Archive for the ‘Gene Therapy Research’ Category

Latest Report on the CNS Gene Therapy Market

Persistence Market Research (PMR) recently published a market study that offers critical insights related to the growth prospects of the CNS Gene Therapy Market during the forecast period 2018 2028. The report takes into account the historical and current market trends to evaluate the top factors that are likely to influence the growth of the market in the upcoming years.

As per the report, the CNS Gene Therapy Market is poised to grow at a CAGR of ~XX% during the assessment period primarily driven by a growing focus on product innovation, a surge in demand for the CNS Gene Therapy in the developed regions, and potential opportunities in the developing regions.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/27514

What Sets Us Apart from the Rest?

The presented market study bifurcates the CNS Gene Therapy Market on the basis of geography, applications, and end-use industries.

The competitive outlook section touches upon the business prospects of some of the most established market players in the CNS Gene Therapy Market. The company profiles of each company are included in the report along with data including revenue growth, production capacity, domestic and regional presence, product portfolio, and more.

Essential findings of the report:

Get Access To TOC Covering 200+ Topics athttps://www.persistencemarketresearch.co/toc/27514

key players and product offerings

In order to get a strategic overview of the market,Access Research Methodology Prepared By Experts athttps://www.persistencemarketresearch.co/methodology/27514

The report aims to address the following queries related to the CNS Gene Therapy Market:

About us:

Persistence Market Research (PMR) is a third-platform research firm. Our research model is a unique collaboration of data analytics and market research methodology to help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

Contact us:

Persistence Market Research305 Broadway, 7th FloorNew York City, NY 10007United StatesPh.no. +1-646-568-7751

This post was originally published on Market Reports Observer

See the original post here:
CNS Gene Therapy Market to Witness a Pronounce Growth During 2018 2028 - Market Reports Observer

Last we heard about Forma Therapeutics funding was in March, when they were laying off employees as longtime partner and benefactor Celgene then in the process of being bought out by Bristol-Myers Squibb severed ties. Cut off from what turned out to be a $757 million IV, they axed 61 staff members, hired a new CEO and began reorienting the company.

The shift has evidently managed to convince some investors. Forma announced today a $100 million Series D that will fuel the companys push to become a clinically focused biotech. The syndicate includes investors long known for taking companies toward an IPO, led by RA Capital and joined by Cormorant Asset Management, Wellington Management, Samsara BioCapital, among others.

In the last month, we have presented important proof of mechanism and differentiating clinical data for our programs in sickle cell disease, glioma, AML and NASH, new CEO Frank Lee said in a statement. We are energized by the commitment from this group of leading industry investors as we move into 2020, another critical year with multiple clinical data readouts and a new program entering the clinic.

The top program of note is a sickle cell drug called FT-4202. Unlike the recent sickle cell approvals from Novartis and Global Blood Therapeutics that attempt to relieve the diseases worst symptoms or complications, and unlike the experimental gene therapies that offer the tentative promise of a cure, FT-4202 modifies the course and underlying biology of the disease without affecting the genome. It activates an enzyme called pyruvate kinase-R that is supposed to help prevent sickling and help hemoglobin bind to oxygen.

Forma announced data from the first Phase I/II trial at ASH but only mentioned positive PK and safety results.

Much has changed at Forma since March. In addition to sickle cell, they also presented early data on glioma (a type of brain tumor), NASH and acute myeloid leukemia. Lee was brought in from Genentech to replace Steve Tregay, who had led the company since founding it in 2009. In September, they hired a new CFO and a new general counsel and promoted Patrick Kelly to CMO and Brian Lesser to therapeutic head.

Forma had for much of its life relied on several partnerships, most prominently a collaboration with Celgene that began in 2013 to develop drugs in oncology and eventually expanded to a handful of other fields. That partnership came to a sudden halt in the last few days of 2018 as the NJ-based giant prepared to announce a merger with Bristol-Myers Squibb. Documents Celgene filed as part of that buyout revealed they had given Forma $757 million over the life of the deal.

The sudden closure left the biotech facing key questions about its future. They cut staff, including 25 members of their discovery group, and began pivoting toward late-stage development. Their most advanced program is in AML, where a pivotal study is now enrolling patients.

Read the original:
Forma Therapeutics bounces back from Celgene fallout with $100 million Series D - Endpoints News

Friday, December 20, 2019 2:40 p.m. CST

By Michael Erman

NEW YORK (Reuters) - Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease.

The company said on Friday that it will be open to refining the process in the future, but it is not making any changes at this time. The program is for patients in countries where the medicine, called Zolgensma, is not yet approved for the rare genetic disorder, which can lead to death and profound physical disabilities.

At $2.1 million per patient, Zolgensma is the world's costliest single-dose treatment.

Novartis said the program will open for submission on Jan. 2 and the first allocation of drugs would begin in February. Novartis's AveXis unit, which developed the drug, will give out 50 doses of the treatment through June for babies under 2 years old, it said on Thursday, with up to 100 total doses to be distributed through 2020.

Patient advocacy group SMA Europe had a conference call with the company on Friday, according to Kacper Rucinski, a board member of the patient and research group who was on the call.

"There are a lot of ethical questions, a lot of design questions that need to be addresses. We will be trying to address them in January," Rucinski said. He said the program has no method of prioritizing who needs the treatment most, calling it a "Russian roulette."

The company said it developed the plan with the help of bioethicists with an eye toward fairness.

"This may feel like youre blindly passing it out, but it may be the best we can do," said Alan Regenberg, who is on the faculty at Johns Hopkins' Berman Institute of Bioethics and was not among the bioethicists Novartis consulted with on the decision. "It may be impossible to separate people on the basis of prognosis out of the pool of kids under 2," he said.

According to Rucinski, the parties will continue their discussion in January "to see what can be improved in the design" of the program.

Novartis said on Thursday that because of manufacturing constraints it is focused on providing treatment to countries where the medicine is approved or pending approval. It has one licensed U.S. facility, with two plants due to come on line in 2020.

Zolgensma, hit by turmoil including data manipulation allegations and suspension of a trial over safety concerns, is the second SMA treatment, after Biogen's Spinraza.

Not all of the SMA community are opposed to Novartis' program.

Rajdeep Patgiri moved from the United Kingdom to the United States in April so his daughter could receive Zolgensma. She has responded well to the treatment, and Patgiri worries that negative attention to the program could keep patients from receiving the drug.

"The best outcome for all patients would be if everybody could get the treatment. Given all the constraints, a lottery is probably the fairest way to determine who" receives the treatment, he said.

(Reporting by Michael Erman; Additional reporting by John Miller in Zurich; Editing by Leslie Adler)

Read the rest here:
Novartis in talks with patients upset about lottery-like gene therapy giveaway - KFGO News

Gene therapy is a way of delivering healthy genes or genetic material to cells in order to treat genetic disorders. The most common way to do this is using adeno-associated viruses (AAVs). The outer part of the virus, called the capsid, is generally retained, but the viral genes are replaced with the therapeutic genes. Attempts have been made to improve the capsid or shell of the virus, but usually fail. George Church and his team at Harvard Medical School with the original researchers at the Karolinska Institute and Lund University in Sweden, have developed a technique to modify the capsid. They have also launched a company, Dyno Therapeutics, to develop the approach.

The groups research, by senior author Tomas Bjrklund, with Lund, was published in PNAS, the Proceedings of the National Academy of Sciences of the United States of America.

The technique allows the researchers to engineer the virus shell to deliver the gene package to the exact cell type in the body they intend to treat. The process leverages computer simulations and modeling with gene and sequencing technology.

Thanks to this technology, we can study millions of new virus variants in cell culture and animal models simultaneously, Bjorklund said. From this, we can subsequently create a computer simulation that constructs the most suitable virus shell for the chosen applicationin this case, the dopamine-producing nerve cells for the treatment of Parkinsons disease.

The technique also dramatically decreases the need for laboratory animals. The millions of variations on the same therapy can be studied in the same individual.

The authors wrote, A challenge with the available synthetic viruses used for the treatment of genetic disorders is that they originate from wild-type viruses. These viruses benefit form infecting as many cells as possible in the body, while therapies should most often target a particular cell type, for example, dopamine neurons in the brain.

Current approaches to finding the most advantageous viruses for gene therapy use random screening, enrichment and, the authors say, serendipity. Their technique is dubbed BRAVE (barcoded rational AAV vector evolution). In BRAVE, each virus displays a peptide derived from a protein. That peptide as a known function on the AAV shell surface and what they call a unique molecular barcode in the packaged genome.

By sequencing the RNA-expressed barcodes, they can map the binding sequences from hundreds of proteins simultaneously. They liken the technique to accelerating evolution from millions of years to just weeks.

Bjorklund said The reason we can do this is that we study each generation of the virus in parallel with all the others in the same nerve cells. Unlike evolution, where only the best suited live on to the next generation, we can also learn what makes the virus work less well through this process. This is crucial when building computer models that interpret all the information.

The study showed the potential for using machine learning for AAV design, although the research fell short of actually designing an improved AAV that could be used in clinical testing. Thats where Dyno Therapeutics comes in, working to improve and develop the technique.

Luk Vandenberghe, director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear, told C&EN, Chemical & Engineering News, What theyve done here is truly a remarkable tour de force.

Original post:
Dyno Therapeutics Launches to Improve Viral Vectors for Gene Therapy - BioSpace

Though the research was intended as a proof of concept, the experimental gene therapy slowed tumour growth and prolonged survival in mice with gliomas, which constitute about 80% of malignant brain tumours in humans.

The technique takes advantage of exosomes, fluid-filled sacs that cells release as a way to communicate with other cells.

The research was carried out by scientists at the Ohio State University and published in the journal Nature Biomedical Engineering.

While exosomes are gaining ground as biologically friendly carriers of therapeutic materials because there are a lot of them and they dont prompt an immune response the trick with gene therapy is finding a way to fit those comparatively large genetic instructions inside their tiny bodies on a scale that will have a therapeutic effect.

This new method relies on patented technology that prompts donated human cells such as adult stem cells to spit out millions of exosomes that, after being collected and purified, function as nanocarriers containing a drug.

When they are injected into the bloodstream, they know exactly where in the body to find their target even if its in the brain.

Senior study author L. James Lee, professor emeritus of chemical and biomolecular engineering at Ohio State University, said: Think of them like Christmas gifts: the gift is inside a wrapped container that is postage paid and ready to go. This is a Mother Nature-induced therapeutic nanoparticle.

In 2017, Lee and colleagues made waves with news of a regenerative medicine discovery called tissue nanotransfection (TNT). The technique uses a nanotechnology-based chip to deliver biological cargo directly into skin, an action that converts adult cells into any cell type of interest for treatment within a patients own body.

