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Category Archives: Genetic Medicine

PFF-supported PRECISIONS Study Aims to Bring Personalized Treatment to IPF – Pulmonary Fibrosis News

The Pulmonary Fibrosis Foundation (PFF) announced a research initiative that aims to expand precision medicine strategies for diagnosing and treatingidiopathic pulmonary fibrosis (IPF).

The study, called PRECISIONS (for Prospective tReatmentEffiCacy inIPF uSIng genOtype forNacSelection), is being funded by a$22 milliongrant from the National Institutes of HealthandThree Lakes Partners, a philanthropic organization dedicated to pulmonary fibrosis care and research.

PRECISIONShas three primary goals.First, it aims to determine whether N-Acetyl-cysteine (NAC) is an effective treatment for individuals with IPF who have a particular variant of the geneTOLLIP, known to play a role in lung immunity.

NAC is an antioxidant, and it has been suggested in can lessen lung damage in IPF, although its efficacy is far from clear, with studies finding conflicting results.Previous researchsuggested that this may be because NAC is only effective in individuals with particular genotypes. Specifically, the variantTOLLIPrs3750920T/T, which is present in around a quarter of people with IPF, is associated with better clinical responses to NAC. PRECISIONS aims to directly test this association.

The second goal of PRECISIONS is to develop blood-based assays to help distinguish IPF from other lung diseases with similar symptoms. This will be accomplished by using unbiased analyses of transcriptomics (which genes are off and on in cells), proteomics (global analysis of proteins in the blood), and other large-scale molecular analyses, in order to identify molecular signatures that are linked with IPF. Such biomarkers could help in predicting an individuals disease course and response to therapy.

PRECISIONSthird and final goal is to identify genetic variants that influence an individuals risk of developing IPF, which may prove useful in monitoring people at a high risk of the disease.

This innovative study highlights the value of a partnership between a broad range of investigators, the PFF, a philanthropic organization (Three Lakes Partners), and the National Heart, Lung, and Blood Institute (NHLBI). Most importantly, it seeks to provide patients with interstitial lung disease (ILD) access to personalized medicine in which the right medication is used for the right patient,Fernando Martinez, MD, one of the researchers leading the project, said ina press release.

The project will rely on the PFF Patient Registry and Biorepository, which have collected clinical data and biological samples from over 2,000 pulmonary fibrosis patients.

The PFF Patient Registry will serve as an invaluable tool to facilitate more efficient enrollment into the NAC pragmatic trial, and to further define the genetic risk factors influencing the development and potential progression of the disease, saidGregory Cosgrove, MD, chief medical officer at PFF. It will hopefully allow for the identification of important biomarkers to assist in the diagnosis and care of patients with PF.

Added Imre North, MD, a co-principal investigator on the project:PRECISIONS has the potential to really change the scientific landscape over how we view IPF and ILDs by providing molecular classifications while determining if a pharmacogenetically driven treatment can change outcomes.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

Total Posts: 110

Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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How Gene Therapy Is Evolving to Tackle Complex… –

Gene therapy has traditionally been applied to well-understood diseases where a single genetic mutation was to blame. A new generation of technology is expanding the potential of gene therapy to treat conditions that were previously unreachable.

Since the first gene therapy clinical trials in the 1990s, the technology has made its way into the market for conditions ranging from blindness to cancer.

Gene therapy has the potential to fix any genetic mutation causing disease by inserting a new copy of the faulty gene. However, its reach has historically been limited.

Weve been constrained with the things we can do with gene therapy, said Dmitry Kuzmin, Managing Partner at 4BIO Capital, a London-based VC that specifically invests in advanced therapies. If you look across the successes in gene therapy in the last five years, most of these were in diseases that are pretty straightforward from the engineering perspective.

Technical limitations have meant that gene therapy has been restricted to rare diseases caused by a single genetic mutation, as well as to certain areas of the body, such as the eye and the liver.

According to Kuzmin, there have been so far three generations of gene therapy technology. Generation one would be classic single-gene replacement, such as Luxturna, a gene therapy to fix a specific genetic mutation causing blindness. Generation two would consist of using gene therapy to introduce new functions. An example is Kymriah, where immune cells are equipped with a molecule that helps them hunt down cancer cells.

The third generation is the one that could hold the key to unlocking the full potential of gene therapy. It englobes several technologies that can be used to introduce a new drug target into the patient, making it possible to turn the therapy on and off as well as to tune its intensity.

