Archive for the ‘Gene Therapy Research’ Category

Thirty-six percent of Hispanic families in the U.S. with a common form of retinitis pigmentosa got the disease because they carry a mutation of the arrestin-1 gene, according to a new study from researchers at The University of Texas Health Science Center at Houston (UTHealth) School of Public Health.

Retinitis pigmentosa is a group of rare, genetic eye disorders in which the retina of the eye slowly degenerates. The disease causes night blindness and progressive loss of peripheral vision, sometimes leading to complete blindness. According to Stephen P. Daiger, Ph.D., senior author of the study, an estimated 300,000 people in the U.S. suffer from the disease, which gets passed down through families.

In the study published recently in Investigative Ophthalmology & Visual Science, UTHealth researchers found that in a U.S. cohort of 300 families with retinitis pigmentosa, 3 percent exhibited a mutation of the arrestin-1 gene. However, more than 36 percent of Hispanic families from the cohort exhibited the arestin-1 mutation and they all came from areas in the Southwestern U.S., such as Texas, Arizona and Southern California.

When I started studying retinitis pigmentosa in 1985, we set out to find the one gene that causes the disease. Thirty-three years later, weve found that more than 70 genes are linked to retinitis pigmentosa, said Daiger, a professor in the Human Genetics Center and holder of the Thomas Stull Matney, Ph.D. Professorship in Environmental and Genetic Sciences at UTHealth School of Public Health.

Some of the genes that cause retinitis pigmentosa are recessive, which means two mutations are required, and some are dominant, which means you only need one mutation. Arrestin-1 piqued Daigers interest because that particular mutation is dominant while all previously found mutations in the gene are recessive. This unexpected finding shows that even a single mutation in the gene is sufficient to cause the disease.

Daiger and his team have identified the genetic cause of retinitis pigmentosa for 75 percent of families in their cohort. Possible treatments for some forms of retinitis pigmentosa are being tested but are still limited. However, the speed at which companies are developing gene therapies and small molecule therapies gives reason to hope, he said. Daiger and his collaborators have begun to connect some of the patients in the retinitis pigmentosa cohort to clinical trials that treat specific genes.

I want our cohort families to know that even if there is not an immediate cure for their specific gene mutation, at this rate it wont be long until a therapy becomes available, said Daiger, who also holds the Mary Farish Johnston Distinguished Chair in Ophthalmology at McGovern Medical School at UTHealth.

UTHealth coauthors include Lori S. Sullivan, Ph.D.; Sara J. Browne, Ph.D.; Elizabeth L. Cadena; Richard S. Ruiz, M.D., and Hope Northrup, M.D. Additional co-authors are from Nationwide Childrens Hospital; Kellogg Eye Center at the University of Michigan; Retina Foundation of the Southwest; Casey Eye Institute at Oregon Health and Science University; Vanderbilt University and the Department of Molecular and Human Genetics at Baylor College of Medicine.

Support for the study, titled A novel dominant mutation in SAG, the arrestin-1 gene, is a common cause of retinitis pigmentosa in Hispanic families in the Southwestern United States, was provided by the William Stamps Farish Fund and the Hermann Eye Fund.

Additional support was provided by the National Institutes of Health (EY007142, EY009076, EY011500, EY010572 and K08-EY026650), a Wynn-Gund TRAP Award, the Foundation Fighting Blindness, the Max and Minnie Voelker Foundation and a grant to the Casey Eye Institute from Research to Prevent Blindness.

Read more:
Gene Mutation Linked to Retinitis Pigmentosa in Southwestern US Hispanic Families - Texas Medical Center (press release)

San Francisco Business Times

Cell, gene therapies are hot. But can this startup make them safer? San Francisco Business Times ... and the CEO of Veneti. Using a cloud-based software platform, San Francisco's Vineti wants to be the FedEx of cell and gene therapies. ... Exclusive Online Tools. Research the 3+ year digital archive, and People on the Move leads database download.

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Cell, gene therapies are hot. But can this startup make them safer? - San Francisco Business Times

New gene editing tools transform disease models and future therapies CRISPR gene editing is taking biomedical research by storm. Providing the ultimate toolbox for genetic manipulation, many new applications for this technology are now being investigated and established. CRISPR systems are already delivering superior genetic models for fundamental disease research, drug screening and therapy development, rapid diagnostics, in vivo editing and correction of heritable conditions and now the first human CRISPR clinical trials.

The continuing patent battle for CRISPR-Cas9 licensing rights and the emergence of new editing systems such as Cpf1 has so far done nothing to slow the advance of CRISPR-Cas9 as the leading gene editing system. There are weekly press releases and updates on new advances and discoveries made possible with this technology; the first evidence is now emerging that CRISPR-Cas9 could provide cures for major diseases including cancers and devastating human viruses such as HIV-1.

The key to CRISPR-Cas9s uptake is its ease of application and design, with retargeting only a matter of designing new guide RNA. It has quickly surpassed TALENs (Transcription Activator-Like Effector Nucleases) and ZFNs (Zinc Finger Nucleases) where editing, now possible with CRISPR, was previously prohibitively complex and time-consuming. As well as correcting gene mutations with scar-less modifications, with CRISPR-Cas9 it is possible to control the expression of entire genes offering longer term expression alteration compared to other methods such as RNAi.

LNA GapmeRs are highly effective antisense oligonucleotides for knockdown of mRNA and lncRNA in vivo or in vitro. Designed using advanced algorithms, the RNase H-activating LNA gapmers offer excellent performance and a high success rate.

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CRISPR-Cas9 systems, tools and basic methodology are very accessible as ready to go toolkits that anyone with lab space and an idea can pick up and start working with. This is thanks largely to the efforts of Addgene and commercial service and product providers. Alongside CRISPR research there are innovations in companion technologies and design software. In response to a growing need, companies such as Desktop Genetics have developed open access software to accelerate CRISPR experimentation and analysis.

It is not all about CRISPR-Cas9 though. Like Cas9, Cpf1 is a DNA-targeting CRISPR enzyme that is also recruited to the target site by sequence homology but with slightly different site requirements. Cpf1 has been reported to be efficient and highly specific in human cells, with low off-target cleavage suggesting a role for Cpf1 in therapeutic applications down the line. Cas13a is an RNA-targeting CRISPR enzyme which is showing promise as a rapid diagnostic tool. Unlike Cas9, the enzyme continues to cut after it has acted on its intended RNA target, a characteristic which has been exploited to develop diagnostic technology for the likes of Zika and Dengue virus. The group behind SHERLOCK (Specific High Sensitivity Enzymatic Reporter UnLOCKing) combined this collateral effect of Cas13a with isothermal amplification and produced rapid DNA or RNA detection at attomolar sensitivity and with single-base mismatch specificity.

A particularly active area of CRISPR activity is the genetic manipulation of patient-derived stem cells to create models for diseases including Parkinsons, cystic fibrosis, cardiomyopathy and ischemic heart disease, to name but a few. With CRISPR it is now possible for researchers to correct disease-causing mutations in patient-derived pluripotent stem cells to create isogenic cell lines to differentiate to any cell type of interest for disease research. Generating these isogenic lines is making it possible, for the first time, to unambiguously show the contribution of gene mutations to a disease phenotype.

Dr Lise Munsie leads the pluripotent stem cell program at CCRM, a Canadian, not-for-profit organisation supporting the development of foundational technologies to support the commercialisation of cell and gene therapies, and regenerative medicine.

Gene editing technology now provides unlimited genetic flexibility to stem cell manipulation. You can target anywhere in the genome with relative ease and make it scar-less, saidDr Munsie.

Dr Munsies program is using CRISPR-Cas9 to produce reporter cell lines (for example with fluorescent protein inserted at a target gene) and isogenic lines from patient iPSCs. In stem cells, CRISPR-Cas9 is introduced with the Cas9 nuclease expressed from plasmid DNA or as purified Cas9 protein and the components are introduced into the cells by transfection or electroporation.

Dr Bjrn Brndl and his colleagues at the Lab for Integrative Biology at the Zentrum fr Integrative Psychiatrie, Universitatsklinikum Schleswig-Holstein, Germany, are also using stem cell gene editing to generate model systems for studying complex neurological disease such as Parkinsons and dyskinesia by correcting mutation in patient lines and introducing these mutations in control cells lines.

One of the biggest contributions of CRISPR to research is the ability to create isogenic stem cell lines. With these, we can create relevant disease models with near-perfect negative controls with the same genomic context varying only in the region of interest. Our goal is to compare disease patient lines with corrected lines by differentiating the induced pluripotent stem cells into neurons and studying differences in the phenotypes. In the biomedical field, we currently have a reproducibility crisis, so with clean and effective tools like isogenic pluripotent stem cells lines, we can improve the reproducibility and validity of our findings. One of the biggest challenges is working with the stem cells which are delicate and much more sensitive to the manipulations required for successful gene editing compared to standard cell lines.

CRISPR has completed upended how cell biology is approached. Being able to copy/paste DNA into the genomes has introduced a lot of ways of thinking about a problem. Genome editing has introduced engineering into the cell biology toolbox. saidDr Brndl.

An alarming number of bacteria are now resistant to our most effective antibiotics. The antibiotic resistance crisis has been given more of the attention it deserves thanks to initiatives from the WHO, UN, NICE and others but, in truth, the situation has been critical for over a decade. No new antibiotics have come out of pharma companies in the last 10 years and interest in their development has waned. Pharma companies are reluctant to invest the large sums required to develop new antimicrobials because of the inevitable resistant strains that will quickly follow and subsequent restrictions on their usage to preserve efficacy.

In short, we need a miracle, but the answer could come from CRISPR. Companies such as Nemesis Bioscience and Eligo Bioscience are developing antimicrobial technology and treatments made possible by CRISPR technology. Both technologies use modified bacteriophage as delivery vehicles for CRISPR-Cas9 gene editing systems that target and inactivate either virulence genes or the resistance genes themselves, leaving the rest of the microbiome intact.

Nemesis Bioscience employs CRISPR to target known bacterial resistance genes to deactivate them in situ and re-sensitise virulent bacteria making existing antibiotics effective again. Dr Frank Massam, CEO at Nemesis Biosciences explains, Killing bacteria stimulates resistance mutations we reasoned it would make more sense to inactivate bacterias ability to resist antibiotics and therefore make existing antibiotics work again. This approach would also mean that newly developed antibiotic assets could be protected from resistance, thereby increasing pharmas ROI and so making antibiotic development attractive again.

Nemesis Biosciences Symbiotics are based on modified CRISPR-Cas9 which enables highly multiplexed guide RNA targeting. Our first expression cassettes encode the S. pyogenes Cas9 plus a CRISPR array encoding guide RNAs that can target for inactivation members of 8 families of beta-lactamase genes. We call them the VONCKIST families, these are: VIM, OXA, NDM, CTX-M, IMP, SHV and TEM. The beta-lactamases encoded by these families are able to degrade >100 different types of beta-lactam antibiotics saidDr Massam.

The symbiotics are delivered by phage Transmids delivery vehicles based on phage architecture that deliver the DNA and then drop off. Once the Symbiotic is inside the bacteria, it can then spread further by conjugation from the edited bacteria to others it encounters, remaining invisible to the immune system. This provides both therapeutic applications as well as prophylactic ones in a probiotic delivery system to disarm the microbiome of antimicrobial-resistant bacteria. The technology is applicable to all bacteria, all antibiotic classes and all known resistance mechanisms and Nemesis have initially targeted resistant E. coli for in vivo testing.

Traditional small-molecule antibiotics target conserved bacterial cellular pathways or growth functions and therefore cannot selectively kill specific members of a complex microbial population. Eligo Biosciences flagship technology SSAMS eligobiotics, uses reprogrammed Cas9 targeted to bacterial virulence or resistance genes delivered by phagemids to produce selective killing of virulent and antibiotic resistant bacteria, leaving all other bacteria unaffected. The Eligo platform is being adapted for other microbial applications including in situ detection of specific live bacterial strains in complex microbiome samples and in situ expression of therapeutics protein to modulate and engineer host-microbiome interactions.

CRISPR-based therapies for human diseases could bring profound benefits to medicine, but there are many hurdles still to overcome. Despite the high degree of specificity of the CRISPR system, the induction of off-target mutations, at sites other than the intended target, is still a major concern especially in the context of therapeutic applications for heritable disease, and there are still considerable safety concerns about using CRISPR in humans. Assays for investigating the intended (on-target) and unintended (off-target) effects of CRISPR guides on in vitro and in vivo models are still in their infancy. The second major challenge is the development of safe carrier systems for CRISPR-Cas9 delivery to human cells in vivo.

Nonetheless, exciting progress is being made in the application of CRISPR gene editing to the treatment of heritable diseases for which there are only symptomatic treatments available, such as retinal myopathy where demonstrated recovery has been reported in a mouse model, and Duchenne muscular dystrophy, where the disease phenotype is reversed in mouse cells in vivo. We will also soon see the completion of the first clinical trials using CRISPR to try and correct genetic defects in vivo, the results of which are eagerly awaited.

There are a growing number of researchers from many disciplines collaborating to bring ambitious CRISPR-based insight, technology and therapeutics into the clinic. As CRISPR continues to undergo technical improvements, the prospects for these applications continues to look promising and as they move rapidly towards reality.

References

1. Yin, C., Zhang, T., Qu, X., Zhang, Y., Putatunda, R., Xiao, X., ... & Qin, X. (2017). In vivo excision of HIV-1 provirus by saCas9 and multiplex single-guide RNAs in animal models. Molecular Therapy.)

2. Hough SH, Kancleris K, Brody L, Humphryes-Kirilov N, Wolanski J, Dunaway K, Ajetunmobi A, Dillard V. Guide Picker is a comprehensive design tool for visualizing and selecting guides for CRISPR experiments. BMC bioinformatics. 2017 Mar 14;18(1):167.

3. Zetsche, B., Gootenberg, J. S., Abudayyeh, O. O., Slaymaker, I. M., Makarova, K. S., Essletzbichler, P., ... & Koonin, E. V. (2015). Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system. Cell, 163(3), 759-771.

