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Archive for the ‘Gene Therapy Research’ Category

Is gene therapy research for single-gene diseases at risk under Trumpcare? – Genetic Literacy Project

[When]the House of Representatives passed the American Health Care Act of 2017,DNA Science addressed the possibility of the AHCA forcing pregnant womento carry doomed fetuses to term, the discussion now in the hands of 13 senators[Now]I fear for the treatments for single-gene conditions,both the short-term and available protein-based ones as well as the not-yet-approved gene therapies.

CNN.comonce told the remarkable story of recent college grad Ryan Dant., [who was diagnosed with] a form of mucopolysaccharidosis (MPS) [at age 3]Ryan wasnt expected to survive beyond age 10, but entered a clinical trial for an enzyme replacement therapy (ERT).It won FDA approval in 2003, [but it is very expensive].

The high cost of lifelong frequent infusions or injections of ERT is why the forever fix of a gene therapy is an attractive alternative, even if a booster or two becomes necessary. Gene therapy delivers the DNA instructions for making the missing enzyme. Another reason to seek gene therapy (or editing) is that enzyme infusions dont reach the brain.

Theoretically, gene therapy should be more economical than ERT, once research costs have been recoupedYet the first FDA approval for a gene therapy has yet to happen.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Will Short Term and Long Term Treatments for Single-Gene Diseases Survive?

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Is gene therapy research for single-gene diseases at risk under Trumpcare? – Genetic Literacy Project

Dr. Xinping Song’s 3D Prostate Treatment Investigates Vectors for … – PR Newswire (press release)

XIANGTAN, Hunan, May 25, 2017 /PRNewswire/ — Incidence of prostate cancer in China is on the rise, cells in the male prostate are overly replicating, creating cancerous growths that leave the patient in severe pain, constant feelings of urination, and a decrease in life expectancy if not treated. Current treatments for prostate cancer include invasive surgeries, radiation, and oral drugs, however, these western remedies often leave patients with adverse side effects (such as hair loss, nausea, weakness, and emotional damage). Medical researchers are now looking for new cancer treatment to treat prostate and other forms of cancer. The 3D prostate treatment based out of the 3D Urology and Prostate Clinic in China is taking this challenge with their research on gene therapy and how genes created can be transferred to the target region. Gene therapy allows for certain genes to be turned on/off creating an enzymatic cascade that can lead to different outcomes, in cancer researchers hope that gene therapy can stop cell growth and induce apoptosis of tumor cells.

Scientists have created novel “suicide genes” that induce apoptosis in vivo. The human body is more complex than test organisms so getting genes to the right location requires a lot of work, simply swallowing a pill will have the suicides genes be destroyed by hydrochloric acid and digestive enzymes in the stomach. To mediate this issue medical researchers are now using injections of suicide genes coupled to vectors. Current day vectors for gene therapy are done through viruses that are tacked with the lab created genes, however, this process leaves patients susceptible to attacks by the virus. Dr. Xinping Song’s 3D prostate clinic has been exploring alternative and more natural methods for gene therapy transfer. Vectors investigated by the Dr. Xinping Song include nanoparticles and cationic liposomes, chitosan, and alkaline polysaccharides, their findings showed that these vectors were all highly specific to cancerous tumors and help get the genes to undergo apoptosis into the tumor cells.

Medical research must continue to experiment with different genes and vectors to push to cure prostate and other forms of cancer. Gene therapy can treat certain forms of cancer naturally with no side effects.

Contact:

Alisa Wang

86-186-7321-6429

prostatecure3d(at)gmail(dot)com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/dr-xinping-songs-3d-prostate-treatment-investigates-vectors-for-gene-therapy-in-prostate-cancer-300463869.html

SOURCE Dr. Song’s 3D Prostate and 3D Prostatitis Clinic

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Dr. Xinping Song’s 3D Prostate Treatment Investigates Vectors for … – PR Newswire (press release)

Keck School of Medicine of USC Receives $2.2 Million NIH Grant to Fund Research on Healing Difficult Bone Injuries – Newswise (press release)

Newswise LOS ANGELES Jay R. Lieberman, MD, chair and professor of orthopedic surgery at the Keck School of Medicine of the University of Southern California has received a five-year, $2.2 million grant from the National Institutes of Healths National Institute of Arthritis and Musculoskeletal and Skin Diseases to research gene therapy to enhance repair of extensive bone injuries. Examples of these types of injuries include fractures with extensive bone loss, non-healing fractures, failed spinal fusion and revision of total joint replacement.