As the first two generations get optimized and the third generation enters the clinic, we are now expanding our reach into areas that have been previously rather inaccessible, Kuzmin told me. One of them is the brain.

Treating the brain has long been a huge challenge for medicine. Take epilepsy, for example.

Epilepsy affects 1% of the whole population and about 30% of people with seizures of epilepsy continue to have seizures despite medication, said Dimitry Kullmann, Professor at University College London. Theres a paradox. We have a good understanding of the mechanisms behind epilepsy, but were unable to suppress seizures in a significant proportion of people with epilepsy.

The reason is that the molecules that we use for drugs dont target the epileptic zone of the brain; they bathe the entire body with medication, Kullmann told me. These drugs dont differentiate between neurons and synapses that derive the seizures, and those parts of the brain that are responsible for memory, sensory functions, motor functions and balance.

Gene therapy could provide a solution for this problem. Kullmanns group has been researching this approach for years and is now getting ready to start the first clinical trial in humans within a year.

A gene therapy can be directly injected in the area of the brain causing seizures. Furthermore, using DNA sequences called promoters, it is possible to restrict the effect of gene therapy to specific neurons within that area. In the case of epilepsy, gene therapy can be used to decrease the activity of only excitatory neurons, which cause epileptic seizures when they are overactive.

Another approach that Kullmans group is testing is chemogenetics. The idea here is to use gene therapy to put a specific receptor into the neurons, explained Kullmann. This receptor is designed to respond to a drug that, when given to the patient, decreases the activity of the neuron to suppress seizures.

The advantage is that you can switch on and off the therapeutic effect on demand by just giving, or not giving the drug, Kullmann said. This approach can thus make gene therapy more precise, being able to tune it to the specific needs of each patient. In addition, it reduces the big challenge of getting the dose right in a one-off treatment.

Ultimately, this technology could allow scientists to target a wide range of conditions that come under the umbrella of epilepsy, rather than just a specific form of the condition caused by a genetic mutation.

The approach could be extended to other conditions involving the brain, such as Parkinsons, ALS and pain. However, this kind of research is still at an early stage and it will take a while until its potential is proven in humans.

Blindness has been a major target of gene therapy because of the fact that the eye is an ideal target for this technology. The activity of the immune system is suppressed in the eye, minimizing the chances of rejection. In addition, unlike other cells in the body, those involved in vision are not renewed over time, being able to retain the injected DNA for years.

However, there are hundreds of genetic mutations that can cause blindness. With the classical gene therapy approach, a different therapy would have to be developed from scratch for each mutation. While some companies are doing just this for the most common mutations causing blindness, many other less frequent mutations are being left behind.

Others are turning to new generations of gene therapy technology. We figured out that it would be very, very difficult to use the classical gene therapy approach in each individual mutation, said Bernard Gilly, CEO of GenSight, a Parisian biotech developing gene therapies for blindness.

While the companys leading programs follow this classical approach, the company has also started clinical trials using a technology called optogenetics. Following a similar principle to gene therapy, optogenetics consists of introducing a protein that reacts to light into a cell.

GenSight is using optogenetics to develop a single therapy for the treatment of retinitis pigmentosa. This genetic condition can be caused by mutations in any of over 200 genes and results in progressive vision loss in children due to the degeneration of photoreceptor cells that perceive light and send signals to the brain.

With optogenetics, it would be possible to transfer the lost photoreceptor function to the cells in the retina that are responsible for relaying visual information to the brain. Using specialized goggles, the images captured by a camera are transformed into light patterns that stimulate these cells in the precise way needed for the brain to form images.

The company is currently testing this approach in clinical trials. We believe that this approach will allow us to restore vision in those patients who became blind because of retinitis pigmentosa, Gilly told me.

Optogenetics would not work a miracle, but it might be able to give people back the ability to navigate an unknown environment with a certain level of autonomy. Recognizing faces is a more challenging goal; although reading is not yet on the horizon, according to Gilly.

Still, the potential of optogenetics to address multiple genetic mutations with a single treatment might be revolutionary. As long as the neurons responsible for sending light signals to the brain are intact, this approach could be extended to other forms of blindness. In addition, conditions affecting the brain such as epilepsy, Parkinsons or ALS could be treated with this approach by introducing an implant to shine light on the target neurons.

However, approaches applying optogenetics to the brain are still further down the line. While optogenetics technology has been around for over 20 years, its application in humans is still very limited and in the early stages of research.