By looking further into the mechanism behind TNTs success, scientists in Lees lab discovered that exosomes were the secret to delivering regenerative goods to tissue far below the skins surface.

The scientists placed about one million donated cells on a nano-engineered silicon wafer and used an electrical stimulus to inject synthetic DNA into the donor cells. As a result of this DNA force-feeding, as Lee described it, the cells need to eject unwanted material as part of DNA transcribed messenger RNA and repair holes that have been poked in their membranes.

The electrical stimulation had a bonus effect of a thousand-fold increase of therapeutic genes in a large number of exosomes released by the cells, a sign that the technology is scalable to produce enough nanoparticles for use in humans.

Essential to any gene therapy is knowing what genes need to be delivered to fix a medical problem. For this work, the researchers chose to test the results on glioma brain tumours by delivering a gene called PTEN, a cancer-suppressor gene. Mutations of PTEN that turn off that suppression role can allow cancer cells to grow unchecked.

For Lee, founder of Ohio States Center for Affordable Nanoengineering of Polymeric Biomedical Devices, producing the gene is the easy part. The synthetic DNA force-fed to donor cells is copied into a new molecule consisting of messenger RNA, which contains the instructions needed to produce a specific protein. Each exosome bubble containing messenger RNA is transformed into a nanoparticle ready for transport, with no blood-brain barrier to worry about.

The testing in mice showed the labelled exosomes were far more likely to travel to the brain tumours and slow their growth compared to substances used as controls.

Because of exosomes safe access to the brain, Lee said, this drug-delivery system has promise for future applications in neurological diseases such as Alzheimers and Parkinsons disease.

The rest is here:
Mother Nature provides new gene therapy strategy to reverse disease - Health Europa

Inside each of our cells is a genetic hourglass. Every time our cells dividewhich they have to do to keep us alivetheir 23 pairs of chromosomes remain nearly identical. Except for one intentional change: After each division, a cells chromosomes get a little bit shorter.

Ten years ago, a group of scientists won the Nobel Prize in medicine for discovering these ever-shortening DNA sequences at the end of our chromosomes, called telomeres. After a cell has divided a certain number of timesknown as the Hayflick limitits telomeres are so short that the cell knows its time to peacefully shut itself down. When enough cells die off, organs wear out, and eventually, we die, too.

This discovery ushered in decades of aspirational research that set out to understand the role of telomeresand the protein that can rebuild them, called telomerasein aging. Perhaps, if scientists could figure out how to flip our biological hourglasses over, our cells could replicate for longer. Our organs would tire more slowly, and we could delay death.

The Nobel-winning research began way back in the 1970s with the work of biologist Elizabeth Blackburn. But even after four decades, its still not clear if telomeres can safely be manipulated to thwart aging.

That hasnt stopped some scientists from betting on artificially extending telomeres to support longevity: Just last week, Kansas-based biotech startup Libella Gene Therapeutics announced that it would begin early clinical trials testing out a gene therapy that could lengthen telomeres, according to OneZero.

That approach, which as of yet has only been tested in mice, is indicative of humans deep desire to roll back the clock. But the deeper scientists go into the field, the more complicated the story behind telomeres gets: Theres evidence that they may play an important role in other aspects of our health, and that cell division may not be the only reason they shrink over time. Before scientists can try to safely harness telomeres to improve our health, theyll have to answer these questions.

One anti-aging strategy that researchers have investigated involves telomerase, the telomere-building protein that Blackburns colleague Carol Greider discovered on Christmas Day in 1984.

Telomerase is an important tool for cells that divide frequentlylike blood cells, the lining of our digestive systems, or sperm and egg cells. These cells regenerate so often that they need an enzyme to regularly rebuild the caps on the end of their chromosomes.

All the other kinds of cells in our bodies shouldnt have telomerase. But if they did, theoretically, their telomeres would never shrink. They could keep dividing beyond their normal Hayflick limit.

Theres one big problem, though: Cells that have telomerase but arent supposed to often wind up to be cancerous.

In approximately 90% to 95% of cancers, during the process of oncogenesis, telomerase is reactivated, says Masood Shammas, a lead scientist at the Dana Farber Cancer Institute in Boston. As cancer cells spread, theyre able to build their telomeres back upallowing them to keep dividing and dividing and dividing.

This means that messing with telomerase to somehow extend lifeas Libella is attempting to, by injecting patients with a virus containing the gene that codes for telomeraseis risky business.

On the other hand, it also means that blocking telomerase could be a way to treat cancer. Shammas has worked on clinical trials that have tested telomerase-targeting drugs with a company called Geron. Although their original drug worked in mouse models, it failed in early-stage clinical trials for people, because it had some nasty side effects. As a result, scientists have had to put stopping telomerase on hold until they can figure out how to make it only work in cancer cells.

An alternative strategy focuses not on rebuilding telomeres, but slowing their shrinkage in the first place. Scientists are trying to understand what, in addition to normal cell division, causes telomeres to contract. Maybe limiting these activities could decelerate aging in a way that doesnt accidentally reactivate a cancer pathway.

The activities that can slow telomere degradation are still being researched. It seems, though, that theres a lot of daily living that may play a role in telomere length. Anything that damages DNA will damage telomeres, says Shammas.

Telomeres are particularly vulnerable because theyre more exposed on the ends of the chromosomes. Smoking, drinking, and eating red meats fried in oilswhich all produce molecules that can bind to and distort DNAmay harm your telomeres, too. They also happen to all be known carcinogens.

Of course, this doesnt mean their effects are felt immediately, or that these activities will definitely lead to telomere shortening or cancer. Its their cumulative effect over a lifetime, plus other factors that scientists havent nailed down yet, that we need to watch out for. And clinicians generally advise against these activities anyway.

Perhaps more surprisingly, a life-affirming action may also cause telomeres to shrink: Pregnancy.

Dan Eisenberg, a biological anthropologist at the University of Washington, has studied how telomeres behave over time for people who become pregnant. A large cohort study he and his team published last year looked at women in the Philippines. After controlling for age, they found that the more times someone had been pregnant, the shorter their telomeres were. Each pregnancy seemed to shorten a persons telomeres by the equivalent of as many as four years of life.

This could be because of how taxing pregnancy can be on the body. Developing a fetus takes about twice the energy a person normally uses. Theres less energy available to maintain and repair cells for the long-term, Eisenberg says.

While it seems counterintuitive that evolution would penalize a person for reproducing, it may be a necessary trade-off. Perhaps the benefit of spreading new genes into the world is worth the cost of slightly shorter telomeres, Eisenberg explained. After all, evolution doesnt affect the processes that happen to us after we after our reproductive years. Weve already achieved the goal of immortality by way of our progeny.

So, lifestyle modifications to prevent telomere shortening dont sound too appealing. And so far, the only activity that researchers have found that can naturally extend telomeres in the slightest may be exercise. The only thing that world show that can activate telomerase activity is regular exercise, says Shammas. But its still not clear why this is the case, and it certainly doesnt mean that hitting the gym can stave off all aging.

Which brings us back to the promises made by companies like Libella, the gene therapy outfit currently promoting a telomere therapy. With four decades of telomere research yet to produce better guidance than cut down on red meat and exercise more, its easy to appeal to the insecurities and fears of the aging population with less-than-fully-baked treatments.

As OneZero reported, Libellas study is slated to begin early next year in Colombia. Likely, its running there to skirt the US Food and Drug Administrations (FDA) requirement for an Institutional Review Board, which ensures the safety of clinical research participants. Generally, clinical trials overseen by the FDA have been preceded by trials in at least two animal species to show theyre safe and effective. So far, the studies that have backed Libellas gene therapy are based just in mice.

This study has caused a lot of experts to raise eyebrows, particularly when it comes to the ethical issue of asking participants to pay for a therapy with high risks. The company is charging $1 million for each of its five aging but otherwise healthy participants, as well as five participants who have Alzheimers disease and five who have a form of artery disease.

But the trial also raises the question of whether aging itself is a disease worth treating. With any disease, there has to be a disease-free state, says Suresh Rattan, biogerontologist at Aarhus University. In the case of a situation like aging whose main cause is life itself, when will we say that we have treated it? Evolution didnt design us to live forever.

Original post:
Science is still studying how telomeres are linked to longevity - Quartz

RESEARCH TRIANGLE PARK AskBio, a gene therapy company based in RTP that recently raised $225 million from investors, will pay up to $240 million in upfront and milestone payments for a license to use gene therapy technology from Massachusetts-based Selecta Biosciences as part of a regime to treat Pompe disease.

The license is for ImmTOR, what Selecta describes as a immune tolerance platform. The technology is addressing barriers to repeat administration of gene therapies.

Pompe disease affects between 5,000 and 10,000 people a year, affectingventilator, cardiac and skeletal muscles and can cause motor neuron dysfunction, with effects on cognition, hearing, speech and fine motor skills, AskBio says.

There is a demonstrated unmet medical need for better treatment approaches for Pompe disease, and this collaboration will enable us to effectively advance our Pompe program with the added benefit of Selectas ImmTOR technology, said Sheila Mikhail, CEO and co-founder of AskBio, in a statement. The opportunity to re-treat patients holds significant promise, and we are pleased to be able to leverage our relationship with Selecta and apply the ImmTOR technology to potentially overcome the challenges associated with re-administering systemic AAV gene therapies.

The companies initially announced a partnership in August.

AskBio gets $235 million in gene therapy support

RTP-based AskBio expands gene therapy target list with acquisition of Scottish biotech

AskBio acquires nano drug delivery tech company RoverMed (+ video: how process works)

More:
Triangle-based AskBio to pay up to $240M for rights to use gene therapy technology - WRAL Tech Wire

Gene therapy is providing unprecedented hope for growing numbers of patients and families. This game changer in medicine restores vision in babies born with congenital blindness, reconstitutes defences against infection in inherited immunodeficiencies and offers the perspective of curing the devastating neuromuscular disease, spinal muscular atrophy.

Gene therapy is also removing the need for repeat blood transfusions in adolescents with the inherited blood disorder, beta-thalassemia. Meanwhile, in oncology, CAR-T therapies, involving genetic modifications of a patients own immune cells, are proving life-saving for children or adults with certain types of blood cancers.

All these revolutionary treatments are now approved by regulatory agencies in Europe or the US. Unfortunately, they carry astronomical price tags which prevent their effective delivery to patients. As one case in point, Bluebird Bios Zynteglo for treating beta-thalassemia, has a list price of 1.57 million.

Can high prices be justified?

Gene therapy manufacturers defend their prices by pointing to high development and manufacturing costs, small markets, and unique therapeutic effectiveness as compared to the current standard of care. However, R&D costs are kept secret, and higher numbers of patients eligible for a given therapy do not translate into lower prices.

Indeed, several arguments the manufacturers put forward are dubious or even far-fetched. As of today, claims that a single administration of a gene therapy product will ensure a lifelong cure are simply not supported by the scientific evidence.