4. Kleinstiver, B. P., Tsai, S. Q., Prew, M. S., Nguyen, N. T., Welch, M. M., Lopez, J. M., ... & Joung, J. K. (2016). Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells. Nature biotechnology, 34(8), 869-874.

5. Gootenberg JS, Abudayyeh OO, Lee JW, Essletzbichler P, Dy AJ, Joung J, Verdine V, Donghia N, Daringer NM, Freije CA, Myhrvold C. Nucleic acid detection with CRISPR-Cas13a/C2c2. Science. 2017 Apr 13:eaam9321

6. Bikard, D., Euler, C. W., Jiang, W., Nussenzweig, P. M., Goldberg, G. W., Duportet, X., ... & Marraffini, L. A. (2014). Exploiting CRISPR-Cas nucleases to produce sequence-specific antimicrobials. Nature biotechnology, 32(11), 1146-1150.

7. Zhang, X. H., Tee, L. Y., Wang, X. G., Huang, Q. S., & Yang, S. H. (2015). Off-target effects in CRISPR/Cas9-mediated genome engineering. Molecular Therapy-Nucleic Acids, 4, e264.

8. Yu, W., Mookherjee, S., Chaitankar, V., Hiriyanna, S., Kim, J. W., Brooks, M., ... & Swaroop, A. (2017). Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice. Nature Communications, 8.

9. Long, C., Amoasii, L., Mireault, A. A., McAnally, J. R., Li, H., Sanchez-Ortiz, E., ... & Olson, E. N. (2016). Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science, 351(6271), 400-403.

10. Nelson, C. E., Hakim, C. H., Ousterout, D. G., Thakore, P. I., Moreb, E. A., Rivera, R. M. C., ... & Asokan, A. (2016). In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science, 351(6271), 403-407.

Read more here:
CRISPR: Emerging applications for genome editing technology - Technology Networks

June 26, 2017 The researchers used the engineered viral vector AAV-PHP.S to label neurons lining the digestive tract with a cocktail of three distinct fluorescent proteins. Due to the stochastic uptake of viruses encoding either a blue, green or red fluorescent protein, cells are labeled with a wide range of hues. This multicolor approach can be used to differentiate neighboring neurons for morphology and tracing studies. Credit: Chan et al., Gradinaru Lab; Nature Neuroscience

Viruses have evolved to be highly effective vehicles for delivering genes into cells. Seeking to take advantage of these traits, scientists can reprogram viruses to function as vectors, capable of carrying their genetic cargo of choice into the nuclei of cells in the body. Such vectors have become critical tools for delivering genes to treat disease or to label neurons and their connective fibers with fluorescent colors to map out their locations. Because viral vectors have been stripped of their own genes and, thereby, of their ability to replicate, they are no longer infectious. Therefore, achieving widespread gene delivery with the vectors is challenging. This is especially true for gene delivery to hard to reach organs like the brain, where viral vectors have to make their way past the so-called blood-brain barrier, or to the peripheral nervous system, where neurons are dispersed across the body.

Now, to enable widespread gene delivery throughout the central and peripheral nervous systems, Caltech researchers have developed two new variants of a vector based on an adeno-associated virus (AAV): one that can efficiently ferry genetic cargo past the blood-brain barrier; and another that is efficiently picked up by peripheral neurons residing outside the brain and spinal cord, such as those that sense pain and regulate heart rate, respiration, and digestion. Both vectors are able to reach their targets following a simple injection into the bloodstream. The vectors are customizable and could potentially be used as part of a gene therapy to treat neurodegenerative disorders that affect the entire central nervous system, such as Huntington's disease, or to help map or modulate neuronal circuits and understand how they change during disease.

The work was done in the laboratory of Viviana Gradinaru, assistant professor of biology and biological engineering, Heritage Medical Research Institute Investigator, director of the Center for Molecular and Cellular Neuroscience in the Tianqiao and Chrissy Chen Institute for Neuroscience at Caltech, and principal investigator of the Beckman Institute's CLOVER (CLARITY, Optogenetics, and Vector Engineering Research) Center.

A paper describing the research appears online in the June 26 issue of Nature Neuroscience.

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"We have now developed a new collection of viruses and tools to study the central and peripheral nervous systems," says Gradinaru. "We are now able to get highly efficient brain-wide delivery with just a low-dose systemic injection, access neurons in difficult-to-reach regions, and precisely label cells with multiple fluorescent colors to study their shapes and connections."

Gradinaru and her team modified the external surface of an AAV developed in 2016, engineering the virus's shell, or capsid, to allow it to more efficiently deliver genes to cells in the brain and spinal cord following intravenous injection. They named the new virus AAV-PHP.eB.

The team also developed an additional capsid variant they call AAV-PHP.S, which is able to transduce peripheral neurons.

"Neurons outside of the central nervous system have many functions, from relaying sensory information to controlling organ function, but some of these peripheral neural circuits are not yet well understood," says Ben Deverman, senior research scientist and director of the Beckman Institute's CLOVER Center. "The AAV-PHP.S vector that we developed could help researchers study the activity and function of specific types of neurons within peripheral circuits using genetically-encoded sensors and tools to modulate neuronal firing with light or designer drugs, respectively."

The new AAV vectors can also deliver genes that code for colorful fluorescent proteins; such proteins are useful in identifying and labeling cells. In this process, multiple AAVseach carrying a distinct colorare mixed together and injected into the bloodstream. When they reach their target neurons, each neuron receives a unique combination of colors, thereby giving it a visually distinct hue that makes it easier for the researchers to distinguish its fine details from those of its neighbors. Furthermore, the team devised a technique to control the number of neurons labeledlabeling too many neurons makes it impossible to distinguish individual onesthat allows researchers to visualize individual neuron shapes and trace their connecting fibers through intact tissues using another technology the Gradinaru laboratory has helped develop, known as tissue clearing.

"Usually, when researchers want a mouse or other animal model to express fluorescent proteins in certain cells, they need to develop genetically modified animals that can take months to years to make and characterize," says former graduate student and first author Ken Chan (PhD '17). "Now with a single injection, we can label specific cells with a variety of colors within weeks after the injection."

"For our new systemic viral vectorsAAV PHP.S and AAV PHP.eBthere are many potential uses, from mapping circuits in the periphery and fast screening of gene regulatory elements to genome editing with powerful tools such as CRISPR-Cas9," says Gradinaru. "But perhaps the most exciting implication is that our tools, when paired with appropriate activity modulator genes, could enable non-invasive deep brain modulation for the treatment of neurological diseases such as Parkinson's disease."

The paper is titled "Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems."

Explore further: Delivering genes across the blood-brain barrier

More information: Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems, Nature Neuroscience (2017). DOI: 10.1038/nn.4593

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It may be true that Brain is Far Away. Rather than on Brains, Work on Intestines first. Just Feed Viruses through Mouth. Find Viruses That can Specifically line up Only The Interior of Mouse Intestines; Each TYPE of Virus should act as MAGNET for a Huge Collection of DIFFERENT TYPE OF BACTERIUM. Then, See Differences in their Effects on Mice! Also, in a Negative Way...i.e Driving Them Away...Making the Intestines Unlivable for those Bacteria that felt it as their home until then. Of course, Start working with Bacteria Natural to Intestinal Mucosa of Mice ONLY First !

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Novel viral vectors deliver useful cargo to neurons throughout the brain and body - Medical Xpress

Sweden aims to establish a new Center for Advanced Medical Products (CAMP) as part of a SEK 320-million ($36.6-million), 8-year Swedish government initiative to position the country as a leading biologics developer.

Swedish regenerative medicine firm Xintela has been appointed a partner in the 6-year project to establish the CAMP cell and gene therapy research center, with SEK 48 million ($5.5 million) in funding from the countrys innovation agency and research council, Vinnova and Vetenskapsrdet. Xintel said that as one of the CAMP initiative founders, it will work with Swedens universities, research institutes, and with firms including AstraZeneca, GE Healthcare and Pfizer. Xintela will initially act as an advisor for development of the center, but in the longer term expects to benefit from emerging R&D.

It is gratifying that the Swedish government, Vinnova and Vetenskapsrdet acknowledge the huge potential of cell and gene therapy and the strong position that Sweden has in this research field,commented Xintela CEO Evy Lundgren-kerlund. Xintela is one of the companies in Sweden with large development potential in cell therapy, which makes us a natural partner for this project.

In the short term CAMP aims to establish itself as an internationally recognised center for R&D, innovation and clinical practice, and to promote industrial growth and SMEs. Longer-term goals include attracting investment from the global pharmaceutical and biotech sectors.

Xintela is exploiting its XINMARK protein marker technology and XACT (Xintela assay for cell therapy) assay platform to develop an allogeneic mesenchymal stem cell-based therapy for repairing cartilage damage in osteoarthritis, and to progress a tumor-targeting antibody treatment for glioblastoma.

Last month the firm established a collaboration with Germany-based CO.DON, which develops autologous cell therapies for cartilage repair. The firms will work together on the development of Xintelas markers both for a next generation CO.DN cell therapy program and for Xintelas cartilage repair cell therapy product.

Read more:
Sweden Launches Initiative to Establish Center for Cell and Gene Therapy Research - Genetic Engineering & Biotechnology News

Photo: Keelin Daly / For Hearst Connecticut Media

Swimmers dive in for the Annual Swim Across America Greenwich-Stamford version in 2015. The funds raised by the event go to cancer gene therapy research. This years swim is set for Saturday.

Swimmers dive in for the Annual Swim Across America Greenwich-Stamford version in 2015. The funds raised by the event go to cancer gene therapy research. This years swim is set for Saturday.

The Annual Swim Across America Greenwich-Stamford in the Sound along the border of Greenwich and Stamford is set this year for Saturday. The funds raised by the event go to cancer gene therapy research.

The Annual Swim Across America Greenwich-Stamford in the Sound along the border of Greenwich and Stamford is set this year for Saturday. The funds raised by the event go to cancer gene therapy research.

Swim Across America Greenwich-Stamford set for Saturday

GREENWICH On Saturday, more than 200 swimmers are expected to take part in the 11th annual Swim Across America Greenwich-Stamford Swim, part of a national effort to raise money and awareness in the fight against cancer.

Event organizers say 100 percent of the proceeds go to the Alliance for Cancer Gene Therapy, which supports cancer cell and gene therapy research.

The local swim is chaired by town residents Michele Graham and Lorrie Lorenz, both of whom have had a child who was diagnosed with cancer. Nicole Graham is celebrating five years cancer free; Brooke Lorenz is six years cancer free.

Out of all the fundraisers we go to, this is the most fun, the most family oriented and the most beautiful, Michele Graham said. Its at a beautiful waterfront setting and people are so happy and joyful to be a part of it. Our vibe is a fun one. We want people to have fun and have it be an event that lifts people up.

Special guest speaker at the event will be Alec Fraser, father of Greenwich High School graduate Julian Fraser, who died of cancer earlier in the year.

Julian Fraser had been captain of GHS swim and water polo teams. He will be remembered at Saturdays swim by Team Julian, led by GHS swim team coaches Terry Lowe and Lorrie Hokayem.

This event attracts a lot of young people who might not otherwise be so directly involved in the fight against cancer, Alec Fraser said. Many of Julian's swim team and water polo teammates participate, both in Stamford and California. Although everyone knows someone who has had cancer, many of the members of Team Julian were his teammates, and so very personally affected by his loss.

Alec Fraser is also swimming in his sons honor.

Although I am not at all a swimmer, it is meaningful to me to be in the water with so many of his friends and former teammates to honor his memory in a sport and venue that was so important to him, the senior Fraser said.

Swimmers will dive into Long Island Sound starting from 96 Cummings Point Road early Saturday right at the border between Greenwich and Stamford. At 7 a.m. the three-mile swimmers will start.

At 8:30 a.m., the mile-and-a-half swimmers the largest group will dive into the water; the half-mile swimmers will jump in at 8:45. Swimmers start to return to shore about 8:50 a.m.; the award ceremony is slated to start at 10 a.m.

Since it started locally, more than $3.1 million has been raised for ACGT by close to 2,000 swimmers totaling 2,500 miles in the water.

Among the participants will be Greenwich resident Andy Alisberg, who is swimming with the team of Peters Defeaters.

Cancer has continued to intrude on the universe of people I know and about whom I care, Alisberg said. Vietor Evans, a childhood of his daughters, his graduate school suite mate Paul Stuka and a friend, Robin Zothian, were among people who have lost their lives to cancer or are fighting the disease.

The memory of those wonderful friends whom we have lost to cancer in recent years burns bright, Alisberg said.

Nationally, the Swim Across America effort has brought in $65 million since it was founded in 1965.

According to the organizations fact sheet, nearly 1.7 million new cases of cancer will be diagnosed and 600,920 people in the United States will die from the diseases in 2017 alone.

More information is online at http://www.SwimAcross

America.org/Greenwich.

kborsuk@greenwich

time.com

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Swim Across America Greenwich-Stamford set for Saturday ... - Greenwich Time

bluebird bio today disclosed its first data, including some promising results, from a pair of clinical studies assessing its LentiGlobin gene therapy candidateincluding a Phase III study that followed a change in its manufacturing process.

In the Phase III Northstar-2 (HGB-207) study of LentiGlobin in patients with transfusion-dependent -thalassemia (TDT) and non-0/0 genotypes, early interim data showed that the first 3 of 15 patients treated to date achieved higher drug product vector copy number (DP VCN) and lentiviral vector positive (LVV+) cell production than in the earlier Northstar (HGB-204) study, bluebird said.

Those results are correlated with higher production of hemoglobin A (HbA)T87Q and ultimately may address known patient-to-patient variability, bluebird bio CMO David Davidson, M.D., said in a statement.

The first patient treated in this study exemplifies the promise of gene therapy: discontinuing blood transfusions approximately a month after treatment and achieving a normal level of total hemoglobin production at six months post-treatment, Dr. Davidson stated.

According to bluebird, the first patient achieved normal total hemoglobin (13.3 g/dL) after discontinuing transfusions; producing 9.5 g/dl of HbAT87Q at last follow-up.

bluebird cautioned that the data was early, and that not all three patients enjoyed results as positive as the first patient. The second patient showed a lower percentage of LVV+ cells (53%) than either patients 1 (77%) or 3 (77% and 82%). However, neither patients 2 or 3 had sufficient follow-up to record clinically relevant data for total hemoglobin or days since last transfusion.