Chemogenetics and optogenetics are just two out of a wave of new technologies addressing the historical limitations of gene therapy. Other approaches are in development, such as using thermogenetics, which consists of introducing proteins that are activated by the heat created by infrared light.

With a growing range of tools available, it is becoming easier than ever for scientists to develop gene therapies that can address the specific challenges of different conditions affecting areas of the body. Traditionally, locations such as the heart, the lungs or the pancreas have been particularly difficult to target with gene therapy. That might soon stop being the case.

As we go forward, were interested in taking gene therapy out of this little box and trying to use all the knowledge we have to benefit patients in larger indications, said Kuzmin.

As gene therapy expands into more mainstream conditions, it could take precision medicine to a whole new level and help address the big variability that is often seen across patients with the same diagnosis.

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Smoking may increase risk of mental health problems study – The Guardian

Smoking tobacco cigarettes could increase the risk of mental health problems such as depression and schizophrenia, research suggests.

It has long been known that smoking is more common among people with mental health conditions. However, it has been unclear whether smoking could be a factor in causing such problems or is simply a form of self-medication among those already living with poor mental health.

Scientists say they have now unpicked the issue. What we found was that there was evidence of causal effects in both directions, said Dr Robyn Wootton, the first author of the research, from the University of Bristol.

Wootton said that while the serious physical consequences of smoking were already known, the new research underlined the importance of preventing people from starting the habit and helping smokers to quit to protect their mental health whether or not they have existing mental health problems.

Of course, if [smoking] is also making the risk of mental illness worse then we should be helping individuals who have existing mental health problems to stop as well, she said.

Writing in the journal Psychological Medicine, Wootton and colleagues report how they compared the risk of developing depression or schizophrenia among people with and without a genetic predisposition to smoking cigarettes. As such genetic variants are randomly distributed across the population and are not changed by factors such as alcohol consumption, income, exercise or other health issues the approach is a type of natural experiment that reduces the chances of any link being down to other factors.

The team focused on 378 genetic variants that have previously been linked to whether people start smoking, as well as 126 genetic variants the team found were linked to a higher score for lifetime smoking a measure encompassing how heavily people smoked, for how long, and if they quit.

Wootton and colleagues then used two separate genetic databases, one including thousands of individuals with schizophrenia and the other including thousands of individuals with major depression, to explore whether the risk of having such conditions was linked to the genetic variants for smoking.

The results reveal that both starting smoking and higher levels of smoking are linked to a greater risk of both depression and schizophrenia. As an example, an individual who smoked 20 cigarettes a day for 15 years but then did not smoke for 17 years had more than twice the odds of developing schizophrenia and almost twice the odds of developing depression than someone who had never smoked.

The team also looked to see whether people with a genetic predisposition to depression or schizophrenia smoked more. While they did indeed find such an effect, it was weaker than for the opposite direction.

However, the study has limitations, including that it focused on people of European ancestry.

Wootton said it was necessary to explore exactly how smoking might increase the risk of schizophrenia and depression, but one possibility was that nicotine influences pathways in the brain linked to mental health problems. That could be important, she added, since nicotine is also found in electronic cigarettes.

Cannabis use might also help to explain the findings, since high-strength cannabis has previously been suggested to increase the risk of mental health problems, and those who smoke have a greater risk of cannabis dependency.

Dr Ian Hamilton, an expert in addiction and mental health from the University of York, said: While the physical harms of smoking are well known, this research points to the mental health risks of using tobacco. This risk should be communicated widely but particularly to school-age children who might be tempted to try smoking.

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Smoking may increase risk of mental health problems study - The Guardian

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GeneSight Psychotropic Test Improved Clinical Outcomes for People with Depression Who Were Taking Medications with Gene-Drug Interactions -…

Figure 1

GeneSight Provided Significant Improvements in All Patient Outcomes

Myriad Genetics, Inc.

Figure 2

GeneSight Clinical Benefits Were Durable and Improved Over Time

Myriad Genetics, Inc.

Figure 3

GeneSight Improved Outcomes for Patients Switching Medications

Myriad Genetics, Inc.

SALT LAKE CITY, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN, Myriad or the Company), a global leader in molecular diagnostics and precision medicine, today announced that a new analysis of the GUIDED1 clinical trial was published online in The Journal of Clinical Psychiatry. The key finding is that the GeneSight Psychotropic test improved clinical outcomes remission, response and symptoms in patients taking medications with gene-drug interactions. Improvement in all three endpoints was statistically significant.