Likewise, value-based pricing is often misconceived. As stated by the US Institute for Clinical and Economic Review in its 2017 white paper on gene therapy, the established value of a treatment reflects the maximum price society might be prepared to pay for it - but should not dictate the price that is actually paid. In an ideal world, actual prices should provide market-consistent returns for shareholders and sufficient incentive to innovate.

The EU, a pioneer in gene therapy

European scientists, institutions and charities have been central to the development of gene therapy. The world's first successful clinical trial was reported in 2000 by Alain Fischer and his team at Necker Hospital in Paris, while the first authorisation of a gene therapy product in a regulated market was granted by the European Medicines Agency in 2012.

According to the Cordis database of EU-supported research, 86 gene therapy projects for rare diseases had funding from the European Commission during the FP7 (2007-2013) and Horizon 2020 (2014-2020) research programmes. One can estimate that overall more than 1 billion has been invested in this area by the EU Commission, member states and not-for-profit organisations.

To ensure European patients benefit from these achievements and investments, it is essential to ensure reasonable pricing of gene therapies. Laudable efforts are currently being made by the World Health Organization to increase transparency, and by some member states to join forces in negotiating prices, but such initiatives are unlikely to solve the current crisis as they do not address its root, namely that the sole objective of most gene therapy companies is to maximise the return on investment and shareholder value.

A way forward: enforcing the corporate social responsibility of gene therapy manufacturers

As I recently argued with Alain Fischer and the economist Mathias Dewatripont in the journal Nature Medicine (November 25, 2019), now is the time to reflect on how to enforce the corporate social responsibility of gene therapy companies.

Among the measures we would like to see considered are the insertion of clauses into technology transfer agreements made between academic organisations receiving grants from the European Commission and for-profit companies to make reasonable pricing compulsory.

We also propose to make reimbursement of gene therapies by EU healthcare payers conditional on the companies which are commercialising these products being certified for their corporate social responsibility. This is in line with several commitments made recently by pharma companies. For example, in August 2019, the CEOs of US-based pharma companies signed the Business Roundtable Statement, affirming their commitment to generate value for all their stakeholders not just their shareholders.

Also in August, Novartis announced it had joined the Value Balancing Alliance, a body whose goal is to increase transparency around business decisions, work with external bodies to develop accounting frameworks, and shift priority from profit maximisation to optimising value creation.

Earlier this year, the pharmaceutical company Chiesi was certified as a Benefit Corporation, meaning its legally defined goals include positive social impact in addition to profit.

Of course, the effective implementation of such commitments and their translation into reasonable pricing policies will require both incentives and regulatory controls. The starting point should be a renewed multi-stakeholder conversation with industry, investors, regulators, payers and, of course, patients.

Professor Michel Goldman is Co-director of the I3h Institute at the Universit Libre de Bruxelles and former Executive Director of the EU Innovative Medicines Initiative.

See the article here:
Viewpoint: EU should take a lead in enforcing the corporate social responsibility of gene therapy manufacturers - Science Business

XconomyBoston

Ring Therapeutics, a Flagship Pioneering spinout, launched Thursday with ambitious plans to expand the universe of vectors available for gene therapy delivery.

Gene therapy, treatments intended to treat disease by inserting a gene instead of using drugs or surgery, has had a banner year, with the second ever such therapy approved this year in the US.

Ring want to use itsresearch into viruses that exist in the human body without apparent negative effects to provide more and better options to fuel the rise of gene therapy treatments.

For the past two years, Flagship Pioneering partner and Rings founding CEO Avak Kahvejian says the company has been exploring the human commensal viromebasically, a group of viruses that exist within humans without negative effectsfor its potential to address limitations of the vectors currently used.

The sector relies heavily on adeno-associated viruses (AAVs), which naturally infect humans but arent known to cause disease, to deliver the DNA. Previous exposure, however, can spark an immune response.

A lot of the workhouses in gene therapy have either been pathogenic viruses or viruses that have been taken from other species or viruses that are highly immunogenic, or all of the above, Kahvejian tells Xconomy. That leads to a certain number of limitations, despite the successes and advances weve made to date.

A number of issues stymie widespread use of AAVs, Kahvejian says, including the fact that 10 percent to 20 percent of people have at one time or another been infected with such a virus, thereby building up an immune response to it. Another concern is where such gene therapies end up, because viruses tend to gravitate toward certain types of tissues, and to go elsewhere, require special tweaking.

The Cambridge, MA-based startup believes the viruses it has found are unlikely to cause an immune response or prove pathogenic, given their ubiquity in the body.

Like extrachromosomal DNAa new discovery at least one company is exploring for its potential as a target in cancer treatmentsthe viral sequencing Ring is studying are circular pieces of DNA that exist outside the 23 chromosomes of the human genome.

Ring says it has found thousands of these viruses that coexist with our immune system. It aims to use those to develop vectors that can facilitate gene replacement throughout the bodymultiple times, if necessary. While gene therapy is thought of as a one-time fix, cell turnover means whatever the fix engendered by the inserted gene could falter over time, necessitating a re-up.

Kahvejian wouldnt share a timeline for Rings plan to develop re-dosable, tissue-targeted treatments.

Were looking at the unique features and activities of these viruses in different tissues to establish the various vectors were going to pursue, he said.

Flagship, which pursues scientific questions in-house and builds and funds companies around the answershas put $50 million toward Ring, which has about 30 employees.

Rings president is Rahul Singhvi, an operating partner at Flagship. Most recently he was chief operating officer of Takedas global vaccine business unit. Its head of R&D is Roger Hajjar, who has led gene therapy trials in patients with heart failure.

Ring is the second startup Flagship has spun out this month. Cellarity launched last week.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

Read the rest here:
Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options - Xconomy

By Dan Ross

Earlier this month, researchers out of the University of Pennsylvania published a breakthrough study which identified several biomarkers associated with shockwave therapy. This study is the first of its kind to identify a possible biological signal revealing shockwave usagean important development for a therapy that leaves no visible trace, but one that has historically generated welfare concerns due to its analgesic effects.

Because its not a drugits applied to the surface of the skinits just not an easy thing to detect, said lead researcher Mary Robinson, assistant professor of veterinary pharmacology and director of the Equine Pharmacology Laboratory at the University of Pennsylvania School of Veterinary Medicine, in a press release. After a lot of trial and error, our study was able to measure changes in levels of five inflammatory factors, some of which we could detect up to three weeks after the shockwave therapy.

The study was conducted on ostensibly healthy horses. Robinson told the TDN that she and her colleagues will continue to research these biomarkers on other populations of racehorses, including injured horses, injured horses that have received shockwave therapy, and healthy horses in training. For this next step, researchers will dip into the universitys large BioBank library of biological samples.

Robinson was also a co-author on a paper published in November proving proof of principle, she said, that they were able to detect experimental gene therapy which had been administered via intra-articular injection. Gene doping and its performance enhancing potential is an emerging problem in horse racing. Broadly speaking, the term refers to the twin practices of gene editingchanging animal DNA before theyre bornand gene therapy, which refers to the implantation of a piece of DNA into an adult animal to prevent or treat a disease or disorder.

In the following Q&A, Robinson discusses shockwave usage, the implications of her shockwave biomarker study, and her ongoing work in gene doping.

TDN: Can you discuss how the shockwave study was conducted?

MR: The study that was just published is looking at the effect of giving a single dose of shockwave to the front of the cannon bone to 11 healthy horses. Whats really novel about this study is we could find changes in the blood that were reflective of these horses receiving shockwave therapy. The biobank is for the next stepsthis initial study was more a proof of concept study.

Essentially, what we did, a week before shockwave was given, we took samples each day. After we gave the dose of shockwave, we then collected samples after that dose for up to three weeks. And we were able to see these changes occurring very rapidly, within 24 hours after the shockwave dose, and some of them lasted for up to three weeks.

What that tells me is we probably will need to follow horses out over time. And so, for each individual animal, in order to make these biomarkers make sense, were going to need to know what their normal profile looks like, and then follow that out over time.

TDN: How did you end up narrowing in on those 10 select biomarkers (of which five indicated shockwave usage)?

MR: That was more a resource limitation than anything else. Its very difficult to do these types of studies on horses, and that maybe speaks to some of the challenges we face in identifying biomarkers. One of the difficult things is there just arent the resources out there.

One of the ways we narrowed in on those 10 was to see which pro-inflammatory and anti-inflammatory proteins were similar between horses and humans, so that we might be able to use the resources available for humans to try to measure these things in horses. We spent quite a lot of time validating the methods that were used to make sure they would work for horses. And we chose pro and anti-inflammatory factors because we know shockwave causes a very acute inflammatory response. It increases the blood flow to the area.

TDN: Whats next in your research?

MR: As you mention, these were healthy horsesthey did not have an injury. And were looking at anti-inflammatory factors which you would maybe expect to be affected by an injury. We need to be able to look at what the effect of an injury is going to do to these factors, and then we need to look at what the effect of giving shockwave on top of that injury is going to do to those factors.

These horses were healthy, but they were not in any type of exercise regimen, so we also need to assess the effect of exercise on these factorsits well documented that [biomarker] IL-6 that we identified is affected by exercise. So, well need to make sure that we take that into account.

TDN: When will you start?

MR: Weve already started. Weve been working on collecting these BioBank samples for several years now. It was just in the last year and a half that weve been able to quadruple the number of samples that weve collected because of the funding from the Horse Breeders Association. Within the next year, were going to be starting to make some comparisons using the markers that weve now identified.

TDN: What can you tell us about the BioBank?

MR: The funding for the BioBank is from the Pennsylvania Horse Breeders Association. I was able to start the bank using some internal funds from the [University of Pennsylvania School of Veterinary Medicine] New Bolton Center, and then the PHBA very generously donated money for us to expand the bank, which allowed us to hire an additional person, and get some more freezers, so that we can collect samples from horses at the racetrack and in training at various training facilities.

TDN: The BioBank primarily contains Thoroughbred samples, right?

MR: Yes, although were actually going to be meeting with some other groups from the Standardbred industry to see if they would be interested in supporting it as well.

TDN: How soon before you possibly narrow in on certain shockwave biomarkers that can be used for regulatory purposes?

MR: I dont know that well ever be able to conclusively prove that shockwave had been administered. I think what we hope to do is identify instead biomarkers that suggest that theres something concerning going on in an individual horse. Whether those relate specifically to shockwave or whether those relate to a horse that has some underlying injurythat somebodys trying to cover up or somebody doesnt even know existsthats what this biological passporthopes to do.

TDN: What are your general thoughts about shockwave usage for horses in training?