Northstar-2 is an ongoing, open-label, single-dose, international, multicenter study designed to evaluate the safety and efficacy of LentiGlobin drug product for the treatment of patients with TDT and non-0/0 genotypes. As of June 2, the drug product had been manufactured for six patients. The median DP VCN for these patients was 3.0 (range: 2.44.0), compared to a median DP VCN of 0.7 (range: 0.31.5) in Northstar.

Although early, these data add to the growing body of clinical evidence that indicate that LentiGlobin may offer a transformative benefit for patients with TDT, added Alexis Thompson, MD, of Ann & Robert H. Lurie Childrens Hospital of Chicago, a primary investigator on the study.

The Phase III study followed what the company said was an improvement in the manufacturing process by which the patients cells are transduced with the LentiGlobin viral vector, a change aimed at increasing vector copy number and the percentage of cells successfully transduced.

bluebird bio also announced results from its Phase I/II HGB-205 study, designed to assess LentiGlobin in patients with TDT and severe sickle cell disease (SCD). Those results, according to the company, showed the potential for durable treatment effect of LentiGlobin, with stable HbAT87Q production through 3.5 years of follow-up and sustained clinical benefit.

The company cited data from four TDT patients, all of whom remained free of transfusions since shortly after receiving LentiGlobin. At the last study visit, one patient had been free of transfusions for 41.9 months, compared with 38.7 months, 20.3 months, and 20.4 months for the other three patientsthe last of which was also homozygous for the severe + mutation IVS1-110.

In the three patients with SCD, the patient with the best results was producing 50% HbAT87Q , well above the approximately 30% anti-sickling hemoglobin level predicted to have potential clinical impact on the disease, at last follow-up 31.7 months following treatment.

The other two SCD patients showed levels of 20% and 15% at last follow-up (6.1 months and 3.4 months after treatment, respectively.)

HGB-205 is an ongoing, open-label, single-center study in which four patients with TDT, and three with severe SCD, have undergone infusion with LentiGlobin drug product as of June 2.

We are beginning to see evidence of the long-term durability of benefit from treatment with LentiGlobin, with some TDT patients even transitioning off of chelation therapy, said the HGB-205 studys primary investigator, Marina Cavazzana, M.D., Ph.D., a professor of medicine at Paris Descartes University, research director at the Centre for Clinical Research in Biotherapy, Necker Hospital, and at the Imagine Institute of Genetic Diseases in Paris.

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Early Data for bluebird bio Gene-Therapy Candidate Show Some Promise - Genetic Engineering & Biotechnology News

NobleGnthon has teamed up with ignominious Sarepta to develop a gene therapy for the Duchenne variety of the muscle wasting disease.

Dedicated to rare diseases since 1990 and more recently to gene therapy, Gnthon is one of veryfew not-for-profit companies in European biotech. Though it has not yet brought a drug to market, it is well established as a nonprofit, credit for which is due toits creator, the French Muscular Dystrophy Association, AFM Tlthon. The company has nowteamed up with the (in)famous American company, Sarepta, to work on a gene therapy for Duchenne Muscular Dystrophy (DMD).

DMD first affects the shoulder and upper arm muscles and the muscles of the hips and thighs. (Source: mda.org)

When I last spoke to Gnthons CEO,Frdric Revah, he told me that while the majority of the companys financial support comes from Tlthon, an increasing amount comes fromsuch partnerships.We get more and more support from industrial partnerships as we outlicense more of our drugs, explains Revah. However, Tlthon will always remain the main source of our funding; the funding from out-licensing is a complement.

But is Sarepta the best partner? While the American biotech can boast about its FDA-approved drug for DMD, Exondys 51, (which is just sold to Gilead for $125M,) this achievement isdubious:not only was the key clinical trial tiny, the FDAadmittedthat patients did not receive aclear benefit fromthe drug in the study. These circumstances prompted arenowned journalist to suefor thedocuments pertaining to the decision, an attack thatSareptas stock into a downward spiral in May.

Nevertheless, under the terms of the partnership agreement,Gnthon will trust Sarepta as a potential co-developer ofits DMD candidates, all of which are preclinical. The French biotechhas been developing amicro-dystrophin gene therapythat has proven itself applicable to the disease. As it countswith Europes largest cGMP vector manufactory, YposKesi, andone of the worlds largest research and clinical groups developing rare disease therapies, Gnthon is an attractive partner for any companies prospecting in the field.

Sareptas pricing practices may also offendthe sensibilities of a nonprofit, since theyraised the hackles of the drug pricing patrol with a plan to charge $300k per year for Exondys 51. According to STAT News, the companysCEO, Edward Kaye, saidthis figure isin the middle of the range for rare disease drugs,and given the sensitivity to pricing, we tried to be reasonable when looking at all the costs.

Though financial details of the agreement have not been disclosed, Gnthon may have enough influence to sway Sareptaaway from gouging.Our main goal is to ensure that patients have access to drugs and that they are affordable. Price should not be an obstacle,Revah told me.

Whatever we do here, we hope to apply the same tech to diseases that affect more people, like sickle cell anemia and cancer,he continued. With a crowded arena of companies like CRISPR Therapeutics, AMO Pharma, andDebiopharmall battling to bring the next DMD drug to market, having a back up plan via a platform seemssensible.

Images via Alila Medical Media, Anatomy Insider / shutterstock.com

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French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy - Labiotech.eu (blog)

SAN FRANCISCO, Calif. Mayo Clinic Ventures has partnered with a California-based company to make cancer-fighting gene therapies available to the public.

Vineti, a pioneering cell and gene therapy software and analytics company, announced Tuesday that it had completed its initial round of funding raising $13.75 million aimed at delivering "the first cloud-based software solution to improve patient access, accelerate life-saving treatment delivery, and promote safety and regulatory compliance for individualized cell therapies."

The funding was provided by Mayo Clinic Ventures, GE Ventures, DFJ and LifeForce Capital. It's just the 15th company that Mayo Clinic Ventures has backed since it was formed, according to Andy Danielson, vice chairman of Mayo Clinic Ventures.

"One thing with Vineti that we liked is that we have a commitment to cell and gene therapies at Mayo," Danielson told TechCrunch.com. "Vineti will make the gene and cell therapy production process more efficient and as a result, less costly. It's all part of the equation of making these therapies more affordable and opening them up to a greater number of people."

The targeted cancer therapy under development by Vineti is part of a thriving field that conducted more than 800 clinical trials in 2016 while investing nearly $6 billion. It's all aimed at positively impacting the oncology field, the largest market in medicine that's expected to grow to $165 billion by 2021.

The first two cell therapies are expected to hit the market later this year.

Vineti touts its plans as one that "integrates logistics, manufacturing and clinical data to improve product performance overall and enable faster, broader access for patients."

"Physicians, medical researchers and pharmaceutical companies are working together to develop successful therapies, transitioning from a one-size-fits-all model to individualized treatments for each patient," said Amy DuRoss, CEO at Vineti. "Now, the process for creating and delivering these treatments can be as innovative as the therapies themselves. We are developing the Vineti platform to help these treatments reach the patients who need them the most, and are confident the partnership between our advances technologies and leading medical research will deliver better outcomes across the globe."

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Mayo Clinic Ventures funds new cancer-fighting cell, gene therapy - Post-Bulletin

Behind the incredible process of developing targeted gene therapies to fight diseases like cancer lies an incredibly mundane problem that prevents these treatments from getting to patients paperwork and procedures.

While $5.7 billion was invested in companies developing cellular and genetic therapies, and with 800 clinical trials initiated worldwide and the first two CAR-T cell therapies expected to launch into market later this year, businesses still saythe ability to get these treatments to patients is limited by paperwork, supply chain management, and last mile delivery.

So GE (through its GE Ventures arm), the Mayo Clinic (through Mayo Clinic Ventures) and the venture investment firm DFJ have invested $13.75 million to back Vineti a software platform the companies are billing as a solution to gene therapys supply chain problem.

Its only the sixth company to have actually been built by GEs internal business team and spun out by the conglomerates venture arm.

According to company co-founder and former GE Ventures managing director Amy DuRoss, the process for developing and managing gene therapies is critical to the success of the treatment.

Amy DuRoss, chief executive at Vineti

To that end, Vinetis software tracks logistics, manufacturing and clinical data to improve treatments and drive down the cost of these therapies (which are mainly only accessible to those people with the very best health plans).

The startups technology was actually born out of necessity (always the mother of invention) and came from conversations that GE was having with a large, undisclosed customer.

A pharma company that is a regular client of GE Healthcare said we are solving late stage cancer and we want to take this commercial but we have not got the technology that can ensure that we can scale out these technologies in the commercial phase, DuRoss told me.

GEs healthcare business then took the problem to the companys venture investment and new business arm and began the development process of building a business.

In addition to DuRoss, who has been a luminary in the life sciences field since she helped with the push to get stem cell research approved in California; Vineti has a murderers row of leading healthcare talent.

Chief strategy officer Heidi Hagen, was the former SVP of Operations for cell immunotherapy pioneer Dendreon; chief technology officer Razmik Abnous was the chief technology officer at the healthcare data management juggernaut Documentum; and Malek Faham, the companys chief science officer, literally worked on some of the foundational science for gene therapies.

While the companys technology could have applications for a number of different treatments, and be used for several kinds of therapies, the focus, for now, is on cancer.

Cancer is a bullseye, says DuRoss. It is arguably the biggest cause of human suffering [and] there are treatments already in phase three, that if brought to market effectively could mark a turning point in medicines battle against the deadly disease, she said.

We see an opportunity as data accrues to the system over time for a use case in predicting therapy based on outcome data but were not making these claims today, said DuRoss.

Mayo Ventures had been working with GE for two years from the initial concept to the close of this new round of financing for Vineti. Its one of only 15 companies that the Clinic has backed since the formation of Mayo Clinic Ventures, and according to Andy Danielsen, the vice chair of Mayo Clinic Ventures.

One thing with Vineti that we liked is that we have a commitment to cell and gene therapies at Mayo, said Danielsen, so the interests were aligned. Vineti will make the gene and cell therapy production process more efficient and as a result, less costly. Its all part of the equation of making these therapies more affordable and opening them up to a greater number of people.

Therapy supply chain

External ordering pages

Product tracking

Therapy scheduling

Identity verification

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GE and the Mayo Clinic back software to bring cancer-fighting gene therapies to market - TechCrunch

LONDON--(BUSINESS WIRE)--Cell Medica today announced the acquisition of Catapult Therapy TCR Limited, a subsidiary of Cell and Gene Therapy Catapult (CGT Catapult), and the initiation of a collaboration to establish cell therapy manufacturing for Cell Medica at CGT Catapults GMP manufacturing facility in Stevenage, UK. Financial terms were not disclosed.

Catapult Therapy TCR Ltd is a special purpose company set up by CGT Catapult, UCL Business and Imperial Innovations, and managed by CGT Catapult, for the development of the WT1 T cell receptor (TCR) cell therapy discovered through research at University College London (UCL) and Imperial College London. The WT1-TCR cell therapy enhances the immune system to fight cancer by genetically engineering the patients T cells to target WT1, a tumour-associated antigen which is expressed in both solid tumours and blood cancers.

CGT Catapult has been developing the WT1-TCR cell therapy for the treatment of acute myeloid leukaemia and myelodysplastic syndrome. Early development work, including initiation of a Phase I trial, was conducted at UCL and Imperial College London with funding from the UK charity Bloodwise. CGT Catapult advanced the product to a larger Phase I/II clinical trial and developed an improved manufacturing process. Having completed the treatment of eight patients with promising results, CGT Catapult will now transfer the WT1-TCR cell therapy rights to Cell Medica for continued development towards regulatory approval.

The WT1-TCR cell therapy will be integrated with the Dominant TCR platform technology which Cell Medica licensed from UCL Business in 2016. Applying the Dominant TCR technology to the WT1-TCR cell therapy is expected to result in a more efficacious product with the potential to treat patients with solid tumours such as mesothelioma and ovarian cancer, which have proven very difficult to treat with conventional therapies. Cell Medica is planning to initiate a Phase I/II clinical trial with a Dominant WT1-TCR version in late 2018.

Cell Medica and CGT Catapult have also initiated a collaboration to establish cell therapy manufacturing operations for Cell Medica at the GMP production facility recently built by CGT Catapult in Stevenage. The collaboration will include transferring the current WT1-TCR cell therapy manufacturing process to Stevenage over the next twelve months while Cell Medica and CGT Catapult work to develop a commercial scale production process using advanced manufacturing techniques. Cell Medica will also evaluate the feasibility of manufacturing additional cell therapy products at the site.

The acquisition of the WT1-TCR cell therapy leverages the investment we made in 2016 for exclusive rights to the Dominant TCR technology, said Gregg Sando, CEO of Cell Medica. Our objective is to show how we can enhance any existing TCR cell therapy with the Dominant TCR technology to create a more effective treatment for patients with solid tumours who otherwise have a very poor prognosis. We are also looking forward to an important collaboration with CGT Catapult to initiate manufacturing at the Stevenage GMP facility where we will work together on scale-up strategies for commercial production.

About Cell Medica

Cell Medica is committed to transforming patients lives through developing the significant therapeutic potential of cellular immunotherapy for the treatment of cancer. In collaboration with our strategic partners, Cell Medica is developing a range of products using three proprietary technology platforms including activated T cells, chimeric antigen receptors (CARs) and engineered T cell receptors (TCRs). Our lead product is CMD-003 is being tested in an international Phase II trial for the treatment of cancers associated with the oncogenic Epstein Barr virus. We are working with the Baylor College of Medicine and the University of North Carolina to develop next generation CAR-modified NKT cells including an off-the-shelf product. In the field of engineered TCRs, we are collaborating with University College London to develop the Dominant TCR technology platform. Cell Medica is headquartered in London with subsidiaries in Zurich and Houston.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 120 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

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Cell Medica Acquires WT1 Cancer Immunotherapy from Cell and ... - Business Wire (press release)

The potential of genome-editing techniques, such as CRISPR/Cas9, to alleviate disease burden has ignited the imagination for thousands of researchers looking for new therapeutic strategies. Scientists were very quickly able to show that this gene-altering technique could eliminate disease-causing mutations within a variety of tissues in vitro. More recently, CRISPR is being positioned to help treat patients directly, with clinical trials in humans already under way in China and soon to begin in the U.S. Yet, no current clinical trials feature drugs made using the technique for the treatment of neurodegenerative diseases.

