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jobsearchtips02 · 4 years

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This Start-up Might Lastly Cure Sickle Cell Disease– After A Century Of Racist Overlook

The uncomfortable blood disorder, which mainly affects Black individuals, is simply among thousands of uncommon illness that might be cured by Beam Therapies’ advanced gene modifying technology.

B eam Therapeutics CEO John Evans likes rocket launches– and not just the ones that are successful.

Gene modifying, Evans believes, is on a comparable trajectory, poised for a series of successful launches in the years ahead. “The stuff we can do now in genome editing would have seemed like magic five or 10 years back,” he says, as technological developments suddenly have biotechs completing to treat unusual genetic diseases long out of reach.

The first generation of gene editing was Crispr, an innovation developed in 2012 that can target and cut areas of DNA like a set of scissors. Beam’s technology, which has yet to be tested in human beings, might theoretically cure thousands of the hereditary illness caused by single letter misspellings, known as point anomalies. Topping the company’s targets is sickle cell disease– a point mutation that primarily affects Black people and has actually been disregarded through more than a century of racist mindsets.

Beam, which was founded in 2017, went public in February and now has a market cap of $1.5 billion despite having no revenues and losing $95 million over the previous year. What the business does have is 12 research programs for 10 various uncommon diseases, consisting of beta thalassemia, another inherited blood disorder, and two types of blood cancer.

Sickle cell disease is the most promising chance. It’s the most typical acquired blood condition in the United States, impacting about 100,000 individuals. The disease produces unusual hemoglobin, the molecule that assists red blood cells bring oxygen throughout the body. While regular red cell are shaped like doughnuts, sickle cells appear like pointy crescent moons (therefore the name), which block capillary and cut off oxygen supply to the bones and organs, causing “excruciating pain,” explains Dr. Robert Liem, director of the comprehensive sickle cell program at the Lurie Children’s Health center of Chicago.

John Evans, CEO of Beam Therapies.

ARCH VENTURE PARTNERS

With one out of every 365 Black children born with sickle cell, the illness has an ugly racial history. “When individuals who were not Black had sickle cell illness then the societal presumption was that then that person wasn’t entirely white,” even though the illness is found in individuals of lots of other ethnicities.

However racist mindsets and a lack of federal funding still continue today. Sickle cell clients, who can end up in medical facility emergency departments with severe pain, waited, usually, 25%longer than basic patients and 50%longer than clients with bone fractures to be seen, according to a study in the American Journal of Emergency Medicine Sickle cell received an average of $812 in federal research funding per individual over the last years, while cystic fibrosis, a lung disease that mainly impacts white individuals, got more than $2,800 per individual, although sickle affects three times as many individuals, according to a paper in JAMA Network Open “If we really have to have this dispute about whether or not Black life matters, I think we require look no more than sickle cell and how it’s been treated as a medical condition to indicate that it truly does not,” states Bediako.

Sickle cell was the first “molecular disease” found, revealing how a modification in one single amino acid could interrupt blood and oxygen supply to the entire body. The first drug to deal with sickle cell, hydroxyurea, wasn’t approved until 1998, even though the illness has been understood in the medical literature considering that 1910, with three more drugs coming to market since2017

Base editing was originated in 2016 in the lab of David Liu, a core professor at the Broad Institute and a teacher at Harvard University. Liu cofounded Beam in 2017 together with Feng Zhang, a teacher at the McGovern Institute at the Massachusetts Institute of Innovation and core professor at the Broad Institute, and Dr. J. Keith Joung, endowed chair in pathology at Massachusetts General Healthcare facility and teacher at Harvard Medical School. This is the second of three gene modifying business the clinical power trio has actually established considering that 2013, along with Cambridge-based Editas Medication, which is establishing Crispr-based therapeutics, and Durham, North Carolina-based Pairwise Plants, which is utilizing both Crispr and base editing to develop more healthy crops. In 2019, Liu founded Prime Medicine, a 3rd generation “search and change” genome modifying technology, which he likens to a word processor.