Lieberman will genetically manipulate human bone marrow cells to overproduce bone morphogenetic protein (BMP), which is a protein that spurs progenitor cells to produce bone.

There are a number of bone injuries that are very difficult to repair and lack satisfactory solutions, Lieberman says. My goal with this grant is to determine whether genetically modifying human bone marrow cells to overproduce BMP will help heal large bone defects in an animal model and, ultimately, provide a better alternative for repairs in humans.

Liebermans study will determine the efficacy and safety of the gene therapy as well as establish a cellular dose of the genetically manipulated cells that can be scaled up for potential use in humans.

An abstract of the grant, 2R01AR057076-06A1, is available on the NIH RePORTER website.

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ABOUT THE KECK SCHOOL OF MEDICINE OF USC

Founded in 1885, the Keck School of Medicine of USC is among the nations leaders in innovative patient care, scientific discovery, education, and community service. It is part of Keck Medicine of USC, the University of Southern California’s medical enterprise, one of only two university-owned academic medical centers in the Los Angeles area. This includes the Keck Medical Center of USC, composed of the Keck Hospital of USC and the USC Norris Cancer Hospital. The two world-class, USC-owned hospitals are staffed by more than 500 physicians who are faculty at the Keck School. The school today has approximately 1,650 full-time faculty members and voluntary faculty of more than 2,400 physicians. These faculty direct the education of approximately 700 medical students and 1,000 students pursuing graduate and post-graduate degrees. The school trains more than 900 resident physicians in more than 50 specialty or subspecialty programs and is the largest educator of physicians practicing in Southern California. Together, the school’s faculty and residents serve more than 1.5 million patients each year at Keck Hospital of USC and USC Norris Cancer Hospital, as well as USC-affiliated hospitals Childrens Hospital Los Angeles and Los Angeles County + USC Medical Center. Keck School faculty also conduct research and teach at several research centers and institutes, including the USC Norris Comprehensive Cancer Center, the Zilkha Neurogenetic Institute, the Eli and Edythe Broad Center for Stem Cell Research and Regenerative Medicine at USC, the USC Cardiovascular Thoracic Institute, the USC Roski Eye Institute and the USC Institute of Urology.

In 2017, U.S. News & World Report ranked Keck School of Medicine among the Top 40 medical schools in the country. For more information, go to keck.usc.edu.

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This press release references support by the National Institutes of Health under award number 2R01AR057076-06A1 ($2,284,028 over five years). One hundred percent of the projects funding will be federally funded.

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Keck School of Medicine of USC Receives $2.2 Million NIH Grant to Fund Research on Healing Difficult Bone Injuries – Newswise (press release)

A New Potential ALS Gene Therapy Delivers A Key Milestone – ALS Research Forum

About 1 out of 5 inherited cases of ALS occur due to mutations in SOD1. How these genetic changes contribute to ALS remains unclear. But according to a growing number of studies, these mutations result in the misfolding and aggregation of this metabolic enzyme, contributing to motor neuron toxicity by multiple mechanisms (see Taylor et al., 2016).

Silence, please. Scientists are developing gene therapy strategies for SOD1 ALS that aim to reduce levels of mutant SOD1 in key tissues affected by the disease including the brain and spinal cord. [Image: Rice University under a CC BY 4.0 license.]

A growing number of researchers are developing potential treatment strategies that aim to reduce levels of mutant SOD1 in the CNS in hopes to slow or stop the progression of the disease. One approach, which involves gene therapy, suppresses expression of the SOD1 gene through RNA silencing-based mechanisms. This strategy, according to a 2016 study led by University of Massachusetts Medical Schools Christian Mueller, can extend the lifespan of pre-onset adult SOD1 mice by up to 20% upon intrathecal injection (Borel et al., 2016).