Now, a group of investigators led by scientists at Emory University is hoping to open up new avenues of neurodegenerative research and rapidly move toward human trials after the release of their new findings. The research team showed that the CRISPR/Cas9 system could snip part of a gene that produces toxic protein aggregates in the brains of 9-month-old mice used as a model for Huntingtons disease. Moreover, the scientists noted that when they looked at the brain region where the vector was applied, some weeks later, the aggregated proteins had almost disappeared. Amazingly, the motor abilities of the mice had improved, although not to the level of control mice.

Findings from the new study were published today in the Journal of Clinical Investigation through an article entitled, CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntingtons disease.

Huntington's disease is caused by a gene encoding a toxic protein (mutant huntingtin or mHTT) that causes brain cells to die. Symptoms commonly appear in mid-life and include uncontrolled movements, balance problems, mood swings, and cognitive decline. The mice used in the current study have a human mHTT gene replacing one of the mouse huntingtin genes. In these mice, motor problems and aggregated mHTT can be observed around the age of 9 months.

We report that permanent suppression of endogenous mHTT expression in the striatum of mHTT-expressing mice (HD140Q-knockin mice) using CRISPR/Cas9-mediated inactivation effectively depleted HTT aggregates and attenuated early neuropathology, the authors wrote. The reduction of mHTT expression in striatal neuronal cells in adult HD140Q-knockin mice did not affect viability, but alleviated motor deficits. Our studies suggest that nonallele-specific CRISPR/Cas9-mediated gene editing could be used to efficiently and permanently eliminate polyglutamine expansion-mediated neuronal toxicity in the adult brain.

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CRISPR Reverses Huntington's Disease in Mice - Genetic Engineering & Biotechnology News

Philly invented the computer then whiffed on becoming home to the tech industry. Will we do better with the next-gen medical breakthroughs were hatching?

Illustration by Muti.

In the conference room of a spacious loft office near 30th Street Station, Usman Oz Azam and Michael Christiano huddle for a meeting. Theyre CEO and chief business officer, respectively, of Tmunity Therapeutics, a start-up launched to commercialize some breakthrough research out of the University of Pennsylvania a method of reengineering a patients own immune system cells so they can destroy cancer tumors.

The two execs are the companys sole employees so far. Their new headquarters still contains chalkboard-style signs and leather couches from the prior tenant, some sort of hipster

telemarketing operation. It looks like an abandoned Cosi restaurant.

Literally the only thing weve done is bring in a coffee machine, Azam says.

Its a microscopically small start to what some people around Philadelphia hope will become very, very big.

Down the hill at the Center for Advanced Cellular Therapeutics, an alliance between Penn Medicine and global drug maker Novartis is working on other pioneering immune system research, which, like Tmunitys technology, comes from the labs of Penns Carl June. It involves extracting T-cells from patients, genetically modifying them so they can attack blood cancer, and putting them back into patients, where the altered cells multiply and become a sort of living medicine. Trials on some leukemia patients have obliterated the disease, and this designed-in-Philly treatment is on track to be the first-ever FDA-approved personalized cellular therapy for cancer.

One floor below, in the same University of Pennsylvania building, scientists at the Parker Institute for Cancer Immunotherapy are working on what could become Americas first use of the genome-editing tool CRISPR in human beings, aimed at cancer. Its funded in part by Sean Parker, the Internet billionaire played by Justin Timberlake in The Social Network (who gave $250 million to six medical schools and cancer centers). Its not the only CRISPR venture in the city. Nearby, on Market Street, the start-up Excision BioTherapeutics is working to commercialize technology from Temple University that uses CRISPR to snip viruses such as HIV out of affected patients.

Just one block up Market Street, in the penthouse-floor laboratory of Spark Therapeutics, researchers cultivate cells in flasks of a liquid that looks like weak cherry Kool-Aid. Spark, founded in 2013 to commercialize research from Childrens Hospital of Philadelphia, is what companies like Tmunity and Excision seek to become. Spark employs around 240 people and has a stock market capitalization of $1.61 billion. In May the company filed with the FDA to create the first gene-therapy treatment for an inherited disease in the United States. The treatment, which involves injecting new genes into patients to replace the imperfect ones they were born with, has reversed a rare form of blindness in clinical trials. It could be approved by the end of the year, with gene treatments for hemophilia to follow. Theyll all be produced right here, at 38th and Market.

And so were having a moment in Philadelphia. Were out to an early lead in the race to become a hub for the next generation of medicine: synthetic biology that augments the human immune system to make it better than nature did. Its a future in which medicine is less about gulping pills to mitigate symptoms than about using targeted gene and cell therapies that begin to approach cures for ailments as serious as blindness, hemophilia, AIDS and cancer. If we dont fumble the opportunity, its also a future that the Philadelphia area can own. People speak hopefully of a regional economy blossoming around these innovations an ecosystem, a magnet, the way Los Angeles is for entertainment, the way Washington is for lobbyists.

I can make the argument that Philadelphia has the greatest concentration of foundational research in gene and cell therapy in the world, says Spark CEO Jeffrey Marrazzo, a Philly native who convinced CHOP to make the company its first commercial spin-off.

Were at a unique moment, says Richard Vague, a managing partner at Philly-based Gabriel Investments who has personally funded some of Carl Junes research. The genome has been mapped. The revolution in technology has occurred. So much that needs to happen has happened. The opportunity for real breakthroughs is palpable.

Some smarties at Penn Medicine have cooked up an optimistic nickname for this region: Cellacon Valley. Its an awkward portmanteau, probably no more likely to catch on than Phillywood. Really, its a brand name for a desire. Maybe this could be our thing (besides cheesesteaks, which we know to be effective in humans). As much as anything, the nickname encapsulates a new attitude, an eagerness to better exploit advances in medicine coming out of local labs. Theres been a history of missed opportunity here, a gap in translating the genius of our academia to the marketplace. Remember, back in 1946, Penn scientists introduced ENIAC, the worlds first electronic computer. But the Delaware Valley didnt become Silicon Valley, or even Vacuum Tube Valley.

Theres a whole negative piece that could be done about what Philadelphia did not do that Boston or San Francisco did, says Steven Nichtberger, a longtime entrepreneur who teaches Wharton undergrads how to commercialize scientific ideas. At MIT and Stanford, pitching moneymaking schemes to venture capitalists is part of the university culture, as routine as joining the Dungeons & Dragons club.

Other places have had reasons they became really good at what they do, Carl June says. Now, this is our chance. I hope we dont blow it.

Looking south fromhis window in the Perelman Center for Advanced Medicine, on Penns rampantly spreading medical campus, Jonathan Epstein can see the smokestacks of Philadelphias industrial legacy.

I think we have a chance to see a new and important industry arise here, he says. Since hes chief scientific officer at Penn Medicine, one of his missions is to help make that happen. On a recent field trip to present Philly to venture capitalists in San Francisco, Epstein floated the phrase Cellacon Valley.

I actually had screens around the auditorium showing the modern parts of Philadelphia, in order to convey the sense that its not the old manufacturing city that many people have in their minds. It is hard to change peoples impressions, he admits.

Epstein thinks of Kendall Square the once-desolate area around MIT in Cambridge, Massachusetts, thats now crawling with shiny biotech buildings as a model. I grew up in Boston, and I like to tell people that Kendall Square is where they used to tow my car, he says. I watched the development of that region.

In West Philly, the makeover has begun. On streets around Penn and Drexel, you can hardly have a conversation that isnt drowned out by the beeps of a construction vehicle erecting another gleaming high-rise. But Penn getting richer or even University City becoming an innovation district isnt the same as success for the region. Around Boston, the prosperity from waves of computer tech and biotech has had a blast radius of 25 miles, to far-flung neighborhoods and suburbs. Its more extreme around San Francisco. Could we have that kind of success here? What would it look like?

If you think of Silicon Valley or Hollywood, theyre places people go with dreams. Its where the jobs they want are. If you leave a company in Mountain View or Kendall Square, theres another across the road or down the highway that needs staff. Small firms grow into big ones and seed new start-ups like acorns. Venture money burns holes in the pockets of investors who live in the area. And behind the headline-making companies are enterprises that support the IPO superstars, providing specialized staffing, equipment and facilities.

Philadelphia has some of these things. It has companies like WuXi AppTech in the Navy Yard, which contracts to do cell production and R&D. Tmunity plans to use a cell-manufacturing facility in East Norriton previously owned by Tengion, whose ambitious plans to grow human tissue ran out of runway, resulting in a 2014 bankruptcy. If you want to make a drug, its easy to hire a contract manufacturer. But if you want to make a cell therapy, it takes particular kinds of facilities and expertise which exist here now, says Nichtberger, the former CEO of Tengion.

The region, of course, has a medical and pharmaceutical legacy that goes back centuries. Ben Franklin founded Pennsylvania Hospital in 1751. In 1830, John K. Smith opened a Philly drugstore that would evolve into Smith, Kline & French and eventually the massive GSK. The Wistar Institute transformed global health with vaccines for rubella, rabies, and other life-threatening diseases. Bob McGrath, who heads Drexels office of technology commercialization, points out that four of the National Cancer Institutes 69 designated cancer centers are here, at Penn, Jefferson, Fox Chase and Wistar. There is infrastructure to build on.

But in this new world, infrastructure isnt enough. Whats also required: money to back academic research, and, no less important, an entrepreneurial culture that lets scientists see a path to making their work available to the world profitably, if possible. Its going to be hard to retain or attract the very best scientists who are entrepreneurial if there isnt an environment here in which they can see their dreams come true, Epstein says.

Thats required a culture change, at Penn in particular.

For Ivy League schools, commercializing technology is a very new event, says June, director oftranslationalresearch at Penns Abramson Cancer Center. Google came out of Stanford. It never could have come out of Penn back then. Ivy League schools kind of turned their nose up at funding from commercial entities. The faculty were encouraged to get peer-reviewed and go for government funding.

These days, the word translational is on business cards and medical buildings all over University City. It means translating science from bench to bedside, from the lab to the patient. That can cost multiple millions of dollars, and government money isnt what it used to be. When I moved to Penn in 1999, I was like most faculty here in that over 80 percent of my research funding was from the NIH, June says. Now, its less than 20 percent. Attracting funding from other sources is more and more important.

Pretty much all the local institutions are trying to get better at it. In a sense, theyve begun throwing open their lab coats to the world and saying, Check us out. Sparks Marrazzo says that in 2011, Steve Altschuler, then CEO at CHOP, invited him to walk the corridors there looking for commercial ideas. Its literally true, Marrazzo says. He said, Why dont you spend three months? Ill give you the authority to walk the halls and meet with scientists, see if theres anything there that you think is worth trying to take a bolder step.

Not-for-profit universities and hospitals cant raise money by selling stock. But they can take equity in the start-up companies built around their intellectual property, which is almost as good. Marrazzo says CHOP invested $35 million in Spark. They have already liquidated between $150 million and $200 million, and they still have 18 or 19 percent of the business, which is worth probably another $300 million to $400 million, he says. CHOP has now spun out three other companies and hired serial entrepreneur Patrick FitzGerald to a new position: Vice President for Entrepreneurship & Innovation.

Penns medical school offers a masters degree in translational research, which MD and PhD candidates can add to their studies. The course helps researchers undergo a Nutty Professor style transformation from meek lab-focused scientists to confident presenters of their assets to potential funders

Theyve got to learn how to articulate their science to different audiences to give the elevator pitch, the TED talk, says Emma Meagher, a cardiologist and clinical pharmacist who runs the program. When they go to the NIH, they have to articulate the science as a scientific discipline. When they go to a not-for-profit foundation, theyve got to articulate the science as it appeals to the unmet medical need for that foundation. To a for-profit funder, its Are you gonna make me money, wheres your business plan?

Drexels McGrath is a matchmaker who connects faculty inventors with commercial partners. A review committee helps them groom pitches. The Lankenau Institute for Medical Research, in Wynnewood, has an incubator to develop companies spawned from its research. CEO George Prendergast calls it a small Bell Labs of bioscience. He invented the word acapreneurial to describe the model marrying academics with entrepreneurship.

At Temples Lewis KatzSchool of Medicine, legal experts from the universitys technology transfer office embed in the med school and look at the facultys papers and grant applications before they go out and see if there are elements we ought to protect with respect to the intellectual property, says Steven Houser, senior associate dean of research there. If you dont protect it, no one in the private sector will ever want to invest in your idea.

Racking up patents isnt an official yardstick for evaluating faculty. Not yet, Houser says. There are discussions at most institutions about whether things like patents should be considered in tenure discussions.

So how do we enter the Valley? It doesnt happen by chance. It happens because people make strategic moves, Penns Epstein says.

The schools outreach to industry is part of it. Public funding and local incentives like tax breaks are also still important, even as the Trump administration pushes for big cuts to NIH and NCI budgets. Pennsylvania has a patchwork of aid. Governor Wolfs new budget proposes selling bonds to raise $45.9 million for CURE, which issues health-research grants. State-supported Ben Franklin Technology Partners offers seed capital to start-ups. Keystone Innovation Zones provide corporate tax credits. But June envies what other states have done. A California voter initiative provided $3 billion for stem-cell therapies. Texas sold bonds to raise $3 billion for cancer research. Its recruiting people and new companies. We need to do that here, June says.

Of course, amid all the possibility, theres a need to proceed carefully. Gene therapy isnt like building a new mobile app. Human health is at stake. Pioneer Jim Wilsons experience tells a cautionary tale and offers another kind of hope.