MR: Currently in Pennsylvania, we dont allow it on the backside of any of the tracks, and that really is because its not something that we can detect currently. We do not want to have it risked being used too close to a race, and thats a problem with shockwave: it has a very strong analgesic effect, just as effective as the nerve block when its used on an injury. The key is, it can be used as a therapeutic, but you have to absolutely rest the horse. You cannot run the horse shortly after its received shockwave.

TDN: Do you agree with the Association of Racing Commissioners Internationals model rule requiring a 10-day stand-down, given what we currently know about the analgesic effect?

MR: I do. But theres only one study thats definitively shown that theres an analgesic affect. I think that they stopped around two days in that study, and its not really clear whether that effect would have lasted longer or not. Theres potential that it could have an analgesic effect for a longer period of time.

TDN: What are the implications of gene doping for horse racing?

MR: Its another new way to treat disease. Instead of having to give multiple doses, the whole point of gene therapy is you can give a single dose and have a more permanent treatment. So, when you think of it in that regard, the potential for using it illicitly to try to enhance performance is huge, because a single dose might be able to create a lasting effect. So, I do think its a serious concern. Its been on the WADA [World Anti-Doping Agency] anti-doping list since 2003. So, our human colleagues have certainly been concerned about it for quite some time.

TDN: Do things that go on in human athletics often makes their way into horse racing?

MR: We certainly have seen that pattern, although the reverse is possible. Some people may feel more comfortable experimenting with horses than with humans. Of course, cost always becomes an issue. But its possible there are people with the means who are willing to try and win at all costs. So, thats what were trying to prevent.

TDN: How big an issue is it currently for horse racing?

MR: Its really hard to know at this point. There certainly has been suspicion and discussion and talk about it. I have to say I was pretty skeptical that this was a problem until more recently. Now that the therapies are being approved in humans, theyve accelerated the ability for people to actually develop these therapies, so, I think its something we need to address as soon as possible as an industry.

Not a subscriber? Click here to sign up for the daily PDF or alerts.

Read the rest here:
Q&A: UPenn's Mary Robinson Talks Shockwave and Gene Doping - Thoroughbred Daily News

DetailsCategory: More NewsPublished on Friday, 20 December 2019 13:54Hits: 103

Selecta eligible to receive upfront and milestone payments of over $240 million

AskBios AAV gene therapy combined with ImmTOR could prevent the formation of neutralizing antibodies and potentially enable re-treatment of patients with Pompe disease

WATERTOWN, MA and RESEARCH TRIANGLE PARK, NC, USA I December 19, 2019 ISelecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced that the companies have entered into a license agreement. Under the terms of the agreement, AskBio has exercised its option to exclusively license rights to develop and commercialize Selectas immune tolerance platform, ImmTOR, for use in adeno-associated virus (AAV) gene therapy for the treatment of Pompe disease. When used in combination with AAV gene therapy vectors, Selectas ImmTOR has been shown to inhibit the immune response to the vector (Nature Communications, October 2018).

Selecta and AskBio previously announced a strategic partnership in August 2019 to jointly develop, manufacture, and commercialize targeted therapeutics for next-generation AAV gene therapies in areas of high medical need. Under the terms of this new license agreement, Selecta is eligible to receive upfront and milestone payments of over $240 million plus royalties on product sales.

We are pleased to advance our ImmTOR platform in a new partnership with AskBio for Pompe disease, a serious and progressively debilitating disease, said Carsten Brunn, Ph.D., President and Chief Executive Officer of Selecta. We are excited to collaborate with AskBio to potentially enable the retreatment of AAV gene therapies.

There is a demonstrated unmet medical need for better treatment approaches for Pompe disease, and this collaboration will enable us to effectively advance our Pompe program with the added benefit of Selectas ImmTOR technology, said Sheila Mikhail, CEO and co-founder of AskBio. The opportunity to re-treat patients holds significant promise, and we are pleased to be able to leverage our relationship with Selecta and apply the ImmTOR technology to potentially overcome the challenges associated with re-administering systemic AAV gene therapies.

Pompe disease is a rare, progressive, debilitating condition that affects 5,000 to 10,000 people worldwide. It impacts ventilator, cardiac and skeletal muscles and can cause motor neuron dysfunction, with effects on cognition, hearing, speech and fine motor skills. Pompe disease can manifest any time between infancy and late adulthood and may have differing symptoms and severity, depending on when it develops. There is a substantial unmet medical need for more efficient and effective treatments for Pompe disease. Currently, the only approved treatment is ERT with recombinant human GAA (rhGAA), a chronically administered therapy requiring increasing doses, which can elevate both costs and treatment burden.

AboutSelecta Biosciences, Inc. Selecta Biosciences, Inc.is a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance technology (ImmTOR) platform.Selectaplans to combine ImmTOR with a range of biologic therapies for rare and serious diseases that require new treatment options due to high immunogenicity. The companys current proprietary pipeline includes ImmTOR-powered therapeutic enzyme and gene therapy product candidates. SEL-212, the companys lead product candidate, is being developed to treat chronic refractory gout patients and resolve their debilitating symptoms, including flares and gouty arthritis. Selectas proprietary gene therapy product candidates are in preclinical development for certain rare inborn errors of metabolism and incorporate ImmTOR with the goal of addressing barriers to repeat administration.Selectais based inWatertown, Massachusetts. For more information, please visithttp://selectabio.com.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy platform company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10 and an extensive AAV capsid library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors and self-complementary DNA. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit https://www.askbio.com/.

About Pompe DiseasePompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

SOURCE: AskBio

See the rest here:
AskBio Licenses Rights to Selecta Biosciences' ImmTOR Immune Tolerance Platform for the Treatment of Pompe Disease | More News | News Channels -...

In a recent study published by Prophecy Market Insights, titled, Global Gene Therapy for Rare Disease Market Research Report, analysts offers an in-depth analysis of global Gene Therapy for Rare Disease market. The study analyses the various aspect of the market by studying its historic and forecast data. The research report provides Porters five force model, SWOT analysis, and PESTEL analysis of the Gene Therapy for Rare Disease market. The different areas covered in the report are Gene Therapy for Rare Disease market size, drivers and restrains, segment analysis, geographic outlook, major manufacturers in the market, and competitive landscape.

Key Players of Gene Therapy for Rare Disease Market:

Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., UniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and Biomarin Pharmaceutical Inc.

Download Sample Copy of This Report @ https://prophecymarketinsights.com/market_insight/Insight/request-sample/397

The research report, Gene Therapy for Rare Disease Market presents an unbiased approach at understanding the market trends and dynamics. Analysts have studied the historical data pertaining to the market and compared it to the current market trends to paint an object picture of the markets trajectory. The report includes SWOT analysis and Porters five forces analysis to give the readers an in-depth assessment of the various factors likely to drive and restrain the overall market.

Market Segmentation:

Request PDF catalogue for this report @ https://prophecymarketinsights.com/market_insight/Insight/request-pdf/397

Table of Contents

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the global Gene Therapy for Rare Disease market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the global Gene Therapy for Rare Disease market is analyzed, taking into consideration price, revenue, sales, and market share by company, market concentration rate, competitive situations and trends, expansion, merger and acquisition, and market shares of top 5 and 10 companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the global Gene Therapy for Rare Disease market as well as some useful information on their business. It talks about the gross margin, price, revenue, products and their specifications, applications, competitors, manufacturing base, and the main business of players operating in the global Gene Therapy for Rare Disease market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Gene Therapy for Rare Disease market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This part of the research study shows how different application segments contribute to the global Gene Therapy for Rare Disease market.

Market Forecast: Here, the report offers complete forecast of the global Gene Therapy for Rare Disease market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Upstream Raw Materials: The report provides analysis of key raw materials used in the global Gene Therapy for Rare Disease market, manufacturing cost structure, and the industrial chain.

Marketing Strategy Analysis and Distributors: This section offers analysis of marketing channel development trends, indirect marketing, and direct marketing followed by a broad discussion on distributors and downstream customers in the global Gene Therapy for Rare Disease market.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

Appendix: Here, we have provided a disclaimer, our data sources, data triangulation, market breakdown, research programs and design, and our research approach.

For More Info: https://prophecymarketinsights.com/market_insight/Global-Gene-Therapy-for-Rare-397

Contact Us:

Mr. Alex (Sales Manager)

Prophecy Market Insights

Phone: +1 860 531 2701

Email: [emailprotected]

Browse Similar Reports:

Bleaching Agent Market to Reap Excessive Revenues by 2020 – 2030

Isobutane Market Evolving Latest Trends to Lead Global Industry By 2020 – 2030

Propylene Oxide Market Research Report By Drivers, Trends, Opportunities and Restraints Prediction to 2020 – 2030

Read this article:
Gene Therapy for Rare Disease Market to Witness Increased Incremental Dollar Opportunity During the Forecast Period 2020 2030 - Bulletin Line

Morsa Images/ Getty Images

Obamacare wasn't just about coverage. The health law created the Center for Medicare and Medicaid Innovation, fueling the move to value-based care, or paying doctors and hospitals for making patients healthy, rather than for each visit or surgery. While many of center's experiments focus on the Medicare health program for the elderly, private insurers are taking similar approaches across the US.

Andy Slavitt, the acting Centers for Medicare and Medicaid Services administrator from 2015 to 2017, said the health law's creation of the innovation center, together with the private experimentation, was creating rapid changes in US healthcare.

"In effect, you're saying to the healthcare system, instead of thinking about how to make revenue, we're going to give you a fixed revenue amount, and you think about the things you can control for," Slavitt said. Slavitt is now a founder of the healthcare research group United States of Care and a general partner at the venture-capital firm Town Hall Ventures.

And while Obamacare helped fuel the move to value, the Trump administration has continued to push the healthcare system in the same direction.

"There's been bipartisan support and, and I would say, work throughout the entire system around value-based care," said Seema Verma, the current Centers for Medicare and Medicaid Services administrator. "There's a lot of hope around and consensus around value-based care."

Read more from the original source:
Biggest healthcare and biotech developments of the decade - Business Insider Nordic

() CEO Doug Doerfler tells Proactive it has appointed a new executive vice president as it continues with a Phase I trial of its MCY-M11 cancer drug. Doerfler says Shruti Abbato will serve as executive VP of business development for its CARMA cellular therapies, which is aiming to provide faster treatment than existing cancer therapies.

Add related topics to MyProactive

Create your account: sign up and get ahead on news and events

The Company is a publisher. You understand and agree that no content published on the Site constitutes a recommendation that any particular security, portfolio of securities, transaction, or investment strategy is...

In exchange for publishing services rendered by the Company on behalf of MaxCyte Inc named herein, including the promotion by the Company of MaxCyte Inc in any Content on the Site, the Company receives from said...