” I definitely, in my wildest dreams, never ever envisioned this sort of precise capability to modify the genome,” says Dr. Francis Collins of the National Institutes of Health.

Liu calls the human genome the “essential present your moms and dads ever gave you.” It’s comprised of 6 billion mixes of four letters referred to as bases: A, T, G, and C. An individual with sickle cell illness has one base set misspelling at a vital place in their adult hemoglobin gene: a ‘T-A’ where there should be an ‘A-T’. That typo, which appears two times among the 6 billion letters, is the distinction between typical hemoglobin and the unusual hemoglobin that causes the stiff crescent-shaped cells.

Beam is the first company to try to directly repair the base set misspelling, though they can’t yet switch a T to an A. Rather, Beam changes the T to a C and the A to a G. While it appears odd to swap one typo for another, the brand-new typo mimics a natural phenomenon, called the Makassar version, which leads to functional red cell rather of the sickle shape.

Beam is also attempting another approach to treating the illness: Introducing a second anomaly in a various place to override the production of sickle hemoglobin. It imitates a naturally occurring phenomenon in which an individual has 2 sets of sickle hemoglobin genes, but does not reveal signs of the illness. The reason? A various anomaly in the fetal hemoglobin gene, which typically turns off in favor of adult hemoglobin as individuals age, but stays turned on and producing normal hemoglobin. “They won the genetic lotto after losing it twice,” says Liu. Both programs showed appealing results in mice.

Liu, 47, prefers to concentrate on his scholastic and research study pursuits, rather than commercialization of the technologies, as do his cofounders Zhang and Joung. The trio like to hire well-credentialed executives to run their companies, like Evans, who has a performance history of effectively bringing precision medications from the laboratory to the market.

John Evans, CEO, Beam Rehabs (L) and cofounders David Liu, J. Keith Joung, and Feng Zhang.

DAN BUDWICK

In 2009, Evans, who made an MBA from Wharton and a Masters in biotechnology from the University of Pennsylvania, was an early staff member at Agios Pharmaceuticals, then a small biotech with a pre-clinical target related to cancer metabolism. The following year he helped broker a distinct alliance between Cambridge-based Agios and huge biopharma Celgene, ultimately resulting in 2 FDA approvals for treatments for acute myeloid leukemia in the period of 10 years, which seems like a long period of time, but is much faster than the usual drug development timeline.

” Celgene, gave us $130 million dollars upfront to explore this location of biology and Agios was able to preserve commercial right, and lots of other important functions, so it was an extremely innovative deal,” says David Schenkein, the former CEO of Agios and a basic partner at Mountain View, California-based venture firm GV (formerly known as Google Ventures), which invested in Beam.

After eight years at Agios, Evans left to end up being an endeavor partner at Arch Endeavor Partners, an early financier in Beam. In late 2016, he fulfilled Liu in his workplace in Cambridge to talk about the company, which was in stealth mode at the time. “I could not sleep that night, I was so thrilled about it,” Evans recalls.

Considering that base modifying is a platform, rather than a single drug, once the technology works in one illness, it will likely work in others. “That ease of retargeting is going to imply that as we get it up and running, we can very quickly go through and deal with a whole bunch of diseases and create a kind of sustainable circulation of brand-new medications,” states Evans.

Beam can’t assault every disease so Evans has pursued tactical collaborations and licensing agreements with other gene modifying business, consisting of Editas, Prime and another Cambridge-based biotech called Vigor, which is utilizing base editing to establish heart disease therapies. “It’s kind of a divide and conquer technique, where we’re minimizing redundancy and now I think more illness, more patients are possibly going to benefit from the innovation than otherwise,” Evans states.