Treatment decisions for people with major depressive disorder are challenging, particularly for patients who have already not benefitted from one or more medications, said Michael E. Thase M.D., lead author and professor of psychiatry, Perelman School of Medicine, University of Pennsylvania. In some cases, nonresponse or intolerance to a standard antidepressant can result from a gene-drug interaction. When thats the case, you want to select a different medication that doesnt have that interaction.

Patients enrolled in the GUIDED clinical trial were diagnosed with major depressive disorder (MDD) and had failed at least one psychotropic medication. Patients were randomized to treatment as usual (TAU) or the guided-care arm in which clinicians had access to the GeneSight test report to inform their medication decisions. The analysis published in the Journal of Clinical Psychiatry today evaluated a total of 787 people from the GUIDED clinical trial who were taking medications with gene-drug interactions at baseline as identified by the GeneSight test.

The results from the analysis showed that people in the GeneSight arm (n=357) achieved statistically significant improvements in all clinical outcomes compared to TAU (n=430) at week 8 (Figure 1).

To view Figure 1: GeneSight Provided Significant Improvements in All Patient Outcomes, please visit the following link:

Importantly, the improvements in all clinical outcomes were durable and continued throughout the six-month follow up period. Remission increased 82 percent, response rates increased 64 percent and symptom improvement increased 56 percent from week 8 to week 24 (Figure 2).

To view Figure 2. GeneSight Clinical Benefits Were Durable and Improved Over Time, please visit the following link:

Patient outcomes in the GeneSight-guided arm were superior to treatment-as-usual. Additionally, patients continued to get better over time and the rate of remission, which is the goal of treatment, nearly doubled from week 8 to week 24, said Dr. Thase. Too often patients with difficult-to-treat depression relapse, and this data shows that the patient benefits in the GeneSight-guided arm were durable.

The new analysis also assessed how the GeneSight test impacted outcomes for patients who were taking medications with gene-drug interactions at baseline and who switched medications, which was defined as dropping at least one medication and adding at least one different medication. Among people who switched, all clinical outcomes were statistically significantly better for those in the GeneSight arm (n=235) compared to TAU (n=225) at week 8, underscoring the value of using combinatorial pharmacogenomic information to help inform treatment decisions (Figure 3).

To viewFigure 3. GeneSight Improved Outcomes for Patients Switching Medications, please visit the following link:

The GeneSight test identified patients taking medications with gene-drug interactions, and physicians were able to use this information to switch patients to medications with fewer gene-drug interactions and improve their outcomes, said Michael R. Jablonski, Ph.D., vice president of Medical Affairs, Myriad Neuroscience. This is an important first step in the journey towards making precision medicine a reality for people suffering from depression.

About GeneSight PsychotropicGeneSight Psychotropic is a pharmacogenomic test that analyzes clinically important variations in DNA. The results of the test can inform doctors about genes that may impact how their patients metabolize or respond to depression medications.

About Myriad GeneticsMyriad Genetics Inc., is a leading precision medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on five critical success factors: building upon a solid hereditary cancer foundation, growing new product volume, expanding reimbursement coverage for new products, increasing RNA kit revenue internationally and improving profitability with Elevate 2020. For more information on how Myriad is making a difference, please visit the Company's website:

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice HRD, EndoPredict, Vectra, GeneSight, riskScore, Prolaris, ForeSight and Prequel are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to combinatorial pharmacogenomic testing augmenting physician decision-making and helping people get well; making precision medicine a reality for people suffering from depression; and the Company's strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decision in the lawsuit brought against us by the Association for Molecular Pathology et al; risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

1 Greden JF, Parikh SV, Rothschild AJ, et al. Impact of pharmacogenomics on clinical outcomes in major depressive disorder in the GUIDED trial: A large, patient- and rater-blinded, randomized, controlled study. J Psychiatr Res. 2019; 111:59-67.

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Shape Therapeutics, Inc. Raises $35.5M Series A Financing, Led by NEA and Announces the Formation of a World-Class Scientific Advisory Board, to…

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTx), a development-stage biotechnology company leading the field of RNA-editing gene therapy, announces $35.5M in Series A financing, led by New Enterprise Associates (NEA), with additional participation from CureDuchenne Ventures. The new capital will enable the company to extend its growing portfolio of intellectual property, recruit and hire top scientific talent and advance its groundbreaking RNA and protein targeting platforms focused on curing human diseases.