Now, Martine Barkats and Maria Grazia Biferi of the Institute of Myology in Paris, France introduce a new gene therapy approach that increases survival of pre-symptomatic adult SOD1 G93A mice by more than 50%. An independent analysis, led by ALS Therapy Development Institutes Fernando Vieira and commissioned by Prize4Life, confirmed the teams results.

The Institute of Myology team received the Prize4Life $1.0 million USD Avi Kremer ALS Treatment Prize in recognition of reaching this key milestone. This is the best efficacy [observed] in a SOD1 mouse, said Nicole Szlezk, Chairman of Prize4Lifes Board of Directors in the US.

Maria Grazia Biferi unveiled the potential treatment strategy on April 25, 2017 at the ALS Association Drug Company Working Group, held during the 69th Annual Meeting of the American Academy of Neurology.

The approach builds on a previous strategy developed by Barkats team, using a related gene therapy delivery vehicle, for spinal muscular atrophy. A similar strategy, known as ChariSMA (AVXS-101), is now at the phase 1 stage and according to interim phase I results appears promising (see December 2015 news).

This is no longer an academic exercise, said Lucie Bruijn, Chief Scientist of the ALS Association. The current clinical trials in SMA confirm that this approach can succeed.

Going viral

In 2007, Martine Barkats team discovered that the recombinant adeno-associated virus AAV9 could penetrate the blood-brain barrier, opening the door to the development of gene therapies for motor neuron diseases. (see December 2008 news; Duque et al., 2009; Foust et al., 2009).

Every dog has its day. Researchers at Tufts University School of Medicine are now evaluating a potential gene therapy for SOD1-linked ALS in dogs. The approach aims to help dogs with a naturally occurring form of the disease known as canine degenerative myelopathy (DM), a late-onset disease first recognized in the 1970s in German Shepards. The trial is a key step toward developing a treatment for ALS, because dogs are larger and the disease is naturally occurring. [Image: Handicapped Pets. CC BY-SA 2.0 license.]

Building on these advances, research teams led by Nationwide Childrens Hospital Brian Kaspar in Ohio and University of Massachusetts Medical Centers Robert Brown began to develop potential therapies for SOD1 ALS. One of these strategies, being developed by Robert Brown and Christian Mueller at the University of Massachusetts Medical School, uses an artificial microRNA to reduce levels of mutant SOD1 synthesis in the brain and spinal cord. The approach is currently being evaluated at the preclinical stage in dogs with degenerative myelopathy (DM), a naturally occurring form of the disease. The clinical trial, being led by Tufts University School of Medicines Dominik Faissler in Massachusetts, launched in December 2016 and is ongoing.

The strategy builds in part, on previous studies led by Martine Barkats which found that AAV10 is more efficient than AAV9 in delivering genes into motor neurons in the spinal cord at lower doses, critical in developing a treatment for a disease (Tanguy et al., 2015). The approach, according to studies led by University of Californias Krystof S. Bankiewicz in San Francisco and University of Massachusetts Medical Centers Christian Mueller can be delivered efficiently intrathecally into motor neurons, at least in non-human primates.

Meanwhile, California Institute of Technologys Ben Deverman and Viviana Gradinaru used Cre-recombination-based AAV targeted evolution (CREATE), to develop novel gene therapy delivery vehicles that penetrate the blood brain barrier, to treat disorders of the central nervous system, including ALS. The vectors, which include AAV-PHP.B, can deliver genes into the motor cortex in the brain and the spinal cord at about 40 times greater efficiency compared to AAV9 upon intravenous injection (Deverman et al., 2016).

The delivery approach is now licensed to Cambridge startup Voyager Therapeutics in Massachusetts, which is also developing a gene therapy for SOD1 ALS. Voyager Therapeutics hopes to file an IND for their potential treatment strategy for ALS, known as VY-SOD101, at the end of 2017.

A key question is whether SOD1 is needed to reduce free radical levels that arise in key tissues affected by the disease. Therefore, a growing number of research teams are developing erase and replace strategies which aim to reduce mutant SOD1 while at the same time, produce the wild-type enzyme to help detoxify key tissues affected by ALS, including the brain, spinal cord and muscles.

A one, two punch?