Of course, it’s difficult to predict whether Beam’s technological edge with base editing will eventually prevail over earlier Crispr technologies that have the first-mover advantage, says David Nierengarten, a biotechnology expert at Wedbush Securities. What sets Beam apart so far based on their work in mice is “more effective” gene editing, meaning that “higher numbers of customized cells” will get into patients, says Nierengarten.

Small Targets

Beam is researching a variety of other unusual illness it hopes to deal with, or even treat, with its accuracy gene modifying technology. Here are the seven it has disclosed.

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Sickle Cell Illness

Estimated U.S. Clients: 100,000

Acquired blood condition causes severe pain.

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Beta Thalassemia

Estimated U.S. Clients: 1,000 -2,000

Inherited blood disorder triggers severe anemia.

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T-Cell Acute Lymphoblastic Leukemia

Estimated U.S. Clients: 500 -1,000 per year

Fast-growing blood cancer.

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Intense Myeloid Leukemia

Approximated U.S. Clients: 20,000 per year

Fast-growing blood cancer.

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Alpha-1 Antitrypsin Deficiency

Approximated U.S. Clients: 60,000

Acquired disorder causes lung and liver disease.

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Glycogen Storage Disoder 1a

Estimated U.S. Clients: 1,400

Inherited disorder where body can’t store sugar.

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Stargardt Disease

Approximated U.S. Clients: 5,500

Inherited eye condition causes progressive vision loss.

Beam has one key benefit: with its innovation, the DNA double helix doesn’t need to be cut, like with very first generation Crispr innovations. This means higher accuracy with less threat of random insertions and removals of code.

Beam’s real test will follow year, when the business prepares to request permission from the U.S. Fda to start clinical trials in people. Even in these early stages, Dr. Francis Collins, director of the National Institutes of Health, the $417 billion (2020 spending plan) federal research study company, states he’s “truly excited” about the capacity of base modifying to handle the 7,000 unusual illness caused by DNA misspellings.

” By delivering this kind of base editor to the best tissue at the right time, you can envision a lot of these [rare diseases] ending up being treatable, maybe even treatable,” says Collins, who is working with Liu on an NIH-funded research study project utilizing base editing (currently in mice) to remedy the point mutation for progeria, a disease that causes kids to quickly age and die by the time they are teens. “I definitely, in my wildest dreams, never envisioned this type of exact capability to edit the genome, where you could go and find one letter out of 3 billion that needed to be repaired, and supply the apparatus to do that with fairly little danger of triggering problem somewhere else,” Collins says reflecting on his profession and the fast progress in gene modifying over the previous couple of years.

But one of the huge technical challenges ahead for lots of diseases will be improving the delivery techniques of successfully getting base editors into the client’s body. While the sickle cell therapy can be done outside the body and placed, for other illness, like progeria, the base editor will have to be straight inserted into the patient, and there are numerous methods being established.

The other obstacle on the horizon will be price. Existing gene therapies tend to be insanely pricey. Novartis, for example, made headlines in 2019 for charging $2.1 million for Zolgensma, a breakthrough cure for spine muscular atrophy. The majority of adult sickle cell patients don’t even have access to hydroxyurea, which is the most affordable generic treatment on the marketplace. “We just have a long way to go before we can reasonably state that a significant variety of patients may gain from these therapies,” states Dr. Liem, who chaired the American Society of Hematology scientific practice guidelines on sickle cell illness. While gene treatments are amazing, “the large bulk of clients out there still need fundamental care,” states Liem.

However Evans hopes Beam can essentially change the guidelines of engagement.

from Job Search Tips https://jobsearchtips.net/this-start-up-might-lastly-cure-sickle-cell-disease-after-a-century-of-racist-overlook/

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bspolink1348 · 5 years

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Les nouveautés de la semaine en BSPO (16/09/2019)

À la une : Contrôle de gestion : perspectives stratégiques et managériales / dirigé par Nicolas Berland, Yves De Rongé

Cote de rangement : JN 8395 A 252842 / Domaine : Gestion

« Cet ouvrage apprend à articuler le contrôle de gestion et la stratégie de l'entreprise pour en faire un outil de management. Les principes fondamentaux du contrôle de gestion sont clairement identifiés et explicités, tout en mettant l’accent sur le pilotage de l’activité.

