In 2017, Sangamo is conducting a Phase 1/2 clinical trial evaluating SB-913 as an in vivo genome editing treatment for MPS II. Sangamo is also conducting Phase 1/2 studies this year evaluating in vivo genome editing treatments SB-318 for MPS I, another rare lysosomal storage disorder, and SB-FIX for hemophilia B, a rare blood disease. Data from these studies and from a clinical trial for a fourth lead program, SB-525, a gene therapy approach for hemophilia A, are expected in late 2017 or early 2018.

Sangamo's In Vivo Genome Editing Approach Sangamo's ZFN-mediated in vivo genome editing approach makes use of the endogenous albumin gene locus, a highly expressing and liver-specific site that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein. The ability to permanently integrate the therapeutic gene in a highly specific, targeted fashion significantly differentiates Sangamo's in vivo genome editing approach from conventional AAV cDNA gene therapy. Ultimately, the target population for these programs will include pediatric patients, and it will be important in this population to be able to produce stable levels of therapeutic protein for the lifetime of the patient.

About Sangamo Therapeutics Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is advancing Phase 1/2 clinical programs in hemophilia A and hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has a strategic collaboration with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at http://www.sangamo.com.

Forward Looking Statements This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation references relating to research and development of therapeutic applications of Sangamo's gene therapy and ZFP technology platforms, the potential of Sangamo's technology to treat hemophilia and lysosomal storage disorders, the expected timing of these clinical trials and the release of data from these trials, the impact of Sangamo's clinical trials on the field of genetic medicine and the benefit of orphan drug status. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to substantial dependence on the clinical success of lead therapeutic programs, the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo Therapeutics, Inc. assumes no obligation to update the forward-looking information contained in this press release.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-receives-orphan-drug-designation-from-the-fda-for-sb-913-genome-editing-treatment-for-mps-ii-300415719.html

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During his address to Congress last night, President Donald Trump introduced Megan Crowley, a 20-year-old woman with a rare disease.

Crowley, who has a neuromuscular disorder known as Pompe disease, credits her survival to her father's small biotechnology company for developing and producing a therapy for the rare genetic mutation.

During the speech, the President suggested that Crowleys story highlighted the need to scale back regulations and to streamline the processes of the Food and Drug Administration. While Crowley's doctor welcomed the spotlight shown on Pompe disease and on rare diseases, in general, others cautioned that rushing experimental treatments through the Food and Drug Administration could have disastrous results.

Related: Speed up drug approvals? The FDA already did

The big problem isnt with the FDAs procedures, but rather a shortage of funds and the small numbers of people in whom scientists can study the ailments, researcher Dr. Puneet Opal told TODAY.

FDA drug approval procedures are intended to prevent patient deaths from experimental treatments, said Opal, a professor of neurology at Northwestern Universitys Feinberg School of Medicine.

"You do need the FDA to be the police dog," said Opal.

Crowleys physician, Dr. Priya Sunil Kishnani, division chief of medical genetics at the Duke University Medical Center, was excited to have rare diseases given such high-profile attention.

We diagnosed Megan at Duke in 1998 when she was just 15 months old. Its been an amazing journey to see how she has not just survived but is thriving despite her limitations, Kishnani, who is also professor of pediatrics and division chief of medical genetics, told TODAY.

Pompe disease occurs in an estimated 1 in 12,000 to 20,000 babies and people with an adult-onset form, according to Duke University experts. Crowley's fathers struggle to find a cure for the disease was profiled in the book The Cure: How a Father Raised $100 Million and Bucked the Medical Establishment in a Quest to Save His Children and also inspired a 2010 movie called Extraordinary Measures.

Megan Crowley listens as President Donald Trump addresses Congress.

Related: Parents race to save girl with rare, deadly disease

For those not familiar with Pompe, here are five things to know:

That means that a child must inherit two copies one from each parent to be affected by it. Children born with just one copy of the mutated gene are healthy and generally are completely unaware they have it.

When someone has both copies of the mutated gene, the body lacks enough of an enzyme that breaks down the starch glycogen into glucose, a form of sugar that fuels the muscles. The result is a damaging overabundance of glycogen.

Because the heart is also a muscle, damage to it is what generally kills patients even before there is respiratory distress, Opal said.

