Category Archives: Genetic Therapy

CAMBRIDGE, Mass. bluebird bio, Inc. (NASDAQ: BLUE) today announced that, due to the public health and safety concerns related to the novel coronavirus (COVID-19) pandemic, to support the health and well-being of its directors, employees and stockholders, the location of the Companys 2020 Annual Meeting of Stockholders (including any adjournments or postponements, the Annual Meeting) has been changed to a virtual format only.

As previously announced, the Annual Meeting will be held at 8:30 a.m., Eastern Time, on Thursday, June 18, 2020. Stockholders will not be able to attend the Annual Meeting in person, but instead will be able to attend virtually. You will be able to participate in the annual meeting, vote your shares electronically and submit your questions during the live audio webcast of the meeting by visiting http://www.virtualshareholdermeeting.com/BLUE2020 and entering your 16-digit control number found on the voting form that was enclosed with the proxy materials for the Annual Meeting that were previously distributed. If a stockholder encounters any difficulties accessing the virtual meeting during the check-in or meeting time, please call the technical support number provided.

As described in the proxy materials for the Annual Meeting that were previously distributed, stockholders are entitled to participate in the Annual Meeting if they were a stockholder of the Company as of the close of business on April 23, 2020, the record date.

Whether or not a stockholder plans to participate in the live webcast of the Annual Meeting, we urge all stockholders to vote and submit their proxy in advance of the Annual Meeting by one of the methods described in the proxy materials for the Annual Meeting that were previously provided to the stockholders. The proxy card included with the proxy materials previously distributed will not be updated to reflect the change in location and may continue to be used to vote stockholder shares in connection with the Annual Meeting.

A notice regarding this change to a virtual meeting format is being filed as additional proxy materials with the Securities and Exchange Commission together with this press release.

About bluebird bio, Inc.

bluebird bio, Inc. (Nasdaq: BLUE) is pioneering gene therapy with purpose. From our Cambridge, MA headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, WA; Durham, NC; and Zug, Switzerland.

bluebird bio is a trademark of bluebird bio, Inc.

Learn more at http://www.bluebirdbio.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200603005781/en/

Contacts

Investors & Media Investors: Ingrid Goldberg, 410-960-5022 igoldberg@bluebirdbio.com OR Elizabeth Pingpank, 617-914-8736 epingpank@bluebirdbio.com

Media: Jenn Snyder, 617-448-0281 jsnyder@bluebirdbio.com

#distro

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bluebird bio Announces That Its 2020 Annual Meeting of Stockholders Will Be Held in Virtual Format - Financial Post

Mark Williams The Columbus Dispatch

TuesdayJun2,2020at5:11PM

A Massachusetts-based biopharmaceutical company plans to create 100 jobs as part of an expansion of its Columbus operations.

Sarepta Therapeutics will open an 85,000-square-foot building at 3435 Stelzer Rd. as part of its Gene Therapy Center of Excellence.

The company says it will invest more than $30 million, and that hiring for research, technician and general operations positions will begin immediately.

Employees currently working out of the companys offices in Dublin will move to the new building over time.

Sarepta, based in Cambridge, focuses on gene therapy programs to treat rare diseases.

It has two approved drugs for Duchenne muscular dystrophy and more than 40 treatments in development.

Duchenne slowly steals muscle, making children weaker and weaker as they grow older. Many died by their mid-20s. The disease afflicts mostly boys.

We are confident that gene therapy will revolutionize genetic medicine, and we chose Ohio for our Gene Therapy Center of Excellence because we believe Columbus will become a hub for genetic medicine innovation, the companys president and CEO, Doug Ingram, said in a statement.

mawilliams@dispatch.com

@BizMarkWilliams

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Sarepta to expand Columbus operations, add 100 jobs - The Columbus Dispatch

The IPO engine is thriving, never mind the rampage of the coronavirus crisis on R&D timelines.

On Friday, along with synthetic lethality-focused biotech Repare Therapeutics, another Bristol Myers partner Forma Therapeutics also unveiled its plans to vault on to the Nasdaq penciling in a target of $150 million.

The Watertown, Massachusetts-based company which poached senior Genentech executive Frank Lee to take over the reins last year after more than a decade under founder Steve Tregay raised a plump $100 million late last year, while shepherding its sickle cell disease (SCD) drug through an early-stage trial.