These platforms include the proprietary ShapeTx RNAfix technology that enables direct in vivo targeting and modification of RNA by leveraging proteins such as Adenosine Deaminases Acting on RNA (ADARs), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix platform differentiates from other contemporary genome engineering technologies by engaging natural human cellular machinery to modify RNA.

ShapeTx was founded on the work of Dr. Prashant Mali, Assistant Professor of Bioengineering at UCSD, who during his postdoctoral fellowship in the George Church laboratory at Harvard Medical School pioneered the use of CRISPR in human cells. ShapeTx RNAfix platform is built upon his lab's most recent work demonstrating in vivo use of guide RNAs to recruit native ADARs and to fix mutations in multiple rare genetic disease mouse models.

Our technology can correct mutations or target specific genes in neurodegenerative, oncology, metabolic and rare genetic disorders by hijacking naturally occurring proteins such as ADARs present in our cells using just a short guide RNA. Our proprietary new platform avoids the risk of in vivo immunogenicity and permanent off-target damages commonly associated with CRISPR-based approaches, explained Francois Vigneault, Ph.D., President and CEO, who was previously VP of Research at Juno Therapeutics after a successful co-acquisition of AbVitro, Inc. by Juno and Celgene.

Ed Mathers, Partner at NEA and Board member at ShapeTx, said, One rarely comes across a proprietary technology platform with such transformative potential led by a focused and data-driven scientific group with a successful track record in pre-clinical and clinical development. The team has shown us an exciting demonstration of the technology in multiple in vivo models, alongside one of the strongest IP estates we have seen in the field. NEA looks forward to backing the company in future rounds as they move the technology toward the clinic.

While the ShapeTx platform will be enabling for many other genetic diseases, Dr. Malis in vivo proof of concept in Duchenne Muscular Dystrophy was quite exciting and could potentially lead to a cure for families suffering from such a debilitating disorder, said Debra Miller, CEO and Founder of CureDuchenne and CureDuchenne Ventures.

The ShapeTx Series A financing coincides with the formation of a world-class Scientific Advisory Board comprised of foremost global experts in genomics, bioengineering, and gene editing, including George Church Ph.D., James Collins Ph.D., and Don Cleveland Ph.D. The scientific advisory board will serve as strategic advisors and ensure that the research and development of its platforms meet the highest standards of scientific merit.

Prashant and Francois are some of the most innovative and brilliant individuals that have come through my lab over the years, and it will be impressive to see these two disrupt the field of gene therapy with this paradigm-shifting technology, said Dr. George Church, Professor in Genetics at Harvard Medical School and member of the ShapeTx Scientific Advisory board.

Shape Therapeutics Scientific Advisory Board Members:

George Church, Ph.D.

George Church Ph.D., world-famous geneticist, molecular engineer, and chemist. He developed the methods used for the first genome sequence & million-fold cost reductions since, as well as pioneered many of the CRISPR advances in genome editing. He is currently a Professor of Genetics at Harvard Medical School and Professor of Health Sciences and Technology at Harvard and the Massachusetts Institute of Technology (MIT). He is Director of the U.S. Department of Energy Technology Center and Director of the National Institutes of Health Center of Excellence in Genomic Science. He has received numerous awards, including the 2011 Bower Award and Prize for Achievement in Science from the Franklin Institute and election to the National Academy of Sciences and Engineering.

James Collins, Ph.D.

James Collins Ph.D., is one of the pioneers of the field of synthetic biology and has made multiple synthetic biology and bioengineering breakthroughs in biotechnology and biomedicine. He serves as the Termeer Professor of Medical Engineering & Science and Professor of Biological Engineering at MIT, as well as a member of the Harvard-MIT Health Sciences & Technology Faculty, and core member of the Wyss Institute. His many awards include a Rhodes Scholarship, a MacArthur Genius Award, a National Institutes of Health Directors Pioneer Award. Jim is also an elected member of the National Academy of Sciences, the National Academy of Engineering, the National Academy of Medicine, the American Academy of Arts & Sciences, as well as a charter fellow of the National Academy of Inventors.

Don Cleveland Ph.D.

Don Cleveland Ph.D. is an award-winning inventor and pioneer in the field of Antisense Oligonucleotide (ASO) and their uses in gene therapy. He was recently awarded the Breakthrough Prize in Life Sciences for his work on the pathogenesis of disease and ASO-mediated treatment approaches in ALS and Huntingtons disease. Don is currently Professor of Medicine and Department Chair of Cellular and Molecular Medicine and Neurosciences at the University of California at San Diego, and Head, Laboratory for Cell Biology at the San Diego branch of Ludwig Cancer Research. He has made pioneering discoveries on the mechanisms of chromosome movement and cell-cycle control during normal cellular division, as well as the principles of neuronal cell development and the relationship to defects that contribute to inherited neurodegenerative disease.