Read more from the original source:
MaxCyte appoints life-sciences vet as EVP, updates on lead therapy to treat solid tumors - Proactive Investors UK

A leading research firm, Zion Market Research added a latest industry report on "Global Gene Therapy Market" consisting of 110+ pages during the forecast period and Gene Therapy Market report offers a comprehensive research updates and information related to market growth, demand, opportunities in the global Gene Therapy Market.

According to the report the Future of Gene Therapy Market Reviewed in a New Research Study 2018-2026

The Gene Therapy Market report provides in-depth analysis and insights into developments impacting businesses and enterprises on global and regional level. The report covers the global Gene Therapy Market performance in terms of revenue contribution from various segments and includes a detailed analysis of key trends, drivers, restraints, and opportunities influencing revenue growth of the global consumer electronics market.This report studies the global Gene Therapy Market size, industry status and forecast, competition landscape and growth opportunity. This research report categorizes the global Gene Therapy Market by companies, region, type and end-use industry.

Request a Free Sample Report on Gene Therapy Market:https://www.zionmarketresearch.com/sample/gene-therapy-market

The Gene Therapy Market report mainly includes the major company profiles with their annual sales & revenue, business strategies, company major products, profits, industry growth parameters, industry contribution on global and regional level.This report covers the global Gene Therapy Market performance in terms of value and volume contribution. This section also includes major company analysis of key trends, drivers, restraints, challenges, and opportunities, which are influencing the global Gene Therapy Market. Impact analysis of key growth drivers and restraints, based on the weighted average model, is included in this report to better equip clients with crystal clear decision-making insights.

The Gene Therapy Market research report mainly segmented into types, applications and regions.The market overview section highlights the Gene Therapy Market definition, taxonomy, and an overview of the parent market across the globe and region wise.To provide better understanding of the global Gene Therapy Market, the report includes in-depth analysis of drivers, restraints, and trends in all major regions namely, Asia Pacific, North America, Europe, Latin America and the Middle East & Africa, which influence the current market scenario and future status of the global Gene Therapy Market over the forecast period.

Get Free PDF Brochure of this Report: https://www.zionmarketresearch.com/requestbrochure/gene-therapy-market

The Gene Therapy Market report provides company market size, share analysis in order to give a broader overview of the key players in the market. Additionally, the report also includes key strategic developments of the market including acquisitions & mergers, new product launch, agreements, partnerships, collaborations & joint ventures, research & development, product and regional expansion of major participants involved in the market on the global and regional basis.

Major Company Profiles Covered in This Report:

UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

Some of the major objectives of this report:

1) To provide detailed analysis of the market structure along with forecast of the various segments and sub-segments of the global Gene Therapy Market.

2. To provide insights about factors affecting the market growth. To analyze the Gene Therapy Market based on various factors- price analysis, supply chain analysis, porter five force analysis etc.

3. To provide historical and forecast revenue of the Gene Therapy Market segments and sub-segments with respect to four main geographies and their countries- North America, Europe, Asia, and Rest of the World.

4. Country level analysis of the market with respect to the current market size and future prospective.

5. To provide country level analysis of the market for segment by application, product type and sub-segments.

6. To provide strategic profiling of key players in the market, comprehensively analyzing their core competencies, and drawing a competitive landscape for the market.

7. Track and analyze competitive developments such as joint ventures, strategic alliances, mergers and acquisitions, new product developments, and research and developments in the global Gene Therapy Market.

About Us:

Zion Market Research is an obligated company. We create futuristic, cutting edge, informative reports ranging from industry reports, company reports to country reports. We provide our clients not only with market statistics unveiled by avowed private publishers and public organizations but also with vogue and newest industry reports along with pre-eminent and niche company profiles. Our database of market research reports comprises a wide variety of reports from cardinal industries. Our database is been updated constantly in order to fulfill our clients with prompt and direct online access to our database. Keeping in mind the clients needs, we have included expert insights on global industries, products, and market trends in this database. Last but not the least, we make it our duty to ensure the success of clients connected to usafter allif you do well, a little of the light shines on us.

Contact Us:

Zion Market Research

244 Fifth Avenue, Suite N202

New York, 10001, United States

Tel: +49-322 210 92714

USA/Canada Toll Free No.1-855-465-4651

Email: sales@zionmarketresearch.com

Website: https://www.zionmarketresearch.com

Sorry! The Author has not filled his profile.

Read the original here:
Future of Gene Therapy Market Reviewed in a New Research Study 2018-2026 - Industry News Ledger

Transparency Market Research (TMR) has published a new report titled, Viral Vector & Plasmid DNA Manufacturing Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20192027. According to the report, theglobal viral vector & plasmid DNA manufacturing marketis expected to exceed a value of US$ 400 Mn by the end of 2019. The global market is anticipated to surpass US$ 2 Bn by 2027 and expand at a high double digit CAGR from 2019 to 2027. Rise in prevalence of cancer, genetic disorders, and increase in number of clinical studies are expected to augment the global market from 2019 to 2027. The viral vector & plasmid DNA manufacturing market is projected to expand owing to an increase in the awareness regarding viral vector based treatments and technological advancements in developing countries.

Increasing prevalence of cancer, genetic diseases, and infectious diseases

According to Cancer Research UK, there were 17 million new cases of cancer in 2018 and four most common types of cancer worldwide were breast, lung, bowel, and prostate cancers, which account for approximately 43% of all new cases. According to WHO estimates, currently, 10,000 of human diseases are known to be monogenic, caused by modifications in a single gene in human DNA. The global prevalence of all single gene diseases at birth is approximately 10/1000. In Canada, it is estimated that monogenic diseases may account for approximately 40% of the work of hospital-based pediatric practice. Increased prevalence of such disorders demands improved treatments, which in turn is anticipated to propel the viral vector & plasmid DNA manufacturing market during the forecast period.

Request a Sample of Viral Vector and Plasmid DNA Manufacturing Market Report

https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=30428

Increase in awareness regarding gene therapy

Awareness regarding gene therapy is increasing worldwide, as compared to the last few decades. According to an article published by Human Gene Therapy, the acceptance of gene therapy for severe disorders, such as Alzheimer Disease, is high as compared to less severe disease, such as attention deficit hyperactivity disorder. Furthermore, acceptability of gene therapy is increasing, and there is a strong need to provide the public and patients with up-to-date information. Moreover, opportunities to engage in the discourse about areas of research in gene therapy is a priority. This is estimated to propel the viral vector & plasmid DNA manufacturing market during the forecast period.

Cancer segment dominates the global market due to large number of clinical trials ongoing worldwide

In terms of disease, the cancer segment dominated the global viral vector & plasmid DNA manufacturing market, followed by genetic disorders. The segment accounted for a prominent market share, due to availability of approved viral vector-based cancer drugs and several ongoing clinical trials for the treatment of a variety of cancers. This creates lucrative opportunity for entry into the viral vector & plasmid DNA manufacturing market.

Plasmid DNA segment dominates the global market due to wide use in the manufacturing of viral vectors as well as DNA based vaccines

In terms of type, plasmid DNA is a highly attractive segment of the global viral vector & plasmid DNA manufacturing market, followed by the adeno-associated virus (AAV) segment. This is attributable to the extensive utilization of plasmid DNA as raw material in the manufacturing of various viral vectors.Furthermore, several studies have emphasized the benefits of DNA- based vaccines over conventional vaccines. The adeno-associated virus (AAV) segment is anticipated to expand at a considerable CAGR during the forecast period, due to several advantages offered by AAV over other vectors, making them the vector of choice for various clinical trials.

Request for a Discount on Viral Vector and Plasmid DNA Manufacturing Market Report -.

https://www.transparencymarketresearch.com/sample/sample.php?flag=D&rep_id=30428

North America dominates the global market owing to high acceptance of viral vector based treatments in the region

North America dominates the global viral vector & plasmid DNA manufacturing market due to a large patient pool, technological advancements, and high acceptance of advanced treatments in the region. The region is estimated to maintain its dominance during the forecast period. Moreover, rising healthcare expenditure, availability of approved gene therapy treatments, and increasing investments are key factors that are anticipated to boost the viral vector & plasmid DNA manufacturing market in the next few years. The viral vector & plasmid DNA manufacturing market in Asia Pacific is projected to expand at a notable CAGR due to increasing awareness regarding viral vector based products in developing countries and rising research initiatives in countries such as Japan and China.

Investments by key players is driving the globalviral vector & plasmid DNA manufacturing market

Major players operating in the viral vector & plasmid DNA manufacturing market include CobraBiologics, Novasep Inc., Cell and Gene Therapy Catapult, Kaneka Eurogentec S.A., FUJIFILM Diosynth Biotechnologies Inc., Spark Therapeutics, Inc. Merck KGaA, uniQure N.V., and Lonza.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through adhoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key to help enterprises reach right decision.

ContactTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY 12207United StatesTel:+1-518-618-1030USA Canada Toll Free:866-552-3453Email:[emailprotected]Website:http://www.transparencymarketresearch.com

Continue reading here:
Viral Vector and Plasmid DNA Manufacturing Market is Estimated to Expand at a Robust CAGR by 2027 - Testifyandrecap

Takeda is spending big to add a new piece to their oncology R&D puzzle.

This morning the global pharma company picked up an alliance with Turnstone Biologics, which has been building a new viral immunotherapy platform to complement its work on oncolytics, partnered with AbbVie for the past 2 years.

Dubbed the vaccinia virus platform out of a lab in Ottawa, R&D chief Mike Burgess describes it as a highly selective virus as a consequence of engineering, exquisitely selective for cancer cells in contrast to normal cells. And it can be used to deliver a payload of transgenes for Flt3 ligand, anti-CTLA-4 antibody, and IL-12 cytokine that replicate in cells.

Takeda is offering up a smorgasbord of cash to close the deal, with $120 million flowing to Turnstone for the upfront, near-term milestones and an upcoming equity investment which goes a long way to funding its next stage of development. Theres also $900 million more in longer-range milestones on the table.

Turnstone has been low key for the last few years, since AbbVie stepped up with an option deal on their oncolytics work, part of a wave of development work aimed at going Amgens Imlygic one better. Turnstone CEO Sammy Farah tells me the pact is still in place something AbbVie confirmed for me as well but has no interest in getting into the details of whats been going on there.

But hes a lot more voluble about the vaccinia platform.

Drawn from the lab of John Bell and his colleagues at The Ottawa Hospital Research Institute and the University of Ottawa, Turnstone turned up at AACR a little more than a year ago to offer preclinical mouse data to back up the potential in using it to fight cancer.

The reason why its so exciting, it offers a multi-pronged attack on cancer, says the CEO. Single modalities dont cut it anymore, but a combination combined with the therapeutic properties of theirs itself can be cutting edge in new therapies.