L’ouvrage étudie également les pratiques plus récentes : la méthode UVA®, l’EVA®, le contrôle de gestion dans le secteur non marchand et à l’international, la mesure des risques psychosociaux.

Ancrés dans le monde réel, tous les chapitres comprennent des exemples issus d’entreprises existantes, des illustrations chiffrées et une partie activités, avec des questions de révision et des études de cas.

Ce manuel s’adresse ainsi tant aux futurs contrôleurs de gestion qu’aux futurs managers, qui se serviront des outils du contrôle de gestion pour piloter leur activité.

Yves De Rongé est professeur à Louvain School of Management à l'université catholique de Louvain où il enseigne la comptabilité de gestion, l’analyse des états financiers et le contrôle de gestion. Il a assuré la présidence de l’école de 2005 à 2007. Il est également professeur à l’université Saint-Louis à Bruxelles et assure des formations en entreprise.

Nicolas Berland est professeur à l'Université Paris-Dauphine et responsable du Master Contrôle de gestion. Spécialisé en contrôle de gestion et dans le pilotage des organisations, il travaille plus particulièrement sur les processus d’implantation dans des contextes stratégiques variés : entreprises privées et organisations publiques. Il intervient en formation continue en MBA en France et à l’étranger. » - Quatrième de couverture

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Sciences politiques

L'entreprise Macron / sous la direction de Bernard Dolez, Julien Fretel et Rémi Lefebvre

Cote de rangement : DC 435 E 258308

Your country, our war : the press and diplomacy in Afghanistan / Katherine A. Brown

Cote de rangement : DS 371 .4135 B 258316

Les nazis ont-ils survécu ? Enquête sur les Internationales fascistes et les croisés de la race blanche / Nicolas Lebourg

Cote de rangement : JC 481 L 258313

Europe, alternatives démocratiques : analyses et propositions de gauche / sous la direction de Benjamin Bürbaumer, Alexis Cukier et Marlène Rosato

Cote de rangement : JN 30 E 258321

Big data électoral : dis-moi qui tu es, je te dirai pour qui voter / Anaïs Théviot

Cote de rangement : JN 2959 T 258310

Repenser les stratégies nucléaires : continuités et ruptures : un hommage à Lucien Poirier / Thomas Meszaros (dir.)

Cote de rangement : U 263 R 258323

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Sciences du travail

Not working : where have all the good jobs gone ? / David G. Blanchflower

Cote de rangement : HD 5709 B 258322

Le capitalisme patriarcal / Silvia Federici

Cote de rangement : HD 6060 .6 F 258309

Social work and social theory : making connections / Paul Michael Garrett

Cote de rangement : HV 245 G 258317

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Communication

Racism and the media / Gavan Titley

Cote de rangement : P 94 .5.M55 T 258320

Cybersecurity : politics, governance and conflict in cyberspace / Damien Van Puyvelde, Aaron F. Brantly

Cote de rangement : TK 5105 .59 V 258318

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Sciences sociales

https://bib.uclouvain.be/opac/ucl/fr/chamo/chamo%3A2034414?i=0

From anthropology to social theory : rethinking the social sciences / Árpád Szakolczai, Bjørn Thomassen

Cote de rangement : H 61 .15 S 258319

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Sociologie

Le jeu du monde / Kostas Axelos

Cote de rangement : B 29 A 258302

Palaces of pleasure : from music halls to the seaside to football, how the Victorians invented mass entertainment / Lee Jackson

Cote de rangement : GV 75 J 258315

Serge Moscovici : un regard sur les mondes communs / sous la direction de Nikos Kalampalikis, Denise Jodelet, Michel Wieviorka, Denis Moscovici et Pierre Moscovici