Engineering a new approach. Researchers at University of Massachusetts Medical School are developing a CRISPR-Cas9-based gene therapy for SOD1-linked ALS that aims to permanently suppress mutant SOD1 synthesis. Meanwhile, Duke Universitys Charles Gersbach is developing a epigenomic editing-based strategy, also presented at AAN 2017, that may be of benefit to a wide range of diseases including the most common forms of ALS, C9orf72-linked disease (see January 2017 conference news; Thakore et al., 2015). [Courtesy of Ran et al., 2013, Nature Publishing Group.]

With the advent of new gene silencing technologies, research teams began to take another look at their gene therapy approaches and began to modify them in hopes to optimize their strategies to combat SOD1-linked disease. Robert Brown turned to CRISPR/Cas9 technologies in hopes to stop further mutant SOD1 synthesis. Their approach, unveiled at AAN 2017, uses imprecise CAS9-based editing to introduce indels in the SOD1 gene. The key obstacle, according to preliminary results presented by University of Massachusetts Medical Schools Zachary Taylor, is delivering sufficient Cas9 to modify the mutant SOD1 gene within motor neurons and glia in the central nervous system. Preclinical studies remain ongoing.

Across the globe, Martine Barkats and Maria Grazia Biferi, are developing a strategy using a novel gene silencing AAV10-based approach to tackle SOD1 ALS. The strategy is now being optimized and is at the preclinical stage. The approach involves the injection of the potential therapy into the blood and the brain. The reason, according to Biferi is to ensure that levels of the misfolded enzyme are reduced in key tissues outside the CNS including skeletal muscle.

This is important, explains Biferi, because ALS is beginning to be considered a multisystemic disease.

Now, the researchers are developing a similar strategy to tackle C9orf72 ALS, the most common form of the disease identified to date.

TARgeting ALS

A detox program for the CNS? Researchers at MeiraGTx in New York, in collaboration with Ronald Klein and Gregory Petsko, are developing a gene therapy strategy that aims to reduce the toxicity of TDP-43 by turning up nonsense-mediated mRNA decay. [Image: Emw, Wikimedia Commons.]

Meanwhile, Louisiana State Universitys Ronald Klein is setting his sights on helping to develop a gene therapy that targets more than 95% of cases of the disease. The approach, based on previous studies led by Gregory Petsko in New York, now at Weill Cornell Medical College in New York and Sami Barmada, now at the University of Michigan, aims to reduce cytoplasmic TDP43-mediated motor neuron toxicity by turning up nonsense-mediated mRNA decay. The gene therapy-based strategy increases levels of hUPF1, a key regulator of this process (see June 2015 news; Ju et al., 2011; Barmada et al., 2015).

The approach, according to a study from Kleins team, prolongs motor function at least for 8 weeks in a rat model of the disease. The study found that the potential therapy, when administered at day 1, preserved strength in the forelimbs of a rat model of ALS (see June 2015 news; Jackson et al., 2015).

It is really amazing that [increasing] hUPF has this really specific protective action against TDP-43, says Klein. We keep seeing it again and again.

How this strategy may mitigate TDP-43-mediated motor neuron toxicity remains unclear. The approach is one of at least two that aims to target TDP-43 buildup in the cytoplasm of motor neurons (see April 2017 news; Becker et al., 2017).

The strategy, now licensed to the New York biotech startup MeiraGTx, is at the preclinical stage. Evaluation of the approach in adult rat models of ALS is ongoing.

References

Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabreja GC, Song L, Su Q, Gao GP, Elmallah MK, Brown RH Jr, Mueller C. Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates. Hum Gene Ther. 2016 Jan;27(1):19-31. doi: 10.1089/hum.2015.122. [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].

Tanguy Y, Biferi MG, Besse A, Astord S, Cohen-Tannoudji M, Marais T, Barkats M. Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice. Front Mol Neurosci. 2015 Jul 28;8:36. doi: 10.3389/fnmol.2015.00036. eCollection 2015. [PubMed].

Barmada SJ, Ju S, Arjun A, Batarse A, Archbold HC, Peisach D, Li X, Zhang Y, Tank EM, Qiu H, Huang EJ, Ringe D, Petsko GA, Finkbeiner S. Amelioration of toxicity in neuronal models of amyotrophic lateral sclerosis by hUPF1. Proc Natl Acad Sci U S A. 2015 Jun 23;112(25):7821-6. [PubMed].