View post:
Takeda puts $120M in near-term cash on the table to complete a new oncology platform deal - Endpoints News

Earlier this year, the US Food and Drug Administration approved the most expensive drug ever to hit the market, a gene therapy for spinal muscular atrophy. SMA is a neuromuscular disorder that, in severe cases, can lead to infant death. The genetic correction is currently used to treat affected newborns, but as symptoms for some types of SMA may appear before birth, an earlier treatment would be potentially more effective.

In a study published December 4 in Molecular Therapy, researchers were able to fix a mutation in the survival motor neuron 1 (SMN1) genewhich causes SMA in humansin mice modelling the disease, while they were still inside their mothers uterus. The treated mice lived longer and had fewer symptoms than untreated animals.

Tippi MacKenzie, a fetal and pediatric surgeon at the University of California, San Francisco, who did not participate in this study, says it is an important paper because it is the first time fetal gene therapy has succeeded in SMA mice. Before you even think about doing something in patients, you have to first do it in the disease model of the mouse . . . so this group has supplied a very important piece to the literature, she adds.

SMN1encodes an essential protein for the maintenance of motor neurons, which are nerve cells in the brain and spinal cord responsible for controlling muscle movement. The result in children with mutations in the gene is the loss of motor neurons, leading to muscle weakness and associated complications. SMA affects one out of every 6,000 to 10,000 babies.

Correcting the SMN1 sequence is a potentially efficient treatment for those born with SMA. Zolgensma, the recently approved medication for this disorder, consists of an intravenous administration of an adeno-associated virus that ferries a functional copy of the SMN1 gene to the brain.

To see if the same fix could be accomplished before birth, the research team tested two different injection methods: one into the placenta (intraplacental or IP) and the other into one of the brain lateral ventricles (intracerebroventricular or ICV). The latter proved to be more effective. By injecting the viral vector into the fetuss brain, the virus will go directly into the cerebrospinal fluid, and it will transduce motor neurons in the spinal cord with a very high efficiency, compared to the IP [injection], says Afrooz Rashnonejad. who participated in this study while working at Ege University in Izmir, Turkey, but has recently moved to Nationwide Childrens Hospital in Columbus, Ohio.

Rashnonejad and her colleagues then monitored the injected mice that were carried to term. Those treated with the vector carrying a functional copy of SMN1 lived a median lifespan of 63 or 105 days (depending on the type of cassette carrying the gene), much longer than untreated SMA mice, which did not survive more than 14 days, but still less than wildtype pups, which had a median lifespan of 405 days. The treated mice were also heavier than untreated mice, but smaller than healthy mice.

The investigators also observed differences at the cellular and molecular levels. SMN protein levels were completely recovered in the brain and spinal cord, and the number of motor neurons was higher in treated animals.

I was just very impressed by what theyve done, says Simon Waddington, a gene therapy researcher at University College London who did not participate in this work, but was one of the reviewers of the paper. He adds that he and other colleagues had previously tried fetal gene therapy on SMA mice, but had failed as it is a technically difficult experiment. So it was really nice to see this group actually did a really good job.

This is the first time viral vectors have been used to successfully boost gene expression in SMA mice before birth. Interventions to edit the genome in utero have been previously used in mice that model other severe genetic diseases. Last year, for instance, Waddington and colleagues used fetal gene therapy to treat mice affected by Gaucher disease, a neurodegenerative disorder that can be fatal for newborns. Other successful attempts include intrauterine gene editing for mice affected by -thalassemia, an inherited blood disorder, and mice suffering a monogenic lung disease that normally results in newborn death.

MacKenzie says that, in a recent national meeting on in utero gene therapy, it was discussed how to move forward with a clinical application to the FDA. We are definitively moving towards that direction, but we dont have a particular application yet, because its still not clear which disease should be the first.

SMA makes a lot of sense because its so severe, MacKenzie adds. But at the same time, the results that are coming out at conferences, she observes, suggest that newborn babies receiving Zolgensma are doing pretty well, better than anybody could have imagined. So its not clear that you have to go before birth. A good candidate, she explains, would be a very rare type of SMA, where the baby dies before birth.

Waddington says that researchers might have to wait for neonatal gene therapy to become standard for certain diseases before using fetal gene therapy in humans. Once we actually understand how efficient this is, and if we come to the point where we discover that the earlier that you go the more effective it is . . . in a human setting, then we may be able to do fetal gene therapy. I think that we are looking at more than five years away before thats even likely to happen, he hypothesizes.

A. Rashnonejad et al., Fetal gene therapy using a single injection of recombinant AAV9 rescued SMA phenotype in mice,Molecular Therapy, 27:212333, 2019.

Alejandra Manjarrez is a freelance science journalist. Email her atalejandra.manjarrezc@gmail.com.

See the article here:
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy - The Scientist

A vector is a microscopic carrier of pieces of DNA. It is used to deliver healthy copies of genes to tissues and organs within patients or deliver the ability to correct the genetic errors. While the technology is moving rapidly, production of vectors is not.

NSW, and in particular the Westmead Precinct, is already at the forefront of international gene therapy research. The aim of this project is to speed up research and translate it into cures for serious genetic diseases affecting children.

The facility will produce vectors to treat illnesses impacting everything from those with life-threatening liver disease to children going blind. Currently the vectors need to imported and its extremely costly to get them to Australia.

Professor Ian Alexander, Head of the Gene Therapy Research Unit at Childrens Medical Research Institute, senior clinician at The Childrens Hospital at Westmead and Professor of Paediatric and Molecular Medicine at the University of Sydney, said the manufacturing facility would be a boost to translation of academic research in NSW.

We see it as the beginning of something much greater, Professor Alexander said.

It is about moving technology into the clinic, which, in future, will benefit many more patients by offering new and better treatment opportunities. This technology could translate into saving the lives of infants with life-threatening conditions.

Dr Leszek Lisowski heads the Translational Vectorology Group at CMRI and is Conjoint Senior Lecturer at the University of Sydney. His team will play a key role in the new facility, through training of staff and developing the manufacturing processes that will underpin operations. In addition, his team specialises in the development of novel vectors optimised for clinical applications targeting liver, eye and many other clinically important organs and tissues.

Dr Lisowski said that this new facility will allow Australian investigators to get around the "bottleneck" of getting vectors from overseas.

The biggest bottleneck that slows down translation of gene therapy tools to the patient is a global lack of vector manufacturing capacity, which significantly extends the timeline and increases the cost of translational studies," he said.

This facility will give Australian researchers prioritised and cost-effective access to clinical gene therapy reagents and will facilitate translation of a large number of exciting preclinical programs from bench to bedside.

The team is excited by this vital investment and looks forward to partnering with government and other funders to enable the facility to achieve its full potential.

The Westmead Precinct is one of the largest health, education, research and training precincts in Australia and a key provider of jobs for the greater Parramatta and western Sydney region. Spanning 75 hectares, the Precinct includes four hospitals, four world-leading medical research institutes, two multidisciplinary university campuses and the largest research-intensive pathology service in NSW.

The University of Sydney has long been a proud partner of the Precinct and is in negotiations about developing a second major campus in the area. By 2050, that campus will include 25,000 students; 1000 staff and researchers; generate $21.7 billion for the NSW economy and support up to 20,000 jobs.

University of Sydney Vice-Chancellor and Principal Dr Michael Spence said that as part of our collaborative work in building a western Sydney global centre of excellence, Precinct partners are growing Australias advanced manufacturing capability.

These developments will strengthen crucial collaborations in the Precinct from R&D and design to distribution in areas such as prevention and wellbeing, biomedical engineering, AI and personalised medicine, Dr Spence said.

Faculty of Medicine and Health Executive Dean Professor Robyn Ward said: This technology will scale up gene therapy using viral vectors from single-condition, life changing successes, for example in spinal muscle atrophy, to a national service.

We are so proud of this leadership at the Westmead Precinct and with our health partners. It is a whole-of-lifespan, true bench-to-clinic approach."

Go here to read the rest:
Westmead advanced manufacturing to transform lives - News - The University of Sydney

Abstract / Synopsis:

Two anti-VEGF gene therapies are being investigated in clinical trials of patients with exudative age-related macular degeneration. Initial efficacy and safety results are encouraging.

Anti-VEGF gene therapy for exudative age-related macular degeneration (AMD) has transformative potential for reducing treatment burden and improving patient outcomes, according to Szilrd Kiss, MD.

Two investigational anti-VEGF gene therapies are currently being investigated in clinical trialsRGX-314 (Regenxbio) and ADVM-022 (Adverum). Dr. Kiss described the two technologies and reviewed some preliminary clinical trial results that support their promise for providing sustained benefit with a single injection.

Considering the treatment burden of anti-VEGF therapy for other ocular diseases, we can imagine that exudative AMD is just the first indication that will be targeted for anti-VEGF gene therapy, said Dr. Kiss, chief, Retina Service, associate professor of ophthalmology, and associate dean at Weill Cornell Medical College, New York, NY.

RGX-314 delivers a gene for an anti-VEGF fab protein that is similar to ranibizumab. It uses adeno-associated virus-8 (AAV8) as a vector and is administered in the operating room as a subretinal injection.

AAV is the most common viral vector carrier used for gene therapy. Different AAV serotypes have different tissue selectivity, Dr. Kiss explained. AAV8 is a wild type AAV that has the propensity for greater transfection of retinal cells compared with AAV2 following subretinal gene therapy delivery.

RELATED:AAO 2019: Encouraging results revealed from early trial of subretinal gene therapy for wet AMD

Disclosures:

Szilrd Kiss, MDe: [emailprotected]This article was adapted from Dr. Kiss presentation at the 2019 meeting of the American Academy of Ophthalmology. Dr. Kiss is a consultant to RegenxBio and Spark Therapeutics and is a consultant and equity owner in Adverum.

Read more from the original source:
Research targets gene therapy for exudative AMD patients - Modern Retina

BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, is hosting an R&D Day today beginning at 8am Eastern Time. During the event, Sangamo executives and scientists plan to provide updates across the Companys clinical and preclinical pipeline, as well as an overview of manufacturing capabilities to support clinical and commercial supply. A live webcast link will be available on the Events and Presentations page of the Sangamo website

The talent, R&D capabilities, manufacturing expertise, and operations infrastructure we have brought to Sangamo have enabled us to advance a genomic medicine pipeline that spans multiple therapeutic areas and now also extends into late-stage development, said Sandy Macrae, CEO of Sangamo. As we make progress in clinical development, we gain insights into the use of our technology and are applying those insights as we advance new programs, such as the gene therapy for PKU and the genome regulation candidates for CNS diseases we are announcing today.

Macrae continued: We will continue to pursue a dual approach of retaining certain programs for our proprietary pipeline while also establishing pharmaceutical partnerships to gain access to therapeutic area expertise and financial, operational, and commercial resources. Strategic collaborations will be a particularly important consideration as we advance programs for diseases affecting large patient populations.