In babies, the earliest symptoms are not meeting motor milestones, Kishnani said. Babies might not hold their heads up or start rolling over at the expected age. They may not have the same strength as other babies of similar age.

"When you pick them up you feel like theyre almost slipping through your fingers," said Kishnani.

Respiratory symptoms or pneumonia might reveal a massively enlarged heart.

The severity of the symptoms and when they appear depend on the exact type of mutation. There are children who show up with worse symptoms than Crowley and others who show up with milder ones, Kishnani said.

The treatment isnt a cure for the disease, but it is lifesaving. Crowley is confined to a wheelchair and must use a ventilator to breathe.

These individuals do have a life," said Kishnani, talking about Megan's spunk and passion.

"She has life goals. People should not just look past her ... and think there are not going to be any cognitive abilities.

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What is Pompe disease? 5 things to know about the rare gene mutation - Today.com

ValueWalk

Scientists Favor Gene Editing, But Only For 'Fixing Diseases' ValueWalk An international body of scientific experts has stated, with caution, that gene editing technologies should be allowed for treating diseases or disabilities. The US National Academy of Sciences and the National Academy of Medicine said in a 200-page ... Gene Editing Could Make You SmarterFuturism all 8 news articles »

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Scientists Favor Gene Editing, But Only For 'Fixing Diseases' - ValueWalk

Researchers may be one step closer to determining the cause of schizophrenia, after uncovering an abnormal genetic process associated with the disease that begins in the womb.

By transforming skin cells from patients with schizophrenia into neuronal progenitor cells - cells that form neurons in early development - researchers identified an abnormal gene pathway called nuclear FGFR1 (nFGFR1) that impairs early brain development.

Senior study author Michal K. Stachowiak, Ph.D., of the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo in New York, and colleagues say that their findings may bring us closer to treatments that could prevent schizophrenia in utero.

The researchers recently reported their results in the journal Schizophrenia Research.

According to the National Institute of Mental Health, around 1.1 percent of adults in the United States have schizophrenia - a mental health disorder characterized by hallucinations, delusions, and abnormal thoughts.

While the exact causes of schizophrenia remain unclear, researchers have long known that the condition can run in families, suggesting a genetic origin. Furthermore, an increasing number of studies have uncovered genetic mutations associated with an increased risk of schizophrenia.

For their study, Stachowiak and colleagues sought to learn more about the genomic processes that occur in utero that might influence the risk of schizophrenia development.

To reach their findings, the researchers collected skin cells from four adults with schizophrenia and four adults without the disorder.

The skin cells were reprogrammed into induced pluripotent stem cells, and these differentiated into neuronal progenitor cells. This enabled the team to assess the processes that occur during early brain development in people with schizophrenia.

The researchers pinpointed a dysregulated nFGFR1 pathway that targets and mutates numerous genes associated with schizophrenia. The team explains that just one of these gene mutations can impact brain development.

According to the authors, these findings provide proof of concept that schizophrenia may be caused by a dysregulated genomic pathway that influences the brain before birth.

"In the last 10 years, genetic investigations into schizophrenia have been plagued by an ever-increasing number of mutations found in patients with the disease. We show for the first time that there is, indeed, a common, dysregulated gene pathway at work here."

Michal K. Stachowiak, Ph.D.

Furthermore, the team says that these findings open the door to new schizophrenia treatments. For example, a drug could be administered to expectant mothers, whose offspring has a high risk of developing schizophrenia, that prevents processes related to the disease occurring in the developing fetus.

In future studies, the researchers plan to grow "mini brains" using the same processes used in the current study, with the aim of gaining a deeper understanding of how dysregulation of the nFGFR1 pathway influences early brain development, as well as to provide a model to test possible treatments.

Learn how B vitamins might improve symptoms of schizophrenia.

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Schizophrenia begins in the womb, study suggests - Medical News Today

February 28, 2017 A South African child is evaluated as part of a Stellenbosch University study. Credit: Stellenbosch University

A child's genetic make-up can play a large, hidden role in the success of efforts to maximize his or her development, South African research suggests.

The study, published February 28 in PLoS Medicine and supported by the Government of Canada through Grand Challenges Canada's Saving Brains program, sheds new light on why some children benefit more than others from interventions and raises complex questions about psychosocial intervention programs in future.