Wilson created Penns gene-therapy program when he arrived there in 1993. There was a tremendous amount of enthusiasm in the mid-90s, he recalls. Its the natural trajectory of an emerging technology: Theres a lot of expectation and anticipation, a little knowledge, and in this particular area it was fueled by all of us. By the PR machines of the universities. By the investigators, who could garner resources. By patient populations, especially those with rare diseases who just were neglected. Gene therapy sounded different. It wasnt just another drug. It was: I have a bad gene and youre gonna fix it.

Then, in 1999, Jesse Gelsinger died, in a Penn gene-therapy trial directed by Wilson. The 18-year-olds body reacted adversely to the virus used as a vector to deliver a new gene to his system. It turned out Wilson owned stock in a company that was funding the research. There was a lawsuit, and much debate about the conflicts that arise when science gets in bed with business. People walked away from the field, Wilson says.

Humbled and censured, Wilson kept a low profile but continued to work, landing on a safer vector thats part of the current rebirth of gene therapy. Two companies based on his new work, though, located elsewhere. RegenXBio is in Rockville, Maryland. Dimension Therapeutics is in Cambridge, despite the fact that the R&D engine was here at Penn, Wilson says. We tried everything we could to convince management and the investors that it would make more sense to place these companies at Penn.

But ecosystems build one victory at a time. Kevin Mahoney, executive vice dean for integrative services for Penns medical school, had hinted to me about such a victory: a secret new gene start-up coming to the city even though the moneymen tried to insist that it be headquartered in Kendall Square. They said, It can only be in Kendall Square. And we said, No way. It has to be in Philadelphia, Mahoney says.

Wilson wanted to tell me the secret, too. Its a new company called Scout Bio, based on gene therapy technology out of my lab at Penn, he reveals. It uses gene therapies in small animal health pets to deliver therapeutic proteins for anemia, atopic dermatitis, cancer. Were building that in Philadelphia.

Yo, dog. Welcome to Cellacon Valley.

Published as Cellacon Valley? in the July 2017 issue of Philadelphia magazine.

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Welcome to Cellacon Valley? - Philadelphia magazine

GenSight has announced Phase I/II data revealing that its gene therapy technology can restore sight in patients suffering from a rare mitochondrial disease.

GenSight Biologics develops gene therapies targeting degenerative diseases that cause blindness. The French biotech has now announced very promising results from its lead candidate, GS010, after 2 years of following patients treated with the gene therapy in an ongoing Phase I/II trial. Thetreatment targets Leber hereditaryoptic neuropathy (LHON), a rare genetic disease for which there is no curative treatment.

Patients sight was evaluated using the ETDRS test, the one consisting on recognizing increasingly smaller letters that most of us have done at some point in our life. Those patients treated with GS010 showed a statistically significant improvement in the number of letters they were able to recognize over time, especially in those that were treated within two years after their diagnosis. Detailed results after 96 weeks of follow-upare now pending publication in a peer-reviewed journal.

TE: treated eye; UTE: untreated eye

According to GenSight, 95% of LHON cases are caused by mutations in the genes that encodes the NADH dehydrogenase complex, which is involved in ATP metabolism within mitochondria. Since it affects amitochondrial gene, the disease is maternally inherited. GenSight is particularly focusing on patients with a mutation in the ND4 gene, which accounts for 70% of LHON cases in Europe and North America and up to 85% in Asia.

GenSight is already running two Phase III studies in Europe and the US evaluating GS010 in patients with the ND4 mutation that have been affected by LHON for a year or less.We are now less than a year away from Phase III efficacy data, and more than ever committed to find a cure for patients and their families affected by this devastating condition, said Bernard Gilly, CEO and co-founder of Gensight, in a statement. Philip recently interviewed him regarding his impressive track in biotech as both a serial entrepreneur and a partner at the VC firm Sofinnova.

So far, GenSight seems to be the only biotech developing a gene therapy for this disease. Ocular disorders are often rare,which leads most companies in the field to focus on age-related macular degeneration instead, which has a significantly higher prevalence. The French biotechs pipeline also includes GS011, a gene therapy to treat the ND1 mutation in LHON, still in the early research stage. The company is also working in GS030, an optogenetic gene therapyto introduce a protein that can respond to light with the aim of restoring sight in patients with retinitis pigmentosa, currently undergoing preclinical investigation.

Images via HQuality / Shutterstock; GenSight

See the article here:
French Biotech Reports Sight Restoration thanks to Gene Therapy - Labiotech.eu (blog)

A delivery for ALS? AveXis is currently using the gene therapy vector NAV AAV9, developed by REGENXBIO in Maryland, to deliver its experimental gene therapy for SMA into the spinal cord. The approach, which aims to increase levels of SMN, is beginning to show signs of benefit including motor function according to interim phase 1 results presented by Nationwide Childrens Hospitals Jerry Mendell and colleagues at the 2017 meeting of the American Academy of Neurology in Boston. [Image: National Human Genome Research Institute.]

AveXis is one step closer to developing a potential gene therapy for SOD1 ALS. The gene therapy company, based in Cleveland, Ohio, announced this month it has obtained the rights to develop treatments for ALS using REGENXBIOs gene therapy delivery vehicle. The emerging vector, derived from adeno-associated virus 9 (AAV9), is being increasingly utilized to deliver potential therapies into the CNS for neurological diseases.

The strategy is one of a growing number of potential gene therapies for SOD1 ALS that aims to reduce levels of misfolded SOD1 in the CNS and in the muscles by silencing the expression of the SOD1 gene (see May 2017 conference news). The approach is being developed by a research team led by Nationwide Childrens Hospitals Brian Kaspar in Ohio, who is also AveXis chief scientific officer and scientific founder (see December 2015 conference news; Thomsen et al., 2014; Foust et al., 2013).

The delivery vehicle, known as NAV AAV9, forms the basis of AveXis experimental gene therapy for the motor neuron disease spinal muscular atrophy (SMA). The strategy, known as AVXS-101, is currently being evaluated at the phase 1 stage at Nationwide Childrens Hospital as a treatment for Type 1 SMA, the most severe form of the disease. The approach builds on previous studies in 2009 led by Institute of Myologys Martine Barkats in France and Brian Kaspar in the United States, which found that AAV9 could cross the blood-brain barrier and therefore, could be used to potentially treat motor neuron diseases (seeDecember 2008news;Duque et al., 2009;Foust et al., 2009).

Meanwhile, Martine Barkats, in collaboration with Maria Grazia Biferi, in France is using a different approach in hopes to treat SOD1 ALS (see May 2017 conference news). The strategy, which uses a related gene therapy delivery vehicle known as AAV10, also aims to reduce motor neuron toxicity by silencing the expression of the SOD1 gene. The strategy is currently being optimized and is at the preclinical stage. The researchers are now developing a similar strategy in hopes to treat C9orf72 ALS, the most common form of the disease.

***

To learn more about gene therapy and its potential for ALS, check out our recent news feature: A New Potential Gene Therapy Delivers A Key Milestone.

References

Thomsen GM, Gowing G, Latter J, Chen M, Vit JP, Staggenborg K, Avalos P, Alkaslasi M, Ferraiuolo L, Likhite S, Kaspar BK, Svendsen CN. Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex. Neurosci. 2014 Nov 19;34(47):15587-600. [PubMed].

Foust KD, Salazar DL, Likhite S, Ferraiuolo L, Ditsworth D, Ilieva H, Meyer K, Schmelzer L, Braun L, Cleveland DW, Kaspar BK. Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol Ther. 2013 Dec;21(12):2148-59. [PubMed].

Duque S, Joussemet B, Riviere C, Marais T, Dubreil L, Douar AM, Fyfe J, Moullier P, Colle MA, Barkats M. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther. 2009 Jul;17(7):1187-96. [PubMed].

Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2009 Jan;27(1):59-65 [PubMed].

Further Reading

van Zundert B, Brown RH Jr. Silencing strategies for therapy of SOD1-mediated ALS. Neurosci Lett. 2017 Jan 1;636:32-39. [PubMed].

Tora MS, Keifer OP Jr, Lamanna JJ, Boulis NM. The challenges of developing a gene therapy for amyotrophic lateral sclerosis. Expert Rev Neurother. 2017 Apr;17(4):323-325. [PubMed].

AAN2017 aav9 c9orf72 disease-als gene therapy SOD1 vector

Read more:
Gene Therapy Biotech AveXis Targets SOD1 ALS - ALS Research Forum

Life-threatening allergies and asthma could one day be treated by a single injection, say researchers who have successfully treated mice using gene therapy.

Dr Ray Steptoeofthe University of Queensland in Australia and colleagues 'turned off' the immune system's memory of an allergen in mice, suggesting that it could be possible for a single treatment to permanently stop the cause of allergic reactions, rather than just managing the symptoms.

The immune system's memory is the underlying cause of both asthma and allergies, as immune cells incorrectly recognise and 'remember' allergens as being potentially dangerous, andmount an immune response. Repeated exposure to an allergen can cause increasingly severe and potentially fatal reactions. However, it is extremely difficult for potential therapies to contend with the permanence ofimmune memory.

The researchers worked with mice who were allergic to a protein found in egg white. They first inserted a gene which regulates the egg white protein into blood stem cells then transplanted these modified stem cells into the allergic mice. Transplanting the modified stem cells was enough to remove the mice's immune memory of the egg white protein as an allergen, meaning that the animals were no longer sensitive to the protein.

'We have now been able "wipe" the memory of these T-cells in animals with gene therapy, de-sensitising the immune system so that it tolerates the protein,' said Dr Steptoe. 'This research could be applied to treat those who have severe allergies to peanuts, bee venom, shell fish and other substances.'

But the findings should be treated with some caution, given the early stages of the research, note some. Professor Adnan Custovic at University College London told The Independent: 'A mouse model is not the same as a human model We can cure allergies in mice but we cannot do it in humans the mechanisms are not identical. Only time will tell whether this approach will be a viable one.'

The researchers are now working on making the treatment simpler and safer and it is hoped that human trials could begin in as little as five years.

Asthma is a major public health issue with some 5.4 million people in the UK with the condition; costing the NHS 1 billion annually. As allergies play a significant role in around 75 percent of asthma cases, as well as affecting the 44 percent of British adults who have at least one allergy, there is a need to produce effective, long-term treatments for these conditions.

The research was published in JCI Insight.

Read more here:
Gene therapy hope for allergies and asthma - BioNews

ANI | Washington D.C. [USA] June 11, 2017 Last Updated at 13:42 IST

Glucocorticoid steroids, such as prednisone, may improve effectiveness of AAV-based gene therapy by reducing immune response, according to a recent research.

The study of gene transfer using adeno-associated virus (AAV)-based gene delivery into skeletal muscle of rhesus macaques showed that oral prednisone reduced immune responses to AAV that can weaken expression of the therapeutic transgene over time.

Animals given prednisone before the gene therapy had a 60% decrease in immune cell infiltrates, mainly comprised of cytotoxic T cells, according to the study.

Megan Cramer, The Ohio State University, Paul Martin, The Research Institute at Nationwide Children's Hospital and The Ohio State University College of Medicine, Columbus, and coauthors also reported that AAV-treated muscles had higher levels of a biomarker called PD-L2, which can induce programmed T-cell death.

"Prednisone is frequently used in conjunction with AAV gene therapy in the hope of blunting harmful immune responses to the AAV capsid. However, very little is known about the precise immune mechanisms involved in its use, or even if it is beneficial with various different routes of AAV administration," said Editor-in-Chief Terence R. Flotte.

The research appears in Human Gene Therapy.

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

Glucocorticoid steroids, such as prednisone, may improve effectiveness of AAV-based gene therapy by reducing immune response, according to a recent research.

The study of gene transfer using adeno-associated virus (AAV)-based gene delivery into skeletal muscle of rhesus macaques showed that oral prednisone reduced immune responses to AAV that can weaken expression of the therapeutic transgene over time.

Animals given prednisone before the gene therapy had a 60% decrease in immune cell infiltrates, mainly comprised of cytotoxic T cells, according to the study.

Megan Cramer, The Ohio State University, Paul Martin, The Research Institute at Nationwide Children's Hospital and The Ohio State University College of Medicine, Columbus, and coauthors also reported that AAV-treated muscles had higher levels of a biomarker called PD-L2, which can induce programmed T-cell death.

"Prednisone is frequently used in conjunction with AAV gene therapy in the hope of blunting harmful immune responses to the AAV capsid. However, very little is known about the precise immune mechanisms involved in its use, or even if it is beneficial with various different routes of AAV administration," said Editor-in-Chief Terence R. Flotte.

The research appears in Human Gene Therapy.

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

ANI

http://bsmedia.business-standard.com/_media/bs/wap/images/bs_logo_amp.png 177 22

Link:
Steroids may up effectiveness of AAV-based gene therapy ... - Business Standard

A small study presented at the worlds largest cancer conference found treating patients with the drug olaparib could slow cancer growth by three months and be less toxic for patients with inherited BRCA-related breast cancer. A type of inherited and incurable breast cancer that tends to affect younger women could be targeted by a new gene therapy, researchers have found. However, researchers have said there was not enough data to say whether patients survived longer as a result of the treatment.

We are in our infancy, said Dr Daniel Hayes, president of the American Society of Clinical Oncology and professor of breast cancer research at the University of Michigan. This is clearly an advance; this is clearly proof of concept these can work with breast cancer. Does it look like its going to extend life? We dont know yet, he said.

The drug is part of the developing field of precision medicine, which targets patients genes to tailor treatment.

It is a perfect example of how understanding a patients genetics and the biology of their tumor can be used to target its weaknesses and personalize treatment, said Andrew Tutt, director of the Breast Cancer Now Research Centre at The Institute of Cancer Research.

Olaparib is already available for women with BRCA-mutant advanced ovarian cancer, and is the first drug to be approved that is directed against an inherited genetic mutation. The study was the first to show olaparib can slow growth of inherited BRCA-related breast cancer. The drug is not yet approved for that use.

People with inherited mutations in the BRCA gene make up about 3% of all breast cancer patients, and tend to be younger. The median age of women in the olaparib trial was 44 years old.

BRCA genes are part of a pathway to keep cells reproducing normally. An inherited defect can fail to stop abnormal growth, thus increasing the risk of cancer. The study examined the effectiveness of olaparib against a class of BRCA-related cancers called triple negative. Olaparib is part of a class of four drugs called PARP-inhibitors that work by shutting down a pathway cancer cells use to reproduce.