R&D Day updates on clinical and preclinical pipeline programs:

Gene therapy product candidates for hemophilia A, Fabry disease, and PKU

SB-525 is a gene therapy product candidate for hemophilia A being developed by Sangamo and Pfizer under a global development and commercialization collaboration agreement. The transfer of the SB-525 IND to Pfizer is substantially completed. Pfizer is advancing SB-525 into a Phase 3 registrational study in 2020 and has recently begun enrolling patients into a Phase 3 lead-in study.

At R&D Day, Sangamo executives are presenting data from the SB-525 program which were recently announced at the American Society of Hematology (ASH) annual meeting.

The cassette engineering, AAV engineering and manufacturing expertise which Sangamo used in the development of SB-525 are also being applied to the ST-920 Fabry disease program, which is being evaluated in a Phase 1/2 clinical trial, as well as to the newly announced ST-101 gene therapy program for PKU, which is being evaluated in preclinical studies with a planned IND submission in 2021.

Engineered ex vivo cell therapy candidates for beta thalassemia, kidney transplantation, and preclinical research in multiple sclerosis (MS)

Sangamo is providing an overview of the Companys diversified cell therapy pipeline this morning. Cell therapy incorporates Sangamos experience and core strengths, including cell culture and engineering, gene editing, and AAV manufacturing. At R&D Day, Sangamo scientists today are reviewing the early data presented this month at ASH from the ST-400 beta thalassemia ex vivo gene-edited cell therapy program, which is being developed in partnership with Sanofi.

Sangamo is also providing updates on the companys CAR-TREG clinical and preclinical programs. CAR-TREGS are regulatory T cells equipped with a chimeric antigen receptor. Sangamo is the pioneer in CAR-TREGS, which may have the potential to treat inflammatory and autoimmune diseases. TX200 is being evaluated in the STEADFAST study, the first ever clinical trial evaluating a CAR-TREG cell therapy. Tx200 is being developed for the prevention of immune-mediated organ rejection in patients who have received a kidney transplant, a significant unmet medical need. Results from this trial will provide data on safety and proof of mechanism, building a critical understanding of CAR-TREGS in patients, and may provide a gateway to autoimmune indications such as Crohns disease and multiple sclerosis (MS). Sangamo is also presenting preclinical murine data demonstrating that CAR-TREGS accumulate and proliferate in the CNS and reduce a marker of MS.

In vivo genome editing optimization

Clinical data presented earlier this year provided evidence that Sangamo had successfully edited the genome of patients with mucopolysaccharidosis type II (MPS II) but also suggested that the zinc finger nuclease in vivo gene editing reagents were under-dosed using first-generation technology. Sangamo has identified potential improvements that may enhance the potency of in vivo genome editing, including increasing total AAV vector dose, co-packaging both ZFNs in one AAV vector, and engineering second-generation AAVs, ZFNs, and donor transgenes.

Genome regulation pipeline candidates targeting neurodegenerative diseases including Alzheimers and Parkinsons

Sangamo scientists today are presenting data demonstrating that the companys engineered zinc finger protein transcription factors (ZFP-TFs) specifically and powerfully repress key genes involved in brain diseases including Alzheimers, Parkinsons, Huntingtons, ALS, and Prion diseases. Sangamo is advancing its first two genome regulation programs toward clinical development:

Sangamo scientists are also presenting data demonstrating progress in the development of new AAV serotypes for use in CNS diseases.

Manufacturing capabilities and strategy

Sangamo is nearing completion of its buildout of a GMP manufacturing facility at the new Company headquarters in Brisbane, CA. This facility is expected to become operational in 2020 and to provide clinical and commercial scale manufacturing capacity for cell and gene therapy programs. The Company has also initiated the buildout of a cell therapy manufacturing facility in Valbonne, France. Sangamos manufacturing strategy includes in-house capabilities as well as the use of contract manufacturing organizations, including a long-established relationship with Thermo Fisher Scientific for clinical and large-scale commercial AAV manufacturing capacity.

R&D Day webcast

A live webcast of the R&D Day, including audio and slides, will be available on the Events and Presentations page of the Sangamo website today at 8am Eastern Time. A replay of the event will be archived on the website.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Sangamo Forward Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of United States securities law. These forward-looking statements include, but are not limited to, the therapeutic potential of Sangamos product candidates; the design of clinical trials and expected timing for milestones, such as enrollment and presentation of data, the expected timing of release of additional data, plans to initiate additional studies for product candidates and timing and design of these studies; the expected benefits of Sangamos collaborations; the anticipated capabilities of Sangamos technologies; the research and development of novel gene-based therapies and the application of Sangamos ZFP technology platform to specific human diseases; successful manufacturing of Sangamos product candidates; the potential of Sangamos genome editing technology to safely treat genetic diseases; the potential for ZFNs to be effectively designed to treat diseases through genome editing; the potential for cell therapies to effectively treat diseases; and other statements that are not historical fact. These statements are based upon Sangamos current expectations and speak only as of the date hereof. Sangamos actual results may differ materially and adversely from those expressed in any forward-looking statements. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to dependence on the success of clinical trials; the uncertain regulatory approval process; the costly research and development process, including the uncertain timing of clinical trials; whether interim, preliminary or initial data from ongoing clinical trials will be representative of the final results from such clinical trials; whether the final results from ongoing clinical trials will validate and support the safety and efficacy of product candidates; the risk that clinical trial data are subject to differing interpretations by regulatory authorities; Sangamos limited experience in conducting later stage clinical trials and the potential inability of Sangamo and its partners to advance product candidates into registrational studies; Sangamos reliance on itself, partners and other third-parties to meet clinical and manufacturing obligations; Sangamos ability to maintain strategic partnerships; competing drugs and product candidates that may be superior to Sangamos product candidates; and the potential for technological developments by Sangamo's competitors that will obviate Sangamo's gene therapy technology. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamos operations. This presentation concerns investigational drugs that are under preclinical and/or clinical investigation and which have not yet been approved for marketing by any regulatory agency. They are currently limited to investigational use, and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated. Any discussions of safety or efficacy are only in reference to the specific results presented here and may not be indicative of an ultimate finding of safety or efficacy by regulatory agencies. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission on March 1, 2019 and Sangamo's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 that it filed on or about November 6, 2019. Except as required by law, we assume no obligation, and we disclaim any intent, to update these statements to reflect actual results.

See the original post:
Sangamo Highlights Advancements in Genomic Medicine Pipeline and Expanded R&D and Manufacturing Capabilities at R&D Day - Business Wire

Recent research on longevity is making the idea of an elixir of life sound increasingly plausible. But a startup thats started selling a $1 million anti-aging treatment is most likely jumping the gun.

Libella Gene Therapeutics says it will administer volunteers with a gene therapy that it claims can reverse aging by up to 20 years, according to OneZero. Despite the fact that this is the first human trial of the treatment, the company is charging volunteers $1m to take part. In an effort to side-step the FDA, the trial will take place in Colombia.

The therapy will attempt to repair peoples telomeres, the caps on the end of our chromosomes that shorten as people get older. Its long been thought that they play a role in aging, and efforts to extend telomeres in mice have shown that it can delay the signs of getting older and increase healthy lifespan, though its yet to be tested in humans.

Libellas therapy will use viruses to deliver a gene called TERT, which codes for an enzyme called telomerase that re-builds teleomeres, to the patients cells.

Experts told MIT Tech Review that the trial is unethical, poorly designed, and presents serious risks to participants, including the danger of activating dormant cancerous cells. But its also still unclear whether the trial will go ahead, because the company has made previous announcements before without following through.

Whether or not it does, though, medical treatments to head off the slow march towards death are likely to become increasingly common. A growing body of research suggests that aging is an entirely preventable condition and that there may be a variety of ways to treat it, from lifestyle changes to dramatic genetic interventions.

In 2017, scientists showed that using drugs to reprogram epigenetic markerschemical attachments responsible for regulating the genomein mice extended their lifespan by 30 percent. And in 2018, another team showed that using a combination of drugs to kill senescent cellszombie cells that leak harmful chemicals, damaging nearby tissuecould boost the longevity of mice by 36 percent.

Famous geneticist George Church has even launched a startup called Rejuvenate Bio that will use proprietary genetic treatments to prolong the lives of dogs, though he has admitted the ultimate goal is to extend its technology to humans. Last month Churchs group at Harvard also showed that using gene therapies to tackle three age-related diseases at once was effective in mice.

The first anti-aging treatments for people are already starting to appear as well. CEO of longevity company BioViva Elizabeth Parrish injected herself with a gene therapy similar to Libellas back in 2015, and the company has claimed it was successful in lengthening her telomeres, though results were never published.

Earlier this year a study on humans found that a cocktail of drugs could reset the epigenetic clock, epigenetic markers used to measure a persons biological age. The participants also showed signs of a rejuvenated immune system.

And more controversially, the FDA recently had to put out a public service announcement telling people to stop injecting blood plasma from younger people. The idea is built upon recent research that showed a rejuvenating effect in mice, but most experts say its far too early to apply it to humans.

Whether the FDA will be able to keep on top of this burgeoning and highly lucrative market remains to be seen, but given the potential side effects of many of these treatments, it should be a priority.

We also need to have a more in-depth conversation about what these longevity therapies mean for society. Assuming this new trial is effective, what does it mean if only those with $1m to spare get to extend their lives? If treating aging becomes trivial, how is that going to change the nature of our communities? These are questions that may become increasingly relevant in the coming decades.

Image Credit: Shutterstock.com

The rest is here:
A New Anti-Aging Therapy Is Starting Its First Human Trialand It Costs $1 Million - Singularity Hub

CAMBRIDGE, Mass., Dec. 17, 2019 (GLOBE NEWSWIRE) -- LogicBio Therapeutics Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients,today announced ithas entered into an exclusive license with Oregon Health & Science University (OHSU) to intellectual property rights owned by OHSU while also extending a sponsored research agreement (SRA) to explore methods for enhancing selective advantage of edited hepatocytes using pharmacological agents with the laboratory ofMarkusGrompe, M.D.,professor at OHSU. The initial phase of the research program provided proof-of-principle of enhanced selective advantage for cells edited by GeneRide in pilot murine experiments. This extension phase will focus on translating the enhancement strategy to non-human primates, a critical step before clinical translation to future GeneRide candidates and other technologies. GeneRide is LogicBios proprietarypromoterless, nuclease-free genome editing technology,whichis designed to provide a stable therapeutic effect by harnessing homologous recombination to precisely integrate corrective genes into a patients genome and leveraging endogenous promoters to drive gene expression.