Last November, the FDA ushered in the approval of two SCD therapies, injecting some optimism into an otherwise barren treatment landscape for patients with the blood disorder that is characterized by atypical hemoglobin molecules, which can distort red blood cells into a sickle, or crescent, shape. Novartis therapy, Adakveo, is designed to prevent periodic episodes of searing pain called vaso-occlusive crises (VOCs) that deprive the deprive the body of oxygen-rich blood, while Global Blood Therapeutics voxelotor is designed to work by increasing hemoglobins affinity for oxygen.

Instead of addressing symptoms of SCD, Formas lead therapy, FT-4202, is designed to change the course of the disease as an activator of the enzyme pyruvate kinase-R (PKR) to improve red blood cell metabolism, function and survival, potentially resulting in both increased hemoglobin levels and fewer VOCs. Forma is in a crowded field of therapies in development, with other drugmakers including bluebird bio, Imara, and partners CRISPR Therapeutics and Vertex, also working on their own drugs.

Meanwhile, Forma also plans to evaluate the use of FT-4202 in beta thalassemia. The company also has a brimming pipeline, including an AML drug and a NASH therapy in mid-stage development, as well as earlier-stage compounds for NASH, NHL (partnered with Bristol Myers Squibb) and solid tumors (in collaboration with Boehringer Ingelheim).

There has been a flurry of biopharma IPOs in recently Generation Bio, Avidity and Vaxcyte set their sights on a combined $325 million and the week before ADC Therapeutics raked in $233 million in an upsized offering despite the general pandemonium on Wall Street due to Covid-19. But the life sciences sector has emerged largely immune from the rout.

Swedens Calliditas, which last week said it was eyeing a $75 million raise (a modest sum compared to some of the splashier public debuts seen in recent weeks), on Monday indicated it was commencing an investor roadshow. The company, which is developing a therapy for an orphan kidney disease, already has a Swedish listing.

Excerpt from:
IPOs abound in the time of coronavirus, as Forma Therapeutics pencils in $150M Nasdaq debut - Endpoints News

THE ultimate warrior would be unable feel fear or pain, capable of running at Olympic speeds, and even immune to modern weapons.

Their existence was once only possible in the realm of science fiction but a new worldwide arms race is pitting nation states against each other to be the first to successfully create real genetically modified super soldiers.

Militaries have a long history of using powerful drugs to temporarily turn their troops into transcendant Terminator-style killers.

Nazis took methamphetamine or "crystal meth" during the Second World War to stay alert and awake for superhuman stretches of time.

And even the British military bought thousands of Modafinil pills which boost brain-power ahead of the Iraq War.

In China, it is reasonable to assume that they are enhancing their battlefield soldiers on all these fronts.

But with advances in technology, it could now be possible to alter soldiers' DNA to give them godlike powers all the time, from Herculean strength to lizard-like limb regeneration.

GM technology is proven with plants, it could absolutely be applied to the person, said Professor John Louth, an expert at defence think tank Rusi.

In China, it is reasonable to assume that they are enhancing their battlefield soldiers on all these fronts.

China's armed forces are the largest in the world, consisting of a staggering 2.2million personnel.

This year alone, Beijing is spending $178.16billion on its defence budget.

But as the country's international relations flare up, they could be looking to be the first army to have genetically modified super soldiers to get ahead of adversaries.

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These combatants would be stronger, faster and even smarter than their battlefield opponents.

Their DNA could also be adapted to help them recover more quickly from injuries or give them superior hearing and night vision.

The threat is obvious and real. Chinese money could be stealing a march on western armed forces and that is deeply concerning," Prof Louth said.

Concerns about China's super soldier plans came after a Chinese scientist, He Jiankui, claimed to have successfully created genetically modified babies using gene editing technology.

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China jailed Dr Jiankui for illegal medical practices over his claims to have made three babies immune to HIV.

But it's now suspected that Chinese military chiefs are backing trials into human gene editing.

But they're not alone.

The US has also conducted some strange super-soldier research projects.

They've already publicly unveiled a 5million Iron Man-style exoskeleton which gives fighters incredible muscle-power.

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And the Defense Advanced Research Projects Agency (DARPA) developed ways for warfighters to scale walls by studying the skin of geckos.

Novelist Simon Conway, who was granted behind-the-scenes access at the secretive Pentagon agency, revealed a string of other super soldier programmes underway there in 2012.

He claims scientists were working on gene modification that would allow soldiers' bodies to convert fat into energy more efficiently, allowing them to go days without eating.

What is gene editing?

"It's all about improving the efficiency of energy creation in the body," Conway told the Sunday Express.

"Soldiers would be able to run at Olympic speeds, carry large weights and go without sleep and without food."