Patients who received olaparib saw cancer advance in seven months, versus four months for only chemotherapy. (Shutterstock )

The study from Memorial Sloan Kettering Cancer Center in New York randomly treated 300 women with advanced, BRCA-mutated cancer with olaparib or chemotherapy. All the participants had already received two rounds of chemotherapy.

About 60% of patients who received olaparib saw tumors shrink, compared with 29% of patients who received chemotherapy. That meant patients who received olaparib saw cancer advance in seven months, versus four months for only chemotherapy.

Researchers cautioned it is unclear whether olaparib extended life for these patients, and that more research was needed to find out which subset of patients benefit most from olaparib.

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Read the original post:
A new gene therapy may help fight against an incurable form of breast cancer - Hindustan Times

Sessions/Tracks

Track 1:Molecular Biology

Molecular biologyis the study of molecular underpinnings of the processes ofreplication,transcription,translation, and cell function. Molecular biology concerns themolecularbasis ofbiologicalactivity between thebiomoleculesin various systems of acell,gene sequencingand this includes the interactions between theDNA,RNAand proteinsand theirbiosynthesis. Inmolecular biologythe researchers use specific techniques native to molecular biology, increasingly combine these techniques and ideas from thegeneticsandbiochemistry.

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2nd World Congress onHuman Genetics&Genetic Disorders, November 02-03, 2017 Toronto, Canada; 9th International Conference onGenomicsandPharmacogenomics, June 15-16, 2017 London, Uk; 6th International Conference and Exhibition onCellandGene Therapy, Mar 27-28, 2017 Madrid, Spain; Gordon Research Conference,Viruses&Cells, 14 - 19 May 2017, Lucca, Italy;Human Genome Meeting(HGM 2017), February 5-7 2017, Barcelona, Spain; Embl Conference:Mammalian GeneticsAndGenomics:From Molecular Mechanisms To Translational Applications, Heidelberg, Germany, October 24, 2017;GeneticandPhysiological Impacts of Transposable Elements, October 10, 2017, Heidelberg, Germany.

American Society for Cell Biology;The Society for Molecular Biology & Evolution;American Society for Biochemistry and Molecular Biology;The Nigerian Society of Biochemistry and Molecular Biology;Molecular Biology Association Search Form - CGAP.

Track 2:Gene Therapy and Genetic Engineering

Thegenetic engineeringis also called asgenetic modification. It is the direct manipulation of an organism'sofgenomeby usingbiotechnology. It is a set of technologies used to change the genetic makeup of the cell and including the transfer of genes across species boundaries to produce improved novelorganisms. Genesmay be removed, or "knocked out", using anuclease.Gene is targetinga different technique that useshomologousrecombinationto change anendogenous gene, and this can be used to delete a gene, removeexons, add a gene, or to introducegenetic mutations. There is an dna replacement therapy, Genetic engineering does not normally include traditional animal and plant breeding, gene sequencing, in vitro fertilization, induction of polyploidy,mutagenesisand cell fusion techniques that do not use recombinant nucleic acids or a genetically modified organism in the process,diseases treated with gene therapywas initially meant to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular dystrophy and sickle cell anemia

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8thWorld Congress onMolecular Pathology, June 26-27, 2017 San Diego, USA; 11thInternational Conference onSurgical Pathology& Practice, March 27-28, 2017, MADRID, SPAIN; 13th EuropeanPathologyCongress, Aug 02-03, 2017, MILAN, ITALY; 28th Annual Meeting, Austrian Society ForHuman GeneticsAnd The Swiss Society OfMedical GeneticsCombined Meeting 2017 march 29, 2017 - March 31, 2017, bochum , Germany.

Association for Clinical Genetic Science;Genetics Society of America | GSA;Association of Genetic Technologists;Molecular Genetics - Human Genetics Society of Australasia;Genetic Engineering - Ecological Farming Association.

Track 3:Cell & Gene Therapy

Cell therapy is also calledcellular therapyorCyto therapy, in which cellular material is injected into patient this generally means intact, living cells. The first category iscell therapyin mainstream medicine. This is the subject of intense research and the basis of potential therapeutic benefit. Such research can be controversial when it involves human embryonic material. The second category is in alternative medicine, and perpetuates the practice of injecting animal materials in an attempt to cure disease.Gene therapyis the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease. Gene therapy is a way to fix agenetic problemat its source. The polymers are either translated into proteins, interfere with targetgene expression, or possibly correct genetic mutations. The most common form uses DNA that encodes a functional,therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a "vector", which carries the molecule inside cells. Vectors used in gene therapy, the vector incorporates genes intochromosomes. The expressed nucleases then knock out and replace genes in the chromosome. The Center forCell and Gene Therapyconducts research into numerous diseases, including but not limited to PediatricCancer, HIV gliomaandCardiovascular disease.

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2nd World Congress onHuman Genetics&Genetic Disorders, November 02-03, 2017 Toronto, 27 Canada ; 7th International Conference onPlant Genomics, July 03-05, 2017, Bangkok, Thailand ; American Society ofGeneandCell Therapy(ASGCT) 20th Annual Meeting, 10 - 13 May 2017, Washington, DC;Genomic Medicine for Clinicians(course), January 25-27, 2017, Hinxton , Cambridge, UK; Embo Conference:ChromatinandEpigenetics, Heidelberg, Germany, May 3, 2017; 14th International Symposium on Variants in theGenomeSantiago de Compostela, Galicia, Spain, June 5 - 8, 2017;

Genetics and Molecular Medicine - American Medical Association;Genetics Society of America / Gsa;British Society for Genetic Medicine;British Society for Gene and Cell Therapy; Australasian Gene Therapy Society.

Track 4:Cell Cancer Immunotherapy

Immunologydeals with the biological and biochemical basis for the body's defense against germs such as bacteria, virus and mycosis (fungal infections) as well as foreign agents such asbiological toxinsand environmental pollutants, and failures and malfunctions of these defense mechanisms. Cancer immunotherapy is the use of the immune system to treat cancer. Immunotherapies can be categorized as active, passive or hybrid (active and passive). Antibodies are proteins produced by the immune system that bind to a target antigen on the cell surface. The immune system normally uses them to fight pathogens. A type of biological therapy that uses substances to stimulate or suppress the immune system to help the body fight cancer, infection, and other diseases. Some types of immunotherapy only target certain cells of the immune system. Others affect the immune system in a general way. Types of immunotherapy include cytokines, vaccines, bacillus Calmette-Guerin (BCG), and some monoclonal antibodies.

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9thAnnual Meeting onImmunologyandImmunologist, July 03-05, 2017 Kuala Lumpur, Malaysia; 8th MolecularImmunology&ImmunogeneticsCongress, March 20-21, 2017 Rome, Italy; 8th EuropeanImmunologyConference, June 29-July 01, 2017 Madrid, Spain; July 03-05, 2017; B Cells and T Follicular Helper Cells Controlling Long-Lived Immunity (D2), April 2017, 2327, Whistler, British Columbia, Canada; Mononuclear Phagocytes in Health,Immune Defense and Disease, 304 May, Austin, Texas, USA;Modeling Viral Infections and ImmunityMAY 2017, 14, Estes Park, Colorado, USA; IntegratingMetabolism and Immunity(E4)292 June, Dublin, Ireland.

The American Association of Immunologists;Clinical Immunology Society ; Indian Immunology Society;IUIS - International Union of Immunological Societies;American Society for Histocompatibility and Immunogenetics.

Track 5:Clinical Genetics

Clinical geneticsis the practice of clinical medicine with particular attention tothe hereditary disorders. Referrals are made togenetics clinicsfor the variety of reasons, includingbirth defects,developmental delay,autism,epilepsy, and many others. In the United States, physicians who practice clinical genetics are accredited by theAmerican Board of Medical Genetics and Genomics(ABMGG).In order to become a board-certified practitioner of a Clinical Genetics, a physician must complete minimum of 24 months of his training in a program accredited by the ABMGG. Individual seeking acceptance intoclinical geneticstraining programs and should hold an M.D. or D.O. degree (or their equivalent)and he/she have completed a minimum of 24 months of their training in ACGME-accredited residency program internal medicine, pediatrics and gynecology or other medical specialty.

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Belgian Society OfHuman GeneticsMeeting 2017 february 17, 2017, Belgium; American College Of Medical Genetics 2017 AnnualClinical GeneticsMeeting march 21-25 2017, phoenix , United States; German Society Of Human Genetics 28th Annual Meeting, Austrian Society ForHuman GeneticsAnd The Swiss Society OfMedical GeneticsCombined Meeting 2017 march 29, 2017 - March 31, 2017, bochum , Germany; Spanish Society OfHuman GeneticsCongress 2017april 25, 2017 - April 28, 2017 madrid , Spain;

Clinical Genetics Associates;Clinical Genetics Society(CGS);The genetic associate;International Conference on Clinical and Medical Genetics;Association for Clinical Genetic Science;The American Society of Human Genetics.

Track 6:Pharmacogenetics

Pharmacogeneticsis the study of inherited genetic differences in drug metabolic pathways which can affect individual responses towards the drugs, both in their terms of therapeutic effect as well as adverse effects. In oncology, Pharmacogenetics historically is the study ofgerm line mutations(e.g., single-nucleotide polymorphisms affecting genes coding forliver enzymesresponsible for drug deposition and pharmacokinetics), whereaspharmacogenomicsrefers tosomatic mutationsin tumoral DNA leading to alteration in drug response.

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Spanish Society OfHuman GeneticsCongress 2017april 25, 2017 - April 28, 2017, madrid , Spain; 8th World Congress onPharmacology, August 07-09, 2017 Paris, France; World Congress onBio therapeutics, May 22-23, 2017, Mexico City, Mexico; 8th World Congress OnPharmacologyAndToxicology, July 24-26, 2017, Melbourne, Australia; German Society Of Human Genetics 28th Annual Meeting, Austrian Society ForHuman GeneticsAnd The Swiss Society OfMedical GeneticsCombined Meeting 2017march 29, 2017 - March 31, 2017 bochum , Germany.

Pharmacogenomics - American Medical Association;Associate Principal Scientist Clinical Pharmacogenetics;European Society of Pharmacogenomics and Personalised Therapy;Genome-wide association studies in pharmacogenomics.

Track 7:Molecular Genetic Pathology

Molecular genetic pathologyis an emerging discipline withinthe pathologywhich is focused in the study and diagnosis of disease through examination of molecules within the organs, tissues or body fluids. A key consideration is more accurate diagnosis is possible when the diagnosis is based on both morphologic changes in tissuestraditional anatomic pathologyand onmolecular testing. Molecular Genetic Pathology is commonly used in diagnosis of cancer and infectious diseases. Integration of "molecular pathology" and "epidemiology" led tointerdisciplinaryfield, termed "molecular pathological epidemiology" (MPE),which representsintegrative molecular biologicand population health science.

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8th World Congress OnMolecular Pathology, June 26-27, 2017 San Diego, USA; 11th International Conference OnSurgical Pathology& Practice, March 27-28, 2017, Madrid, Spain; 13th EuropeanPathologyCongress, Aug 02-03, 2017, Milan, Italy; Embl Conference:Mammalian GeneticsAndGenomics, Heidelberg, Germany, October 24, 2017; Embo|Embl Symposium: TheMobile Genome: Genetic And Physiological Impacts Of Transposable Elements, Heidelberg, Germany, October 10, 2017.

Clinical Pathology Associates Molecular Pathology; Association mapping Wikipedia;Association for Molecular Pathology(AMP);Molecular Pathology - Association of Clinical Pathologists;SELECTBIO - Molecular Pathology Association of India.

Track 8:Gene Mapping

Genomemappingis to place a collection of molecular markers onto their respective positions ongenome.Molecular markerscome in all forms. Genes can be viewed as one special type of genetic markers in construction ofgenome maps, and the map is mapped the same way as any other markers. The quality ofgenetic mapsis largely dependent upon the two factors, the number of genetic markers on the map and the size of themapping population. The two factors are interlinked, and as larger mapping population could increase the "resolution" of the maps and prevent the map being "saturated". Researchers begin a genetic map by collecting samples of blood or tissue from family members that carry a prominent disease or trait and family members that don't. Scientists then isolate DNA from the samples and closely examine it, looking for unique patterns in the DNA of the family members who do carry the disease that the DNA of those who don't carry the disease don't have. These unique molecular patterns in the DNA are referred to as polymorphisms, or markers.

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3rd WorldBio Summit&Expo, Abu Dhabi, UAE, June 19-21, 2017; 9th International Conference onGenomicsandPharmacogenomicsJune 15-16, 2017 London, Uk; Keystone Symposium: Mononuclear Phagocytes in Health,Immune DefenseandDisease, 304 May 2017, Austin, Texas, USA;Molecular Neurodegeneration(course) Hinxton, Cambridge, UK, January 9-14, 2017;

Association for Clinical Genetic Science;Genome-wide association study Wikipedia;Gene mapping by linkage and association analysis NCBI;Gene mapping by linkage and association analysis | Springer Link.

Track 9:ComputationalGenomics

Computational genomics refers to the use of computational and statistical analysis to decipherbiologyfromgenome sequencesand related data, including DNA and RNA sequence as well as other "post-genomic" data. This computational genomics is also known asComputational Genetics. These, in combination with computational and statistical approaches to understanding the function of the genes and statistical association analysis, this field is also often referred to as Computational and Statistical Genetics/genomics. As such, computational genomics may be regarded as a subset of bioinformatics and computational biology, but with a focus on using whole genomes rather than individual genes to understand the principles of how the DNA of a species controls its biology at the molecular level and beyond. With the current abundance of massive biological datasets, computational studies have become one of the most important means to biological discovery.The field is defined and includes foundations in thecomputer sciences,applied mathematics, animation, biochemistry, chemistry, biophysics,molecular genetics,neuroscienceandvisualization. Computational biology is different from biological computation, which is a subfield of computer engineering using bioengineering and biology to build computers, but is similar tobioinformatics.