LogicBiois currently working primarily in disorders where patients can benefit substantially even when only a modest percentage of their cells are modified and begin expressing the corrective transgene introduced by GeneRide. The Company has found, however, that in some genetic contexts, integrating the transgene gives hepatocytes a naturally-occurring selective advantage over cells that have not been modified. Over time, the percentage of modified cells expressing that transgene rises, potentially leading to more robust patient benefits. This wasobserved inan experimentin which a murine GeneRide construct was introduced into mice with and without a functioning copy of theMutgene (deficient in the pediatric disease methylmalonic acidemia) in the liver. The initial GeneRide integration frequency was less than 1% in both sets of mice. Over time, this percentage remained stable in heterozygous mice that naturally expressMutin the liver (Mut+/- in liver). However, the share of cells expressing Mut increased to approximately 25% over more than a year in the mice genetically deficient in liverMut(Mut-/- in liver). This selective advantage could be attributed to improvements in mitochondrial function as a result ofMutexpression and restoration of the deficient essential metabolic pathway.These data were presented at the 2019 American Society of Gene & Cell Therapy Annual Meeting and can be found on the LogicBio website at the following link: https://investor.logicbio.com/events-and-presentations/presentations.

The goal of the expanded SRA with OHSUis to refine the pharmacological approachto providing a selective advantage to gene modified cells even when the transgene does not naturally confer a selection advantage at the cellular level. One such method involves adding an element to a GeneRide construct that gives cells incorporating that element a selective advantage when patients are treated with an external approved pharmacological agent.This research could enable expansion of the GeneRide platform to address genetic disorders in which clinical benefit emerges only after a higher percentage of cells are modified and begin expressing the corrective transgene.

We are excited to explore novel methods for enriching the number of cells expressing the therapeutic gene, said Dr. Grompe. Such methods could improve the likelihood that patients derive long-term therapeutic benefit from a single treatment. They could also expand the range of serious genetic disorders we can address with GeneRide.

Dr. Grompes lab studies monogenic disorders, particularly metabolic liver diseases affecting children. He has focused extensively on the use of in vivo selection to enhance cell and gene therapies. Dr. Grompe received the E. Mead Johnson Award for research excellence from the Society for Pediatric Research in 2002. He retains an active clinical practice, focused on metabolic disease.

About LogicBio TherapeuticsLogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free andpromoterlessapproach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

For more information, please visitwww.logicbio.com.

Forward Looking Statements This press release contains forward-looking statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on April 1, 2019 with the SEC, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Stephanie SimonTen Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

Read more:
LogicBio Therapeutics Extends Sponsored Research Agreement with Oregon Health & Science University to Explore Translation of...

One of the best things about being on the University of WisconsinMadison campus is the opportunity to learn from its passionate scientists and students. Covering the research stories of the institution keeps us busy, but it also provides the opportunity to have some fun (but dont tell the bosses!). While we believe that every story we do is important, here are some of the ones that taught us the most in 2019.

Chris Barncard, Eric Hamilton and Kelly April Tyrrell, Research Communications

Earths last magnetic field reversal took far longer than once thought

Some scientists think were living through the beginning of the next reversal of Earths magnetic field, which would affect our heavily electronic world in bizarre ways. Thankfully, new research from geoscientist Brad Singer found evidence that the last time Earths field reversed, it took about 22,000 years to complete. Thats several times longer than researchers previously thought and means that humanity would likely have generations to adapt to the next lengthy period of magnetic instability.

Genes behind lager yeasts cold- and sugar-loving success revealed

Lager beer is cold, crisp, dry and worth hundreds of billions of dollars. Genetics professor Chris Todd Hittinger and his lab recently discovered that lager yeast tolerates the cold because it inherited the power-generating portion of the cell from its cold-loving ancestor. And they figured out how this hybrid could have evolved the ability to digest all the sugars in wort to ferment a dry, crisp beer. Together, these two traits helped lager yeast and beer take over the world.

Memorial Union steam whistle sets rhythm of summer evenings and saves lives

To Terrace-goers, the steam whistle on Helen C. White Hall might just seem like a quaint way to herald another sunset. But to university and public boaters alike, its true mission is clear: keeping everyone on Lake Mendota safe enough to head out another day. UWPD Lake Rescue and Safety and the Hoofer Sailing Club collaborate to train sailors, monitor lake conditions and blow the whistle when approaching storms threaten boaters, ultimately saving lives. Thanks, whistle.

Reddit competes to visualize Madisons prized Lake Mendota ice data

For 166 years, university and state scientists have carefully tracked Lake Mendotas annual freeze and thaw. That trove of data was just what Reddit was looking for. The DataIsBeautiful subreddit competed to find the best way to visualize this long, icy record. As each graphic, animation and chart revealed, Lake Mendota has lost about a months worth of ice since record-keeping started as the result of a warming climate. That adds up to big changes for Madisons ice fishers and cross-country skiers and to the watery world beneath their feet.

Ancient poop helps show climate change contributed to fall of Cahokia

Nearly 1,000 years ago, the ancient city of Cahokia, near present-day St. Louis, was the most sophisticated prehistoric settlement north of Mexico. But then, the Mississippi River began to flood, and Cahokias population rapidly declined. Researchers at UWMadison and California State University, Long Beach found evidence using remnants of human poop and layers of sediment deposited in a nearby lake to suggest climate change contributed to Cahokias fall. Cultures can be very resilient in the face of climate change but resilience doesnt necessarily mean there is no change, says UWMadison anthropology professor Sissel Schroeder.

Fear of more dangerous second Zika, dengue infections unfounded in monkeys

Zika virus, already connected to heartbreaking consequences for newborns in recent epidemics in the Americas, was cause for additional concern among public health officials because it is so closely related to dengue virus which is dangerous enough in an initial infection, but can be even more life-threatening during a second infection. This year, UWMadisons Zika virus researchers allayed some fears that the viruses would make each other more dangerous by showing that, in monkeys, an earlier infection with one of the viral cousins does not make a later infection with the other more virulent.

Study confirms horseshoe crabs are really relatives of spiders, scorpions

When is a crab not actually crab? When its a horseshoe crab. These hard-shelled, blue-blooded creatures belong, it turns out, to the spiders and the scorpions. UWMadison evolutionary biologists Prashant Sharma and Jess Ballesteros subjected the genomes of horseshoes crabs, which have existed on Earth for 450 million years, to intense computational scrutiny and found they should live on the arachnid family tree. They are part of a lineage that makes them among the most successful animals on the planet.

Tiny capsules packed with gene-editing tools offer alternative to viral delivery of gene therapy

Gene therapy using chemical tools to edit a patients genetic code for inherited diseases, some cancers, and even stubborn viral infections is most often delivered using engineered viruses, but those viruses are hard to steer to specific cells within the body and can cause trouble by exciting the immune system. UWMadison biomedical engineers have created an alternative to viral delivery, loading tiny synthetic capsules with gene-editing tools and coating the shell with molecules that help them zero in on their therapeutic targets.

With fire, warming and drought, Yellowstone forests could be grassland by mid-century

Yellowstone National Park may be at a tipping point. Its forests are adapted for periodic fires, but large wildfires that once blazed the landscape every 100-to-300 years are now sweeping through much more frequently as the climate warms and drought conditions increase. UWMadison ecologist Monica Turner has studied the forests of Yellowstone for three decades and her work suggests forests may not be able to regenerate quickly enough. By the middle of this century, some of them may become grassland.

Lessons of conventional imaging let scientists see around corners

By playing the angles, UWMadison researchers are developing cameras that can see around corners. Led by Andreas Velten, a professor of biostatistics and medical informatics, the scientists can bounce thousands of pulses of laser light off a wall or other surface into an unseen space and collect the scattered photons that ricochet back to their sensors. Using math to reconstruct the path of the returning light, they can piece together a picture of the hidden space as seen from the perspective of their reflecting surface.

Share via Facebook

Share via Twitter

Share via Linked In

Share via Email

See more here:
2019's most memorable research stories - University of Wisconsin-Madison

IOWA CITY A University of Iowa clinician and neuroscientist has received an $18 million grant to continue a decade-long study on how a potential treatment for Huntingtons disease may affect childrens brain development.

Dr. Peg Nopoulos, chairwoman of the University of Iowa department of psychiatry, was awarded a five-year grant by the National Institution of Neurological Disorders and Strokes, part of the U.S. Institutes of Health.

These are really high-in-the-sky questions, she said. Were just boots on the ground, trying to understand how this affects children at risk for Huntingtons disease.

Huntingtons disease is a fatal genetic disorder that causes the progressive breakdown of nerve cells, diminishing thinking skills and emotions and disrupting fine motor function. If a parent has Huntingtons disease, there is a 50 percent chance her or his child will develop the same disease.

There is no cure for Huntingtons, but an emerging gene therapy in clinical trials has presented promising findings for slowing its progression, Nopoulos said. The gene therapy has been given only to individuals who have Huntingtons disease in an attempt to slow the progressive breakdown.

Nopoulos said the next step is to give the therapy to individuals before the genetic disorder takes effect, to test whether it could prevent it altogether.

However, she said this possibility presents a key question as the gene that causes the disease also is important for the growth and development of an individuals brain

ARTICLE CONTINUES BELOW ADVERTISEMENT

Our study is really examining how this gene affects brain development, Nopoulos said. And in that context, were examining how we would deliver a gene therapy and how that would impact brain development.

We certainly dont want to prevent the disease only to cause problems for brain development.

The gene that causes Huntingtons, called HTT, is key for an area of the brain called the striatum, which helps control voluntary movement, among other functions. Through their study, Nopoulos and other researchers on the project hope to understand what consequences could result from the therapy on development.

If we were to give the gene modifying therapy too early, it could prevent maximum brain function, she said.

Nopoulos has been studying this question for 10 years at the UI and will continue that work under the newly funded project, called Children to Adult Neurodevelopment in Gene-Expanding Huntingtons disease.

The $18 million grant from the national institute is a renewal of the same study from the UI, allowing Nopoulos and other researchers to expand the scope and size of the original study by including five times as many participants from five sites across the United States.

In addition to the UI, those sites will include the Childrens Hospital of Philadelphia, Columbia University in New York, the University of California, Davis and the University of Texas in Houston.

All subjects in the study, who are between the ages of 6 and 30, have a parent with Huntingtons, which means they are 50 percent at risk for developing the genetic disorder sometime in their lifetime.

Nopoulos said the test subjects dont know if they carry the genetic marker for the disease.

ARTICLE CONTINUES BELOW ADVERTISEMENT

Weve been doing this for 10 years, and Im always amazed at how strong these kids are, Nopoulos said. The study gives them meaning and an opportunity to help gain knowledge about the disease their parent is going through.

A lot of these children really take on the altruism of wanting to give back to the community, even when they know theyre at risk themselves, she said.

Comments: (319) 368-8536; michaela.ramm@thegazette.com

See the original post:
University of Iowa researcher gets $18 million to study Huntington's disease - The Gazette