Cote de rangement : HM 1031 M 258312

Histoire rurale de l'Europe : XVIe-XXe siècle / sous la direction de Laurent Herment

Cote de rangement : HD 1917 H 258311

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Économie

Les grandes questions économiques et sociales / sous la direction de Pascal Combemale

Cote de rangement : HD 75 G 258314

100% monnaie / Irving Fisher

Cote de rangement : HG 221 F 258307

Le casino climatique : risques, incertitudes et solutions économiques face à un monde en réchauffement / William Nordhaus

Cote de rangement : QC 903 N 258304

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Comptabilité

Les normes comptables internationales (IFRS) / Bernard Raffournier

Cote de rangement : HF 5626 R 258305

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Psychologie

Penser en algorithmes / Brian Christian et Tom Griffiths

Cote de rangement : BF 39 C 258306

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Tous ces ouvrages sont exposés sur le présentoir des nouveautés de la BSPO. Ceux-ci pourront être empruntés à domicile à partir du 30 septembre 2019.

#Gestion #Sciences Politiques #Sciences du Travail #Communication #Sciences Sociales #Sociologie #Economie #Comptabilité #Psychologie

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mbacrewbr · 4 years

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Olá, nós somos a MBA Crew Brasil!

Seremos a sua melhor fonte de informações e atualizações de cada membro.

Vocês podem nos seguir também no Twitter e Instagram.

Hello, we're MBA Crew Brazil!

We'll be your best source of information and updates for each member.

You can also follow us on Twitter and Instagram.

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tuyetthienduong · 5 years

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nnoctis · 5 years

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wait you know MBA right? saw you in somebodys insta story sksksksks

only gv actually

#like hes the only one i hang with ?????????????#asks #ive met the rest but idk them lmao #best part about being friends with him is the ridiculous fucking videos of EK hell show you lmao

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theworldilivedintoday · 4 years

Video

AOMG SIGN HERE EPISODE 6 AOMG SIGN HERE EPISODE 6 SIGN HERE EPISODE 6 Sign Here Episode 6 is finally here, and congratulations to the talented girl, …

#AOMG #BOLA #COVA) (Prod. Neal)#먼치맨 #GRAY #GV #편지 (feat. 사이먼 도미닉) (Prod. dress)#안경잽이 #k hip hop #Korean Hip Hop #Make A Movie #MBA – POP (Feat. 박재범 #Sign Here #Sign Here Final #Spread The Love (Feat. 우원재) (Prod. GRAY)

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robotcenter-blog · 4 years

Video

youtube

Robocare Interview

Facebook: https://www.facebook.com/robocarelab/

Robot Phil: http://www.robophil.com

Philip English- Right. Hi guys. Innorobo 2016. We're here with a gentleman that's gonna introduce his company, which is Robocare, and he's gonna tell us a little bit about his products, one being a telepresence product, and another one being Milo. So can you introduce yourself and tell me a little bit more about your company?

Faisal- Hi, my name is Faisal, and Robocare Lab is a French-based company. We have plan, by the way, to extend in UK, and we develop a full range of services to improve the lives of people. So our range of services is named PRESENCE+ and it includes services to help autistic kids to better communicate and for that we use Milo. Milo is manufactured in the US and it includes over 120 lessons to develop the capabilities of kids with autism to better communicate.

Philip English- All right, okay. So this is Milo to the right of you. So the lessons are all to do with autism, really. To help the kids speak better, or visualize better, I suppose. How long and how old is the company?

Faisal- The company has been created one year ago, but we have been working off the rider for roughly over two years now.

Philip English- Right, two years development. So, you've got Milo, so that's one of your products for the autism side. So the other product you've got is to do with telepresence. So can you tell us a little about this one?