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Modeling Viral Infections and Immunity,10. MAY 2017, 14, Estes Park, Colorado, USA;Integrating Metabolism and Immunity(E4)292 June, Dublin, Ireland; EMBL Conference:Mammalian GeneticsandGenomics, Heidelberg, Germany, October 24, 2017; EMBO|EMBL Symposium: The Mobile Genome:GeneticandPhysiological Impacts of Transposable Elements, Heidelberg, Germany, October 10, 2017;

American Association of Bio analysts - Molecular/Genetic Testing;ISCB - International Society for Computational Biology;International Society for Computational Biology Wikipedia;Bioinformatics societies OMICtools;Towards an Australian Bioinformatics Society.

Track 10:Molecular Biotechnology

Molecular Biotechnologyis the use of living systems and organisms to develop or to make products, or "any technological application that uses the biological systems, living organisms or derivatives, to make or modify products or processes for specific use. Molecular biotechnology results from the convergence of many areas of research, such as molecular biology, microbiology, biochemistry, immunology, genetics and cell biology. It is an exciting field fueled by the ability to transfer genetic information between organisms with the goal of understanding important biological processes or creating a useful product. The completion of the human genome project has opened a myriad of opportunities to create new medicines and treatments, as well as approaches to improve existing medicines. Molecular biotechnology is a rapidly changing and dynamic field. As the pace of advances accelerates, its influence will increase. The importance and impact of molecular biotechnology is being felt across the nation. Depending on the tools and applications, it often overlaps with the related fields of bioengineering,biomedical engineering, bio manufacturing andmolecular engineering.Biotechnologyalso writes on the pure biological sciences animalcell culture, biochemistry,cell biology, embryology, genetics, microbiology, andmolecular biology.

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8th EuropeanImmunologyConference, June 29-July 01, 2017 Madrid, Spain; World Congress onBio therapeutics, May 22-23, 2017, Mexico City, Mexico;Human Genome Meeting(HGM 2017), February 5-7 2017, Barcelona, Spain;Integrating MetabolismandImmunity (E4), 292 June, Dublin, Ireland.

Biotech Associations - Stanford University;Indian Society of Genetics, Biotechnology Research & Development;Genetics and Molecular Medicine - American Medical Association;Genetics Society of America | GSA, British Society for Genetic Medicine;Heritability in the Era of Molecular Genetics - Association for Psychological science.

Track 11:Genetic Transformation

Genetic Transformationis the genetic alteration of cell resulting from the direct uptake and incorporation ofexogenous genetic materialfrom its surroundings through thecell membrane. Transformation is one of three processes for horizontal gene transfer, in which exogenous genetic material passes from bacterium to another, the other two being conjugation transfer of genetic material between two bacterial cells in direct contact andTransductioninjection offoreign DNAby a bacteriophage virus into thehost bacterium. And about 80 species of bacteria were known to be capable of transformation, in 2014, about evenly divided betweenGram-positiveandGram-negative Transformation" may also be used to describe the insertion of new genetic material into non-bacterial cells, including animal and plant cells.

RelatedMolecular Biology Conferences| Genetics Conferences|Gene Therapy Conferences|Biotechnology Conferences| Immune Cell Therapy Conferences

13th EuropeanPathologyCongress, Milan, Italy; Embl Conference:Mammalian GeneticsAndGenomics, Heidelberg, Germany, October 24, 2017; Embo|Embl Symposium: TheMobile Genome: Genetic And Physiological Impacts Of Transposable Elements, Heidelberg, Germany, October 10, 2017; 2nd World Congress onHuman Genetics&Genetic Disorders, November 02-03, 2017 Toronto, Canada; 9th International Conference onGenomicsandPharmacogenomics, June 15-16, 2017 London, Uk;

American Society of Gene & Cell Therapy: ASGCT;Gene Therapy Societies and Patient Organizations - Gene Therapy Net;European Society of Gene and Cell Therapy (ESGCT);British Society for Gene and Cell Therapy;Gene Therapy - American Medical Association.

Track 12:Genetic Screening

Genetic screenis an experimental technique used to identify and select the individuals who possess a phenotype of interest inmutagenized population. A genetic screen is a type ofphenotypic screen. Genetic screen can provide important information on gene function as well as the molecular events that underlie a biological process or pathway. While thegenome projectshave identified an extensive inventory of genes in many different organisms, genetic screens can provide valuable insight as to how thosegenes function.

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13th EuropeanPathologyCongress, Aug 02-03, 2017, Milan, Italy; 2nd World Congress onHuman Genetics&Genetic Disorders, November 02-03, 2017 Toronto, 27 Canada; 7th International Conference onPlant Genomics, July 03-05, 2017, Bangkok, Thailand; Embl Conference:Mammalian GeneticsAndGenomics, Heidelberg, Germany, October 24, 2017; Embo|Embl Symposium: TheMobile Genome: Genetic And Physiological Impacts Of Transposable Elements, Heidelberg, Germany, October 10, 2017, 10 - 13 May 2017, American Society ofGeneandCell Therapy(ASGCT) 20th Annual Meeting, Washington, DC;

Association for Clinical Genetic Science; Association for Molecular Pathology (AMP);Mapping heritability and molecular genetic associations with cortical;Genetics and Molecular Medicine - American Medical Association.

Track 13:Regulation of Gene Expression

Regulation of Gene expressionincludes a wide range of mechanisms that are used by cells to increase or decrease the production of specific gene products (protein or RNA), and is informally termed gene regulation. Sophisticated programs of gene expression are widely observed in biology, Virtually any step of gene expression can be modulated, fromtranscriptional initiation,RNA processing, and post-translational modificationof a protein. Often, one gene regulator controls another in a gene regulatory network. Any step of gene expression may be modulated, from theDNA-RNA transcriptionstep to post-translational modification of a protein.

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7th International Conference onPlant Genomics, July 03-05, 2017, Bangkok, Thailand; EMBO|EMBL Symposium: The Mobile Genome:GeneticandPhysiological Impacts of Transposable Elements, Heidelberg, Germany, October 10, 2017; 10. MAY 2017, 14, Estes Park, Colorado, USA,Modeling Viral Infections and Immunity; 292 June, Dublin, Ireland,Integrating Metabolism and Immunity(E4); MAY 2017, 14, Estes Park, Colorado, USA,Modeling Viral InfectionsandImmunity; 8th EuropeanImmunologyConference, June 29-July 01, 2017 Madrid, Spain; 9th International Conference onGenomicsandPharmacogenomics, June 15-16, 2017 London, Uk;

Gene Therapy Societies and Patient Organizations - Gene Therapy Net;European Society of Gene and Cell Therapy (ESGCT);British Society for Gene and Cell Therapy;Gene Therapy - American Medical Association

Track 14: Cancer Gene Therapy

Cancer is an abnormal growth of cells the proximate cause of which is an imbalance in cell proliferation and death breaking-through the normal physiological checks and balances system and the ultimate cause of which are one or more of a variety of gene alterations. These alterations can be structural, e.g., mutations, insertions, deletions, amplifications, fusions and translocations, or functional (heritable changes without changes in nucleotide sequence). No single genomic change is found in all cancers and multiple changes (heterogeneity) are commonly found in each cancer generally independent of histology. In healthy adults, the immune system may recognize and kill the cancer cells or allow non-detrimental host-cancer equilibrium; unfortunately, cancer cells can sometimes escape the immune system resulting in expansion and spread of these cancer cells leading to serious life threatening disease. Approaches to cancer gene therapy include three main strategies: the insertion of a normal gene into cancer cells to replace a mutated (or otherwise altered) gene, genetic modification to silence a mutated gene, and genetic approaches to directly kill the cancer cells. Pathway C represents immunotherapy using altered immune cells. Another unique immunotherapy strategy facilitated by gene therapy is to directly alter the patient's immune system in order to sensitize it to the cancer cells. One approach uses mononuclear circulating blood cells or bone marrow gathered from the patient.

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8th EuropeanImmunologyConference, June 29-July 01, 2017 Madrid, Spain; World Congress onBio therapeutics, May 22-23, 2017, Mexico City, Mexico;Human Genome Meeting(HGM 2017), February 5-7 2017, Barcelona, Spain;Integrating MetabolismandImmunity (E4), 292 June, Dublin, Ireland.

Biotech Associations - Stanford University;Indian Society of Genetics, Biotechnology Research & Development;Genetics and Molecular Medicine - American Medical Association;Genetics Society of America | GSA, British Society for Genetic Medicine;Heritability in the Era of Molecular Genetics - Association for Psychological science.

Track 15:Genetic Transplantation

Transplantation genetics is the field of biology and medicine relating to the genes that govern the acceptance or rejection of a transplant. The most important genes deciding the fate of a transplanted cell, tissue, or organ belong to what is termed the MHC (the major histocompatibility complex). Genetic Transplantation is the moving of an organ from one body to another or from a donor site to another location on the person's own body, to replace the recipient's damaged or absent organ. Organs and/or tissues that aretransplantedwithin the same person's body are calledauto grafts. Transplants that are recently performed between two subjects of the same species are calledallografts. Allografts can either be from a living or cadaveric source Organs that can be transplanted are the heart, kidneys, liver, lungs, pancreas, intestine, and thymus. The kidneys are the most commonlytransplanted organs, followed by the liver and then the heart. The main function of the MHC antigens is peptide presentation to the immune system to help distinguish self from non-self. These antigens are called HLA (human leukocyte antigens). They consists of three regions: class I (HLA-A,B,Cw), class II (HLA-DR,DQ,DP) and class III (no HLA genes)

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American society of Transplantation;American Society of Transplant Surgeons: ASTS; Patient associations. Donation and transplantation;American Society of Gene & Cell Therapy ASGCT;Gene Therapy Societies and Patient Organizations - Gene Therapy Net.

Track 16:Cytogenetics

Cytogeneticsis a branch ofgeneticsthat is concerned withstudy of the structure and function of the cell, especially thechromosomes. It includes routine analysis of G-banded chromosomes, othercytogenetic banding techniques, as well as molecular Cytogenetics such as fluorescent in suitable hybridization FISH and comparativegenomic hybridization.

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Molecular Biology 2016

Molecular Biology 2016 Report

2ndWorld Bio Summit & Molecular Biology Expowas organized during October 10-12, 2016 at Dubai, UAE. The conference was marked with the attendance ofEditorial Board Members of supporting journals, Scientists, young and brilliant researchers, business delegates and talented student communities representing more than 25 countries, who made this conference fruitful and productive.

This conference was based on the theme Recent advances in Bio Science which included the following scientific tracks:

Molecular Biology

Microbiology

Analytical Molecular Biology

Bioinformatics

Biochemistry and Molecular Biology

Molecular Biology and Biotechnology

Cancer Molecular Biology

Computational Biology

Molecular Biology of the Cell

Molecular biology of the cardiovascular system

Molecular Biology in Cellular Pathology

Molecular Biology of Diabetes

Molecular Biology and Genetic Engineering

Enzymology and Molecular Biology

Molecular Biology of the Gene

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Molecular Genetics - Cell and Gene Therapy Conferences

Columbus Business First

Nationwide Children's spins out fourth gene therapy company this one is staying in Central Ohio Columbus Business First Nationwide Children's Hospital has spun out its fourth gene therapy startup to result from decades of research and millions in investment in manufacturing equipment and commercialization personnel.

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Nationwide Children's spins out fourth gene therapy company this one is staying in Central Ohio - Columbus Business First

Without RSRT championing gene therapy for the treatment of Rett Syndrome, we would not be where we are ... Brian Kaspar, CSO of AveXis

Trumbull, CT (PRWEB) June 08, 2017

The Rett Syndrome Research Trust (RSRT) is excited to announce that AveXis, Inc., has committed to advance a gene therapy candidate for Rett Syndrome into human clinical trials. AveXis, a pioneering clinical-stage gene therapy company, has already advanced a transformative gene therapy treatment for spinal muscular atrophy Type 1, a devastating disease that strikes infants and kills in toddlerhood. Remarkably, in a Phase 1 clinical trial, infants treated with the gene therapy achieved developmental milestones, including the ability to sit unassisted, talk and walk in some cases, achievements never seen in untreated babies with the disease. It is our shared belief that gene therapy, the introduction of healthy MECP2 genes to compensate for the mutated ones, may also produce benefits for individuals with Rett Syndrome.

This milestone represents the culmination of research originating in 2010 when RSRT funded the collaboration between Dr. Gail Mandel and Dr. Brian Kaspar, the now scientific founder and Chief Scientific Officer of AveXis, to explore the potential of gene therapy for the treatment of Rett Syndrome. Encouraged by the results of that collaboration, in 2014 RSRT conceived of, recruited the scientists for and funded the Gene Therapy Consortium to definitively evaluate the feasibility of gene therapy as a treatment for Rett Syndrome.

The Consortium scientists, Stuart Cobb, Steve Gray, Brian Kaspar and Gail Mandel, exceeded our expectations by developing a gene therapy product candidate with impressive efficacy, safety and delivery characteristics. Importantly, therapeutic benefits in the mouse models are much greater than that of any drug ever tested for Rett Syndrome.

Adrian Birds 2007 reversal study showing that symptoms in mice can be dramatically reversed suggests that a diagnosis of Rett need not translate to lifelong disability. While gene therapy seems an obvious approach for any of the numerous single gene disorders, the quality of the data generated by the Consortium was an essential factor in AveXis decision to prioritize development of a gene therapy for Rett Syndrome.

RSRT recognized early on that the gene therapy approach would move forward only if we aggressively championed it. We brought together scientists with strong track records of success and provided them with the financial support and infrastructure to work effectively and efficiently. Our strategy worked and I am thrilled that AveXis, a leader in gene therapy, is now taking this work forward at an industry scale. I am beyond grateful to the many Rett families who believe in our vision and to the generosity of their networks that make our vital work possible, said Monica Coenraads, Executive Director of RSRT and mother to a young woman with Rett Syndrome.

As was made clear in RSRTs recently launched strategic research plan, Roadmap to a Cure, our mission is a cure for Rett Syndrome. Todays announcement is a huge step forward towards our goal. By addressing the core cause of Rett, gene therapy has the potential to be a life-changer. We have complete confidence in the expertise and leadership of the AveXis team and will continue to provide our full support as this program moves forward, said Randall Carpenter, Chief Scientific Officer, RSRT.