Jackson KL, Dayton RD, Orchard EA, Ju S, Ringe D, Petsko GA, Maquat LE, Klein RL. Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis. Gene Ther. 2015 Jan;22(1):20-8. [PubMed].

Ju S, Tardiff DF, Han H, Divya K, Zhong Q, Maquat LE, Bosco DA, Hayward LJ, Brown RH Jr, Lindquist S, Ringe D, Petsko GA. A yeast model of FUS/TLS-dependent cytotoxicity. PLoS Biol. 2011 Apr;9(4):e1001052. [PubMed].

Becker LA, Huang B, Bieri G, Ma R, Knowles DA, Jafar-Nejad P, Messing J, Kim HJ, Soriano A, Auburger G, Pulst SM, Taylor JP, Rigo F, Gitler AD. Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice. Nature. 2017 Apr 20;544(7650):367-371. [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 c9orf72 disease-als gene therapy SOD1 tdp-43 topic-preclinical topic-randd

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A New Potential ALS Gene Therapy Delivers A Key Milestone – ALS Research Forum

An Experimental Gene Therapy Uses Viruses to Stop Age-Related … – Futurism

In Brief Researchers from Johns Hopkins Medicine in Maryland have discovered a rather unusual way to treat a severe form of age-related blindness. They found a virus inserted into the retina can be used to halt or even reverse the disease. A Unique Treatment

They say you dont fight fire with fire. However, researchers from Johns Hopkins Medicine in Maryland have found that sometimes a virus may be the best weapon against a disease.Their studyhas been publishedin The Lancet

The researchers werelooking for ways to treat a particular type ofage-related macular degeneration (AMD)known as a wet AMD. Its a rare and more severe form of the disease,affecting just 10 percent of all AMD patients, and it causes new blood vessels to grow under the retina, which then leak blood and fluid into the eye, leading to vision problems.

The researchers knewthey could halt and even reverse the condition by suppressing an overactive protein called vascular endothelial growth factor (VEGF). Other researchers had been able to do it with monthly eye injections, but this team was hoping to do it with just one injection.

The best way they found to do this was by using a common cold-like virus called AAV2 as a carrier of gene that activates the production of a differentprotein,sFLT01, tocounter VEGF.

In a preliminary trial involving 19 men and women 50 years old and above, the researchers injected the patients with a form of AAV2that was genetically engineered to penetrate retinal cells and deposit the gene. After the virus deposited the gene, the cells began secreting sFLT01 which bound to VEGF and prevented it from stimulating leakage and growth of abnormal blood vessels, explained a Johns Hopkins press release.

The clinical trial showed promising results, with the condition of four of the patients improving dramatically after just one viral injection. Two others saw some reduction in the fluid build up, and the treatment didnt produce any side effects in any patients. Even at the highest dose, the treatment was quite safe. We found there were almost no adverse reactions in our patients, said researcher Peter Campochiaro.

Of the patients that didnt respond, the researchers discovered that five naturally produced antibodies that would attack the AAV2 virus, rendering it unable to complete its gene depositing mission. They think these antibodies could be prevalent throughout the population, making it difficult to determine how effecting the treatment would actually be.

Nevertheless, this research is a step in the right direction, especially with AMD expected to affect almost 5.44 million people in the U.S. by 2050. This preliminary study is a small but promising step towards a new approach that will not only reduce doctor visits and the anxiety and discomfort associated with repeated injections in the eye, but may improve long-term outcomes, Campochiaro said.

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An Experimental Gene Therapy Uses Viruses to Stop Age-Related … – Futurism

Gene therapy might someday help mend badly broken bones … – Health24

19 May 2017 Gene therapy might someday help mend badly broken bones This experimental method combines gene therapy, stem cells and ultrasound

Researchers have found that an experimental technique has healed large bone breaks in lab animals, researchers have found.

The technique is still far from becoming a reality, but, it repaired large bone gaps in the mini-pigs scientists studied. The hope, the researchers say, is to eventually help people with badly broken bones that won’t heal.