About Shape Therapeutics, Inc.

Shape Therapeutics, Inc. is creating the worlds leading RNA and protein targeting platforms focused on the cure of human diseases. These include developing precision RNA editing through proteins such as ADAR (Adenosine Deaminase Acting on RNA), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix technology allows for the editing of RNA using natural human cellular machinery, limiting the risk associated with immunogenicity, cellular toxicity, or off-target DNA editing. The teams founders include Prashant Mali, Ph.D., Francois Vigneault, Ph.D., and John Suliman. ShapeTx is headquartered in Seattle, Washington, with a satellite site opening in Cambridge, Massachusetts. For additional information, visit

About NEA

New Enterprise Associates, Inc. (NEA) is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors, and geographies. With more than $20 billion in cumulative committed capital since the firm's founding in 1978, NEA invests in technology and healthcare companies at all stages in a company's lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 225 portfolio company IPOs and more than 375 acquisitions. For additional information, visit

About CureDuchenne Ventures

CureDuchenne Ventures supports Duchenne research by using philanthropic donations to encourage the development of new Duchenne drugs. Through an impact financing model, we can provide equity or royalty financing to biotech and pharmaceutical companies. CureDuchennes portfolio includes 16 wide-ranging projects with several successful exits. Investments from CureDuchenne Ventures have successfully de-risked and leveraged more than $2.3 billion in follow-on financing from venture capital, biotech, and pharmaceutical companies to fund emerging projects to find treatments for Duchenne. For additional information, visit

Shape Therapeutics, Inc. Raises $35.5M Series A Financing, Led by NEA and Announces the Formation of a World-Class Scientific Advisory Board, to...

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Cannabis to be given to patients to test impact on seven conditions – Sky News

Thousands of patients are to be given cannabis in the first large-scale study of the drug's clinical effect.

Medical cannabis, which is grown to a precise grade of active ingredient, was legalised in the UK a year ago.

But only a handful of patients have since been prescribed the drug on the NHS because of what medical authorities have called a "paucity of evidence" that it works and is safe.

The only option for patients is to either source cannabis illegally, and risk prosecution, or pay for a private prescription of the drug

The new study, called Project Twenty21, will subsidise cannabis for 20,000 patients to test its impact on seven conditions: chronic pain, multiple sclerosis, epilepsy, post-traumatic stress disorder, Tourette's syndrome, anxiety disorder and substance abuse.

Professor David Nutt of the organisation Drug Science, which is running the study, told Sky News: "I believe cannabis is going to be the most important innovation in medicine for the rest of my life.

"Cannabis medicines can be life-saving in disorders like severe childhood epilepsy.

"There are children who have died in this country in the last couple of years because they haven't had access to cannabis.

"It's outrageous, it's unnecessary and we want to rectify it."

Lucy Stafford used to suffer severe chronic pain from the genetic connective tissue disorder Ehlers Danlos syndrome, which meant she could dislocated her joints simply by rolling over in bed. She was treated with opioid drugs, but they had little effect and had serious side effects.

Since paying privately for medical cannabis, at a cost of 800 a month, her pain has reduced so much that she has stopped other treatments and started university.

She told Sky News: "Now I can get up in in the morning and even if I'm in the most unbearable amount of pain and feel like passing out and being sick I can take my medication and be able to function and focus and live my life.

"If other medications were effective for us we wouldn't need this.

"But the whole point is that everything that is currently being offered, such as opiates, diazepam and other horrible medications, just do not manage the conditions that we are living with.

"Medical cannabis does."

But the Project Twenty21 study will have to overcome medical scepticism. The clinical watchdog NICE said cannabis should not be prescribed for a range of medical conditions, including chronic pain.

The new study has, however, been backed by the leading body of psychiatrists.

Professor Wendy Burn, President of the Royal College of Psychiatrists, said: "The College welcomes this pilot project which it hopes will make an important contribution towards addressing the paucity of evidence for the use of cannabis-based medicinal products.

"We hope that this pilot, along with other research such as more much-needed randomised control trials, will continue to build the evidence."

Medical cannabis was legalised last November following a series of high profile cases, including eight-year-old Alfie Dingley, whose mother claimed the drug eased his severe epilepsy.

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