Faisal- Yeah, mobile telepresence. It's a great services that we are deploying in different segments. One of them, of course, is the retirement homes. We think that a lot of families today are living away from their grandparents. In France, or even in UK, you have millions of citizens who are living abroad, and I think it's very important for everybody to visit their grandparents. To support the only member of the family who is living next to the grandparents, who is doing the whole job. And we plan through PRESENCE+ Visitor, to offer the services we have already deployed in few retirement homes already in France, where the families can go onto our gateway, PRESENCE+ Visitor, book a slot, and then it is validated by the retirement homes, and then the family can visit the families using SAM. We name it SAM, for System Audio Mobile, and they can move around, just like with a feeling to be there. Yeah, you know when you call your Grandma over Skype, it will last three minutes. With SAM, I think you can stand over 30 minutes and it's like a real visit. You can really come with your Grandma to the restaurant or the retirement homes and visit her.

Philip English- Right, okay. And that's the senior level, but I remember you speaking earlier that you've also got some new sort of levels, like a campus level, is that the next one?

Faisal- Yeah, we are very pleased to announce that we are launching next week PRESENCE+ Campus. It will be announced actually next week in the press, working with one of the top 20 worldwide MBA business schools, where PRESENCE+ Campus will allow some students who are living abroad, or teachers to be present in the campus through SAM.

Philip English- Right, brilliant, brilliant. So it sounds like you guys are sort of like the integrators. You take a product, whether it's telepresence or Milo, and then you integrate it so it works for the end user, for the actual customer, so yeah.

Faisal- Yes, exactly. Our main focus is to take products who are very reliable. If you look at SAM, it is named Beam in the US, and Beam is already present in thousands of units in the corporate world. So Milo is already present in 200 hospitals or schools in US, so we take the progress of robotics with something very reliable and we bring new services where we can supply a region key solutions with the hardware, with the software, with the whole integration solution, to make it easy. To make it easy for the people.

Philip English- For the end user. So, what's next then? So you've got the two products here. Is there something else that you guys are starting to look into, or?

Faisal- Yeah, I mean PRESENCE+ has a lot of other opportunities. I mentioned PRESENCE+ Visitor for the retirement homes. We are launching PRESENCE+ Senior for the old people living alone at home, to facilitate the communication with the family, and to check that everything is going fine. We are planning, so PRESENCE+ launch in, I will say, a nice hotels or bar, to facilitate communication with colleagues and friends, and we have also other plans in our mind that we will be launching in the next month.

Philip English- All right, brilliant, brilliant. Okay, no that's brilliant. I mean that's a brilliant overview of your company Robocare, and I think it's given us a good insight to where you guys are going, and you said you're coming to the UK soon. Is that right, or?

Faisal- Yeah, I mean London actually. I am very pleased to be invited at the London Founder Forums, that will take place mid-June, by the way. And I'm also invited by the National Autistic Society where we'll be presenting Milo to 10 Head of Schools in charge of autistic kids, and we have also planned to deploy our PRESENCE+ services opening as soon as possible at GV in London. Hopefully we'll get some investors by the way, in UK, to starting deploying in the UK market.

Philip English- Okay, that's brilliant, that's brilliant. Right, what I'll do guys, is I'll put loads of links so you can find out where to find these guys, and yeah I thank you very, very much for the interview.

Faisal- Thank you, very much. Much appreciated.

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robotphilip · 4 years

Video

youtube

Robocare Interview

Facebook: https://www.facebook.com/robocarelab/

Robot Phil: http://www.robophil.com

Faisal- The company has been created one year ago, but we have been working off the rider for roughly over two years now.

Philip English- Right, okay. And that's the senior level, but I remember you speaking earlier that you've also got some new sort of levels, like a campus level, is that the next one?

Philip English- For the end user. So, what's next then? So you've got the two products here. Is there something else that you guys are starting to look into, or?

Faisal- Thank you, very much. Much appreciated.

0 notes