The results of the study were published in Science Translational Medicine.

When a bone sustains a simple fracture, it is usually able to self-repair with time (and a cast). However, severe fractures can leave large gaps in the bone that the self-healing process cannot bridge.

The aim of gene therapy

Health24 previously reported that the basic function of genes is to regulate the production of proteins required for the healthy working of cells. Thus, genetic defects manifest in either too little or too much of a protein being produced.

The aim of gene therapy is to replace the defective gene with a healthy one. The correct amount of proteins will be produced, and the disease will then be cured.

Geneticists are literally snipping defective pieces out of a strand of DNA with molecular scissors called CRISPR.

Bone grafting

Right now, the “gold standard” treatment for those fractures is bone grafting, said Dan Gazit, one of the senior researchers on the new study.

There, surgeons take bone tissue from elsewhere in the body or from a donor and use it to repair the damaged bone.

When bone grafting is done with a patient’s own tissue often taken from the pelvis that means additional surgery. And it can leave patients with prolonged pain and added risk of infection, Gazit said.

Newapproach

First, the researchers implanted a matrix of collagen a protein in bone into the gap between the two sides of a fractured bone.

That collagen then attracted the bone’s resident stem cells, and gave them a structure to settle into. Stem cells are early cells that develop into mature tissue, including bone.

Once those cells have populated the gap in the bone, the next step involves gene therapy. The researchers injected a mixture of “microbubbles” and genetic material for a bone-promoting protein, called BMP, into the injury site.

That spurred the stem cells to form new bone tissue, according to the report.

In this study, the tactic healed bone breaks in all of the lab animals the researchers treated, Gazit said. In untreated pigs, the breaks did not heal, the findings showed.

A sophisticated approach needed

“But there are problems with BMP,” said Dr Joseph Lane, an orthopaedic trauma surgeon at the Hospital for Special Surgery in New York City.

A central issue, he said, is that very high BMP doses may be needed, and side effects including infections and excess bone growth are common.

The new approach is “going in that direction”, said Lane, who was not involved in the research. The “beauty” of it, he said, is that it harnesses the bone-promoting effects of BMP in a more natural way.

A number of questions should be answered before human trials are done, according to Lane. For example, he said, future animal research should look at more complicated fractures. The bone injuries used in this study are relatively easy to heal, versus severe fractures, Lane said.

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The information provided does not constitute a diagnosis of your condition. You should consult a medical practitioner or other appropriate health care professional for a physical exmanication, diagnosis and formal advice. Health24 and the expert accept no responsibility or liability for any damage or personal harm you may suffer resulting from making use of this content.

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Gene therapy might someday help mend badly broken bones … – Health24

New gene therapy for vision loss is safe in humans, study suggests – Science Daily

New gene therapy for vision loss is safe in humans, study suggests
Science Daily
In a small and preliminary clinical trial, Johns Hopkins researchers and their collaborators have shown that an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving

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New gene therapy for vision loss is safe in humans, study suggests – Science Daily

Tiny bubbles and a bit of gene therapy heal major bone fractures in pigs – Science Magazine

By Robert F. ServiceMay. 17, 2017 , 2:30 PM

It takes more than a cast and a little time to heal many broken bones. Whether its a soldier wounded in battle, a car accident victim, or an elderly person who has fallen, bone damage can be so extensive that the bones never heal properly, leaving people crippled or with other severe problems. Now, researchers have combined ultrasound, stem cells, and gene therapy to stimulate robust bone repair. So far the work has only been performed in animals. But it has already been so successful that its expected to move quickly toward human clinical trials.

The new research has huge clinical significance, says David Kulber, who directs the Center for Plastic and Reconstructive Surgery at Cedars-Sinai Medical Center in Los Angeles, California, and who was not part of the study. The technology of being able to stimulate bone growth is really remarkable.

Its also one for which there is a glaring need. In the United States alone, some 100,000 people a year suffer from what is known as a nonunion fracture. In these cases, parts of a bone may be missing altogether or so badly splintered that the bone cant be reassembled. In such cases, doctors typically graft other bone into the site. Ideally this bone comes from the same personoften taken from the pelvis, a painful procedure that compounds a persons injuries and recovery time. When this isnt possible, physicians will turn to cadavers for the extra bone. But this bone must be sterilized before its implanted, robbing it of proteins and other signaling molecules that encourage its regrowth once transplanted, and lessening the chances of a full recovery.