But the US isn't just looking at how biotechnology can give their soldiers the upper-hand on the battlefield.

They're also carrying out research into medical regeneration, allowing severely injured soldiers who've lost limbs or suffered extensive burns to heal organically.

This is already a reality in the animal kingdom, where lizards can regrow amputated tails and salamanders can restore entire severed limbs.

"We would like it to be as restorative as possible, resist infection and be durable," said Army Lt. Col. David Saunders, extremity repair product manager for the U.S. Army Medical Materiel Development Activity.

"[There are] many wonderful things emerging in the field of regenerative medicine to restore form and function to our wounded warfighters."

As recently as January 2020, the US military was unveiling incredible advances in warzone genetics.

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Army researchers developed a gene therapy that allowed mice to create proteins that would protect them against nerve agents deadly chemical weapons that attack the nervous system, like the Russian Novichok used in the Salisbury poisonings in 2018.

The same gene therapy given to the mice that made them chemical weapon-proof could theoretically be used in soldiers entering hazardous environments.

Before Russia's nerve agents were used with terrible effect, president Vladimir Putin had warned of an even more terrifying weapon.

Speaking at a 2017 youth festival in Sochi, Putin spoke openly about the destructive possible consequences of gene-editing.

"A man has the opportunity to get into the genetic code created by either nature, or as religious people would say, by the God," he said, The Express reports.

"He can be a genius mathematician, a brilliant musician or a soldier, a man who can fight without fear, compassion, regret or pain.

"As you understand, humanity can enter, and most likely it will in the near future, a very difficult and very responsible period of its existence.

"What I have just described might be worse than a nuclear bomb."

But instead of this being something in the "near future", Russia is already factoring genetics into its military strategy.

Alexander Sergeyev, the head of the country's Academy of Sciences, revealed the armed forces were researching "genetic passports" in 2019, Forbes reports.

The passports would predict a soldier's "resistance to stress, ability to perform physical and mental operations under the conditions of this stress, and so on."

Sergeyev added that they could be used to sort which branch of the armed forces personnel would be sent to.

"There are already serious developments in this area," he said.

"It is about understanding at the genetic level who is more prone to, for example, to service in the fleet, who may be more prepared to become a paratrooper or a tankman."

What I have just described might be worse than a nuclear bomb.

And unlike other world leaders, Putin has a very close interest in genetic editing.

That's because his eldest daughter, Maria Vorontsova, is a scientist who specialises in genetic engineering and acts as his adviser on the matter.

In 2018, before He Jiankui revealed his HIV-immune babies in China, Putin had already allotted $2billion for genetic research, Bloomberg reports.

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He even put Vorontsova in charge of the 30-person panel overseeing the experiments.

Watching world superpowers will undoubtedly be paying attention to Putin's potential to weaponise the research.

After all, as Putin says, genetic editing is an area of science which will "determine the future of the whole world".

Read more:
Inside the super-soldier arms race to create genetically modified killing machines unable to feel pain or fe - The Sun

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New York/Minneapolis: The years biggest meeting of cancer researchers was subjected to a coronavirus overhaul this year, but even in scaled-back form it forced investors to recalibrate their expectations for some closely watched medicines.

The American Society of Clinical Oncology meeting is the fields most important gathering each spring, providing a stage for major pharmaceutical companies to unveil major findings and tout promising treatments. Its also an annual opportunity for all kinds of researchers, doctors, executives and investors to rub elbows.

With Covid-19 making travel uncomfortable and splashy conferences impossible this year, the summit was mostly a virtual affair. Still, it delivered many of the kinds of important victories and stinging setbacks it often does. And the meeting showed that even as the drug industry races to identify virus treatments and vaccines, cancer remains perhaps its most important business overall.

When the coronavirus wanes and we have a vaccine, and this infectious disease is brought under control, we will still have cancer and the need for new treatments, said Richard Schilsky, ASCOs chief medical officer, in an interview. We have millions of patients around the world who need new and improved treatments for cancer.

Among the most noteworthy winners was U.K. pharmaceutical giant AstraZeneca Plc, which reported that its blockbuster Tagrissoreduced the riskof dying from lung cancer or relapse by four-fifths over three years. The drug is already AstraZenecas biggest product, bringing in $982 million in sales in the first quarter alone.

Some smaller drug companies also logged what looked like significant victories: Trillium Therapeutics Inc. said that a second patient taking its experimental lymphoma therapy responded to the treatment. Shares of Trillium, which has a market value of about $560 million, surged more than 17% on Friday

And as ASCO played out, other big cancer-research news also roiled drugmaker stocks.

