats to Novartis alum
@TheDawnBell
who is CEO of a new biotech startup, Antila Bioscience, developing long-acting biologics for patients w/severe cardiovascular disease & they raised $12M seed backed by Curie Bio (
@c_lengauer
,
@zachweinberg
) & F-Prime. Public update below:
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Rhode Island has a similar-sized population as Bhutan. According to press, Bhutan vaccinated 90% of their eligible population in a week. Rhode Island still has 40% of their population listed as unvaccinated h/t
@meganranney
cc
@ashishkjha
A few months ago we read about the early vaccination success of the kingdom of Bhutan. Now, it has done it again, fully vaccinating 90% of its eligible population in just one week — many in hard-to reach areas. Tashi Delek to our Bhutanese friends.
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A new term sheet template 4 early-stage life science startups spun from academic research labs is now avail after >2 yrs of work by individuals from university tech transfer offices, VCs, & law firms. Could slash time & cost of licensing IP 80%
@BioCentury
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Johnson & Johnson/Janssen released their US Pricing Transparency Report. They said they paid $11.2B in '22 to commercial payers (health insurance) & pharmacy benefit mgrs (prescription drug insurance aka PBM). What would happen if J&J didn't pay this amt?
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National security expert from Boston
@juliettekayyem
interview discusses WH's efforts for coronavirus & pandemics: "lack of planning from American health officials that may put us in more danger than the disease actually warrants
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Physician scientist
@RonDePinho
has co-founded several biotech companies incl 2 cancer drug companies w/FDA-approved products (KPTI & AVEO) He shared advice 4 academics starting a biotech company incl capital funding needs 4 IND-enabling studies $12-$15M cost/asset small molecule
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"The kidney came from a pig engineered by the biotech company eGenesis, [
@eGenesisBio
] which removed 3 genes involved in potential rejection of the organ. In addition, 7 human genes were inserted to enhance human compatibility. Pigs carry retroviruses that may infect humans, &
"Surgeons in Boston have transplanted a kidney from a genetically engineered pig into an ailing 62-year-old man, the first procedure of its kind. If successful, the breakthrough offers hope to hundreds of thousands of Americans whose kidneys have failed."
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Deloitte report said the cost to take a drug candidate from discovery stage all the way to market launch hit $2.3B (if it makes through clinical trials & FDA approval)—a $298M increase from 2021, & below 2019 & 2020 levels they said
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Pfizer received its first-ever FDA approval for a gene therapy. Beqvez is a new 1-time treatment for eligible adults living w/ hemophilia B. $PFE's CSO Mikael Dolsten comments on Linked In incl: "The road to this breakthrough has been a 40+ year effort in which we have worked to
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CBS News
@margbrennan
asked
@ScottGottliebMD
about the cancer drug shortage crisis (incl chemo drugs carboplatin & cisplatin) on 5/21. comments at 9:20: "The problem is that these drugs are manufactured at such a low margin, that the only way to do it
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More from
@LifeSciVC
on AI: I think true generative AI—coming up with brand new molecules never before seen by man, I think that remains elusive. A lot of the discovery-focused AI shops are mining patent literature and adding methyl groups here and there to existing things—and
Great conversation between Simone/
@biocentury
& Bruce/
@atlasventure
. On impact of AI on drug discovery/development,
@LifeSciVC
said: I think anyone today has to acknowledge the power of AI & machine learning to solve specific problems here. I do worry how far hype has gone beyond
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@teddyschleifer
Are they going to have a telemarketing class w/scripts training people how to ask their parents & grandparents to donate to the campaign :)
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I went to CVS a few hours ago to pick up a prescription but pharmacy closed a few min early & the pharmacist told me on her way out I could get the prescription at another CVS pharmacy that was still open. Went to 2nd CVS- pharmacist told me that since the 1st CVS pharmacy 1/n
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Amazon is now selling prescription medicines in US- Amazon Pharmacy. "Prime members get discounts of up to 80% on generic & 40% on brand-name medications when paying without insurance." They said you can see if insurance or paying out of pocket is cheaper
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Pfizer board member Scott Gottlieb update on potential availability for covid vaccines for ages 5-11 & ages 2-5. Data for 2-5 will be avail in Nov he said. Ages 5-11: data to be filed w/FDA sometime Sept, application filed as early as Oct. 4-6 wks review?
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@MikeIsaac
In any case, Missouri just became a blue state :) I'm seeing residents tweet that.
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A pregnant woman who carries the gene for cystic fibrosis was given Trikafta at 24 wks by her medical team at Stanford Medicine Children’s Health’s Center for Pregnancy & Newborn Services who worked w/her insurance company for coverage. After the baby was
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@jayrosen_nyu
@mendyboyd
It's scary that some of the major news organizations still haven't learned lessons from 2016.
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FDA approved new "glowing tumor" imaging drug, Cytalux,"which is attracted to ovarian cancer tissue & illuminates it when exposed to fluorescent light, allowing surgeons to more easily find & more precisely remove the cancer."
@PennCancer
@OnTargetLabs
1/n
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Johnson & Johnson's Chief Data Science Officer & SVP Strategy & Portfolio Org R&D Najat Khan, PhD announced her resignation from J&J after 6 yrs to join techbio
@RecursionPharma
as Chief R&D Officer & Chief Commercial Officer. She was appointed to $RXRX's board of directors too
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NY Gov Cuomo answered "when will this be over" & said that testing will be the key."You tell me when they come up with an inexpensive home test or point of care test that can be brought to volume I think that's probably when you see a real return to normalcy in the workforce. "
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A reminder of the history of profitable biotech companies by
@cngarabedian
during
@thebiotechclub
presentation. It's a very small group. Chiron, Centocor, Genentech, Immunex, Genzyme were acquired >10 yrs ago. Alexion merged with AstraZeneca last year.
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Please don't support or use the narrative that the patient "failed" when a drug didn't work. It probably wasn't intentional on her part to label the patient as a failure but words matter.
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On biotech burn rates, according to below in '22 it could cost $130k per patient in autoimmune disease clinical trials & $250k per patient in oncology Phase 1 or Phase 2 clinical trials.
What's the average external cost per patient in clinical studies? The numbers we are seeing right now are jaw dropping: $130k per patient in autoimmune diseases, $250k in oncology P1/2. It can def explain some of the crazy burn rate biotechs report...
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