In a study led by Professor Mark Tomlinson of Stellenbosch University, the study followed-up an intervention implemented between 1999 and 2003, in which expectant mothers underwent mentoring to improve attachment with their childrenattachment being a measure of a child's psychological security, and predictive of future wellbeing. In the original study, a control group of roughly equal size was composed of expectant mothers who did not receive mentoring.

The original study concluded that the intervention had a small-to-moderate effect on mother-child attachment, evaluated once the children reached 18 months of age.

The follow-up study, conducted thirteen years after the intervention, re-examined the original attachment results and revealed something surprising: the intervention had in fact worked well for toddlers who had a particular genetic characteristic.

Conducted in collaboration with colleagues from the University of Reading, University College London, and Western University, the study re-enrolled and conducted genetic tests on 279 of the original 449 children.

220 children had both genetic and attachment data, enabling the investigators to test whether the original attachment outcomes were influenced by their genes.

The researchers factored in whether the child had the short or long form of gene SLC6A4the serotonin transporter gene, which is involved in nerve signalling, and which other studies have linked to anxiety, depression and other conditions. Serotonin is popularly thought to contribute to feelings of well-being and happiness.

The attachment of children with the short form of the gene, and whose pregnant mothers were mentored, were almost four times more likely to be securely attached to their mothers at 18 months old (84 percent were secure) than children carrying the short form whose mothers did not receive mentoring (58 percent were secure).

Meanwhile, children with the long gene were apparently unaffected by their mother's training or lack thereof: in both cases, the rate of secure attachment was almost identical (70 and 71 percent).

Subject to further validation, says Professor Tomlinson, the insight has "important implications for scientists designing and evaluating interventions to benefit as many people as possible in South Africa and worldwide."

"Without taking genetics into account, it is possible that other studies have under-estimated the impact of their interventions, as we originally did."

Says lead author Dr. Barak Morgan of the University of Cape Town: "The immediate significance of this research is the revelation that in principle, and probably in many cases in practice too, the effectiveness of interventions has been mis-measuredunder-estimated for genetically susceptible individuals and over-estimated for those who are genetically less susceptible. But even more worrying is the implication that the negative consequences of not receiving an intervention also differ by genotype."

"This is an enormously important insight because, in this case, the subgroup with the short form of the SLC6A4 gene is also the one with the most to lose if not helped."

"Individuals with the long form of the gene, on the other hand, appear less sensitive and derived little benefit from the same intervention, and little detriment from not getting it."

Adds Professor Tomlinson: "In the original study, we did not see such a big impact from this intervention because only those with the short gene improved, and this improvement was 'diluted' by the large number of children with the long gene who did not improve."

The researchers caution that, among other limitations, this study involved a relatively small sample and only measured one gene and one outcome (attachment).

Dr. Morgan stressed: "We are certainly not saying that only some people should receive the interventionthose who are 'susceptible' to improving from it. There is little scientific justification for this. For example, many children with the non-susceptible long genotype of the SLC6A4 gene may carry the susceptible form of another gene which renders them much more likely to benefit from the same intervention but for a different but equally important outcome.

"Going forward, the implications are therefore two-fold. Firstly, measuring genetic differences allows for proper assessment of the effectiveness or lack of effectiveness of an intervention for a particular outcome in different individuals. Secondly, this information can then be used to find out how to intervene effectively for allto guide what might be done to improve outcomes for a non-responsive gene-intervention interaction while continuing to optimise outcomes for the responsive one."

Says Dr. Karlee Silver, Vice President Programs of Grand Challenges Canada: "This work is fundamentally about better understanding the impact of interventions which is an important step forward to creating a world where every child can survive and thrive."

Says Dr. Peter A. Singer, Chief Executive Officer of Grand Challenges Canada: "This is a startling finding that changes the way I think about child development. Why is it important? Because child development is the ladder of social mobility used to climb out of the hole of inequity by millions of children around the world."

Explore further: Study explores how to tell children they have HIV

More information: "Serotonin Transporter Gene (SLC6A4) Polymorphism and Susceptibility to a Home-Visiting Maternal-Infant Attachment Intervention Delivered by Community Health Workers in South Africa: Re-analysis of a Randomized Controlled Trial" DOI: 10.1371/journal.pmed.1002237

Journal reference: PLoS Medicine

Provided by: Grand Challenges Canada

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To maximize a child's development, genetics provide important insight - Medical Xpress