Pfizer Inc. shares fell 6% late Friday after the U.S. drug bellwether said a late-stage study of its treatment for metastatic breast cancer, Ibrance, was unlikely to show a statistically significant improvement in invasive disease-free survival for patients with early breast cancer.

Also read: New blood test proves successful in detecting multiple cancers early on

Though the Covid-19 pandemic has been the defining story of the health-care business this year, the ASCO meeting was a reminder that most of the worlds pharmaceutical companies remain keenly focused on finding new cancer treatments to generate profits. And that focus has paid off for patients: The U.S. death rate from cancer has beenfalling at a record pace, thanks largely to big advances in treating lung tumors.

Data presented at the meeting showed progress in combating the second leading cause of death worldwide, Schilsky said. Researchers found medicines used for patients with advanced disease can have an even bigger benefit for those with recently diagnosed tumors, while medicines are emerging that are effective against a wide range of tumors that are driven by specific gene mutations.

Large drugmakers have remained on the prowl for promising cancer therapies that they can acquire through mergers or other transactions. Even Gilead Sciences Inc., which has been in the headlines because of its potential coronavirus treatment remdesivir, has been getting cancer-focused deals done against the backdrop of the pandemic.

Last week, Gilead agreed to work on immunotherapy drugs with biotech Arcus Biosciences Inc. And earlier this year it agreed to buy cancer-drug maker Forty Seven for $4.9 billion. Gilead has been pressured by investors to find new drugs that can take the place of some of its aging blockbusters. It madeanother big bet on cancer when it bought Kite Pharma and its drug Yescarta, though sales of the highly priced genetic therapy have so far failed to liveup toexpectations.

At ASCO, Gilead presented encouraging new data on magrolimab, an immunology drug that was developed by Forty Seven, a sign that the companys wager could pay off. Gileads management is successfully building a pipeline of potential new therapies, Jefferies & Co. analyst Michael Yee said in a note sent to clients.

Gilead shares climbed 3.3% to $77.83 on Friday and now have gained 20% so far this year, in part because of excitement over remdesivir.

Breakthroughs at ASCO could make smaller companies enticing to Gilead and to other giants looking to add to their rosters of experimental therapies. Adaptimmune Therapeutics Plcs U.S.-traded shares more than doubled in value after the company presented early studies for therapies that could treat a number of cancers, including lung and head and neck tumors.

Allogene Therapeutics Inc., which is developing a CAR-T therapy similar to Yescarta, gained 3.5% Friday after it said its treatment benefited 63% of patients with blood cancer in an early-stage trial. Allogenes founders ran Kite Pharma before selling it to Gilead for $11 billion in 2017.

Also read: Cancer, dialysis patients struggle for treatment as hospitals are stretched by Covid-19

Like Gilead, Bristol-Myers Squibb Co. has also sought to refashion itself into a cancer-fighting juggernaut, mostly notably with its $74 billion takeover of Celgene Corp. last year. Even before that transaction, the company was locked in a battle with rival Merck & Co. for supremacy in the market for lung-cancer immunotherapies.

Mercks Keytruda and Bristol-Myerss Opdivo are blockbusters for each company, and the drugmakers have raced to expand their use in a range of tumor types. But at ASCO, a study of Opdivo in combination with another Bristol-Myers drug, Yervoy, supported what JPMorgan Chase & Co. analyst Chris Schott said in a note was a niche role in treating certain lung-cancer patients not one that will upset Keytrudas dominance.

Bristol-Myers shares declined 0.2% on Friday, while Merck advanced 2.1%.

Last year, Opdivo generated $7.2 billion in revenue for Bristol-Myers, while Yervoy brought in $1.5 billion. Together, the two drugs accounted for roughly a third of the companys sales. Meanwhile, Keytruda generated $11 billion for Merck, about 24% of its sales. Bloomberg

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Lets not forget cancer treatment message from oncology summit held in the shadow of Covid - ThePrint

TMR Research states that global gene therapy for inherited genetic disorders market will see a witness major developments in the coming years.Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer.

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Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.

Some of the most prominent competitors operating in the competitive landscape of global gene therapy for inherited genetic disorders market include

Global Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease).

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

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Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practicaland accessiblegene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations.

Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Gene Therapy for Inherited Genetic Disorders Market Evaluation of Industry Trends, Growth Drivers and Forecast To 2025 - Medic Insider