Without RSRT championing gene therapy for the treatment of Rett Syndrome, we would not be where we are preclinically in our understanding of the potential for gene therapy to treat this devastating disease, said Brian Kaspar, Chief Scientific Officer of AveXis. Bringing our expertise and focus on rare monogenic diseases, we are excited by the possibility that gene therapy may address the needs of individuals with Rett Syndrome.

About the Rett Syndrome Research Trust The Rett Syndrome Research Trust (RSRT) is a nonprofit organization with a highly personal and urgent mission: a cure for Rett Syndrome and related MECP2 disorders. In March of 2017 RSRT announced Roadmap to a Cure, a three-year, $33 million strategic research plan. The plan prioritizes four curative approaches with gene therapy as our lead program. RSRT operates at the nexus of global scientific activity enabling advances in knowledge and driving innovative research through constant engagement with academic scientists, clinicians, industry, investors and affected families. Since 2008, RSRT has awarded $42 million to research. To learn more, please visit http://www.reverserett.org.

About AveXis, Inc. AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The companys initial proprietary gene therapy candidate, AVXS-101, recently completed a Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit http://www.avexis.com.

About Rett Syndrome Rett Syndrome is a genetic neurological disorder that almost exclusively affects girls. It strikes randomly, typically at the age of 12 to 18 months, and is caused by random mutations of the MECP2 gene on the X chromosome. Rett Syndrome is devastating as it deprives young children of speech, hand use, normal movement often including the ability to walk. As the children enter childhood the disorder brings anxiety, seizures, tremors, breathing difficulties, severe gastrointestinal issues. While their bodies suffer, it is believed that their cognitive abilities remain largely intact. Although most children survive to adulthood, they require total round-the-clock care.

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AveXis to Advance Gene Therapy Program Initiated by the Rett Syndrome Research Trust - PR Web (press release)

Posted on June 6, 2017, 6 a.m. in Allergy Gene Therapy Immune System

Using gene therapy, scientists have been able to 'turn-off' the immune response which causes allergic reaction in animals.

It might soon be possible for a single treatment to provide life-long protection against harsh allergies including asthma. An immunology research team at The University of Queensland led by Professor Ray Steptoe has figured out how to disable the immune response that triggers allergic reactions. The research team operates out of the university's Diamantina Institute. Professor Steptoe's lab is situated at the Translational Research Institute. The research was funded by the National Health and Medical Research Council and the Asthma Foundation. The research team's findings are published in JCI Insight.

The Basics of Allergies and Asthma

When an individual has an allergy or a flare-up of asthma, the symptoms he experiences stem from immune cell reactions to proteins within the allergen. Allergies and asthma recur over and over again as the immune cells, referred to as T-cells, gradually create a type of immune memory. As a result, they resist treatments. Steptoe and his research team are now capable of wiping the memories of T-cells in animals. They have successfully done so with gene therapy that desensitizes the immune system to allow for the tolerance of pain.

About the Breakthrough

Steptoe's research team made use of an experimental asthma allergen. They took blood stem cells, inserted a gene that regulates the allergen protein and put it into the recipient. These engineered cells generated new blood cells. The protein is expressed in these new blood cells. Specific immune cells are targeted in order for the allergic response to be turned off.

The experimental asthma allergen worked so effectively that it is possiblethe research could be used to treat those who suffer from traditional allergies to foods. Examples include allergies to nuts, shellfish, bee venom and an array of other substances. Professor Steptoe indicates the findings will soon be subjected to additional pre-clinical investigation. The next step is to replicate the results with human cells in a lab setting.

The Goal of Gene Therapy in the Context of Allergies

Professor Steptoe states the end goal is to make use of single injected gene therapy rather than repeated short-term treatments that attempt to reduce allergy symptoms. Such short-term treatments are successful in some instances and unsuccessful in others.

Professor Steptoe's team has not reached the point where gene therapy is as straightforward as receiving a flu jab yet his group is hard at work on making it as simple and safe as possible. Their aim is for gene therapy to be used on an extensive cross-section of those plagued by allergies and asthma as well as those who endure potentially deadly food allergies. It is possible that a completely safe one-off style gene therapy treatment for traditional allergies, asthma, and food allergies will be available in the near future.

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Allergy Breakthrough with Gene Therapy - Anti Aging News

Doctors are reporting unprecedented success from a new cell and gene therapy for multiple myeloma, a blood cancer that's on the rise. Although it's early and the study is small 35 people every patient responded and all but two were in some level of remission within two months.

In a second study of nearly two dozen patients, everyone above a certain dose responded.

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Experts at an American Society of Clinical Oncology conference in Chicago, where the results were announced Monday, say it's a first for multiple myeloma and rare for any cancer treatment to have such success.

Chemotherapy helps 10 to 30 percent of patients; immune system drugs, 35 to 40 percent at best, and some gene-targeting drugs, 70 to 80 percent, "but you don't get to 100," said Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society.

"These are impressive results" but time will tell if they last, he said.

Multiple myeloma affects plasma cells, which make antibodies to fight infection. More than 30,000 cases occur each year in the United States, and more than 115,000 worldwide. It's the second fastest growing cancer for men and the third for women, rising 2 to 3 percent per year, according to the National Cancer Institute. About 60,000 to 70,000 Americans have it now.

Nine new drugs have been approved for it since 2000 but they're not cures; only about half of U.S. patients live five years after diagnosis.

With cell therapy, "I can't say we may get a cure but at least we bring hope of that possibility," said Dr. Frank Fan. He is chief scientific officer of Nanjing Legend Biotech, a Chinese company that tested the treatment with doctors at Xi'an Jiaotong University.

The treatment, called CAR-T therapy, involves filtering a patient's blood to remove immune system soldiers called T cells. These are altered in a lab to contain a gene that targets cancer and then given back to the patient intravenously.

Doctors call it a "living drug"- a one-time treatment to permanently alter cells that multiply in the body into an army to fight cancer. It's shown promise against some leukemias and lymphomas, but this is a new type being tried for multiple myeloma, in patients whose cancer worsened despite many other treatments.

In the Chinese study, 19 of 35 patients are long enough past treatment to judge whether they are in complete remission, and 14 are. The other five had at least a partial remission, with their cancer greatly diminished. Some are more than a year past treatment with no sign of disease.

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Doctors in London say one-year-old Layla Richards is thriving after receiving an experimental gene therapy treatment for leukemia. CBS News' Tin...

Most patients had a group of side effects common with this treatment, including fever, low blood pressure and trouble breathing. Only two cases were severe and all were treatable and temporary, doctors said.

The second study was done in the U.S. by Bluebird Bio and Celgene, using a cell treatment developed by the National Cancer Institute. It tested four different dose levels of cells in a total of 21 patients. Eighteen are long enough from treatment to judge effectiveness, and all 15 who got an adequate amount of cells had a response. Four have reached full remission so far, and some are more than a year past treatment.

The results are "very remarkable" not just for how many responded but how well, said Dr. Kenneth Anderson of Dana-Farber Cancer Institute in Boston.

"We need to be looking for how long these cells persist" and keep the cancer under control, he said.

Dr. Carl June, a University of Pennsylvania researcher who received the conference's top science award for his early work on CAR-T therapy, said "it's very rare" to see everyone respond to a treatment. His lab also had this happen - all 22 children testing a new version of CAR-T for leukemia responded, his colleagues reported at the conference.

"The first patients we treated in 2010 haven't relapsed," June said.

Dr. Michael Sabel of the University of Michigan called the treatment "revolutionary."

"This is really the epitome of personalized medicine," extending immune therapy to more types of patients, he said.

Legend Biotech plans to continue the study in up to 100 people in China and plans a study in the U.S. early next year. The treatment is expected to cost $200,000 to $300,000, and "who's going to pay for that is a big issue," Fan said.

"The manufacturing process is very expensive and you can't scale up. It's individualized. You cannot make a batch" as is done with a drug, he said.

Nick Leschly, Bluebird's chief executive, said the next phase of his company's study will test what seems the ideal dose in 20 more people.

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Blood cancer treatment called "revolutionary" after all study patients responded - CBS News

On May 12, Sam and Taylor Sabky received heartbreaking news: Their son, Purnell, was diagnosed with Niemann-Pick TypeA, a rare and fatalgenetic disease with no treatment options. Most children with Niemann-Pick Type A die in early infancy.

When we left that appointment, essentially we left with no hope, Sam said. It was kind of a death sentence.

But after learning that research into treatment options is further along than they initially thought and that the biggest obstacle standing in the way is funding the Sabkys decided to create a GoFundMe, with 100 percent of the proceeds going toward funding research into gene therapy, the most promising treatment option for Niemann-Pick Type A.

The campaign went viral, and theyve managed to raise more than $314,000in 10 days, with agoal of $750,000 raised by the end of June. Now, theyre not feeling hopeless anymore.

We have so many people working on his behalf, on our behalf, in many unexpected places, and were so appreciative to have the support of everyone and the response that weve received, Taylor said. I mean, it really makes this feel like its possible and gives us hope and renews your faith in humanity.

Niemann-Pick Type A is a lysosomal storage disease, a type of disease that occurs when toxic materials build up in the cells, as a result of enzyme deficiencies. With Niemann-Pick Type A, symptoms include enlargement of the liver and spleen, and failure to gain weight.

Typically, children diagnosed with Niemann-Pick Type A have a plateau period, which begins around 8or 9months and lasts until theyare between 18-20 months old. During this time, children appear to be interacting normally and possibly even learning some new skills.

Often, after that time, theyll have progressive neurological deterioration, where they really have a hard time interacting, said Dr. Melissa Wasserstein, a Niemann-Pick specialistat the Childrens Hospital at Montefiore in New York City.They lose their focus. Theyre progressively weaker.

Most children with Niemann-Pick Type A die between 2and 3 years of age, according to Wasserstein.

The Sabky family first learned of potential treatment options when they connected with Steve Lafoon. Lafoon is president of the Wylder Nation Foundation, an Arizona-based organization dedicated to accelerating the discovery and development of treatment options for Niemann-Pick Type A.

Lafoons son, Wylder, was diagnosed with Niemann-Pick Type A in 2009 and passed away in 2012. Lafoon said it was difficult for him to grasp not having any treatment options for Wylder, and thats part of what inspired him to start the foundation.

When youre given a diagnosis, and the only option youre told is to go home and enjoy the short amount of time youre going to have, its just something I had a really hard time getting my head around, not having any options, hesaid. So that was our whole premise. We feel that everybody deserves some sort of option moving forward.

According to Lafoon, the most promising treatment option is gene therapy, which is under research at University of California, San Fransiscos Bankiewicz Lab.In order to complete their research and send the treatment option to a clinical trial, the lab has to first build a clinical vector.

[The vector] is basically just a harmless virus that doses a healthy gene to Purnells brain, pretty much replacing the malfunctioning one, Sam said.

But funding is preventing this step from moving forward. The Wylder Nation Foundation has been providing funding to the lab, but researchers need more in order to build the vector. All of the donations to the Sabkys GoFundMe page will go to the Wylder Nation Foundation, which will use it to continue financing the labs research. And since the treatment still has to be approved by the FDA, timing, Sam says, iscritical.

The timing really could line up, which is just so fantastic, but its really a matter of, as soon as the funds are available for that vector, its going to go right to the lab, and theyre going to order and build the vector, and then we can get started, Sam said.

The Sabkys have been overwhelmed by the support theyve received a wealth of different sources. Theyve found strength within the Niemann-Pick Type A community, who have shared Purnells story, as well as from Taylors students at Boston International High School in Dorchester, who have made signs and posters with words of encouragement and inspirational messages for Purnell.

We have everyone in our corner rooting on a cure for this disease, Taylor said.

In the meantime, theyre enjoying every second they have with Purnell, planning vacations and attending weddings, to create lasting memories with their son.

The only thing that has really changed is that when we sit down, were really soaking in the time and being so much more appreciative of him, of the time we have with him, Sam said. Something like this puts stuff in perspective, so we have a lot more gratitude.

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A Roslindale family has raised over $310000 to fund life-saving research for their 1-year-old son - Boston.com

The team hopes to develop a single, injected, gene therapy treatment that could eliminate many severe allergic responses (Credit: University of Queensland)

Scientists may be one step closer to discovering a way to genetically "turn off" allergic responses with a single injection. A team of researchers at the University of Queensland has developed a new process that has successfully silenced a severe allergic response in mice, using blood stem cells engineered with a gene that can target specific immune cells.

The big challenge previous allergy researchers faced was that immune cells, known as T-cells, tended to develop a form of "memory" so that once someone developed an immune response to an allergen, it would easily recur upon future contact. The key was finding a way to erase that "memory" response to the protein in the allergen causing the immune reaction.

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"We take blood stem cells, insert a gene which regulates the allergen protein and we put that into the recipient," says Professor Ray Steptoe, explaining the new process developed by his team at The University of Queensland. "Those engineered cells produce new blood cells that express the protein and target specific immune cells, 'turning off' the allergic response."

The team's initial clinical investigations looked at an experimental asthma allergen, with the new process found to successfully terminate established allergic responses in sensitized laboratory mice. While the initial research has focused on a very specific asthma allergen, Professor Steptoe believes the process could be applied to many other severe allergic responses, such as peanuts, bee venom and shell fish.

The long-term goal of the research would be to develop a therapy that could cure specific allergies with a single injection, much like a vaccine.

"We haven't quite got it to the point where it's as simple as getting a flu jab," says Professor Steptoe, "so we are working on making it simpler and safer so it could be used across a wide cross-section of affected individuals."

The team is realistic about the time it will take before this discovery results in practical benefits for allergy sufferers, with at least five years more laboratory work needed before even human trials can be conducted. But this new discovery could mean that, within 10 or 15 years, asthma and other lethal allergic responses might be eliminated with a single, one-time treatment.

The findings were recent published in the journal JCI Insight.

Watch Professor Ray Steptoe from The University of Queensland discuss his team's findings in the video below.

Source: The University of Queensland

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Gene therapy could wipe immune memory and "turn off" severe allergies - New Atlas