Researchers have long tried to improve matters by growing new bone without use of a graft. To do so they typically first fill gaps in bone with a natural scaffolding material called collagen. This scaffolding encourages a persons own bone-forming stem cells, called mesenchymal stem cells (MSCs), to migrate into the area. The trouble is MSCs dont only differentiate into osteocytes, the bone-producing cells. They can also develop into either fat tissue cells or scar tissue.

Researchers have tried for years to steer MSCs into becoming osteocytes by exposing them to one or more bone morphogenetic proteins (BMPs), signaling molecules that trigger the cells to transform into bone-forming cells. But for this differentiation to occur, MSCs must be exposed to BMPs for up to a week. Yet if the BMPs are simply injected into the site of a fracture, they dissipate in just hours.

In an effort to produce a lasting BMP signal, researchers led by Dan Gazit, a regenerative medicine expert at Cedars-Sinai, as well as other groups, have previously turned to using viruses to introduce extra copies of BMP genes into MSCs so that the cells themselves will produce the proteins long enough to trigger their own differentiation. But success has been halting here, too.

Over the last several years, Gazits teamamong othershasdeveloped an alternative strategy for efficiently getting genes into MSCs without viruses. The researchers start by packing the wound with the usual collagen matrix and waiting for a couple of weeks for the stem cells to infiltrate the scaffold. They then create a solution containing numerous copies of their gene of interest alongside gas-filled micron-sized bubbles encased by a thin shell of fat molecules. After injecting this solution into the fracture site, they go over the area with an ultrasound wand, much as its done by obstetricians to check on the health of a fetus. The wands ultrasound pulses burst the microbubbles, briefly punching nano-sized holes in any adjacent stem cells, which allows the genes in the solution to enter.

In 2014, Gazit and his colleagues reported that they used this procedure to introduce nontherapeutic reporter genes into large fractures in animal models. But when they used the procedure to introduce genes for two different BMPsBMP-2 and BMP-7they detected some bone regrowth in the animals, but not enough to heal the fractures.

Gazits group has gotten better results by using the same approach to insert copies of the gene for BMP-6 into pigs that had been surgically given 1-centimeter gaps in a leg bone. After waiting 8 weeks, they found that the bone gap was closed and the leg fracture was healed in all of the treated animals. In fact, the procedure was so effective that the fractures healed as well as when bone grafts were carried out using bone from the same animal, the currently preferred treatment, they report today in Science Translational Medicine.

The results are just the type of thing we need to move this field forward, says Johnny Huard, an orthopedics researcher at the University of Texas Health Science Center in Houston. However, he notes, the pigs used in this study were all under 1 year in age. Younger animals, including people, tend to have far more MSCs than older ones, he says, yet large fractures are far more common in the elderly than the young. So Huard suggests that before the approach is ready for testing in people with bone fractures, it would be good to first see whether its equally successful in older animals.

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Tiny bubbles and a bit of gene therapy heal major bone fractures in pigs – Science Magazine

SENS Research Foundation Announces New Research Program on Somatic Gene Therapy With Buck Institute for … – Markets Insider

MOUNTAIN VIEW, CA–(Marketwired – May 15, 2017) – SENS Research Foundation (SRF) has launched a new research program focused on somatic gene therapy in collaboration with the Buck Institute for Research on Aging. Brian Kennedy, PhD, a leading expert on the biology of aging, will be running the project in his lab at the Buck.

Many potential treatments of age-related diseases require the addition of new genes to the genome of cells in the body, a technology known as somatic gene therapy. The technology has been hampered, up until now, by the inability to control where the gene is inserted. That lack of control resulted in a significant risk of insertion in a location that encourages the cell to become malignant.

SRF has devised a new method for inserting genes into a pre-defined location. In this program, this will be done as a two-step process, in which first CRISPR is used to create a “landing pad” for the gene, and then the gene is inserted using an enzyme that only recognizes the landing pad. SRF has created “maximally modifiable mice” that already have the landing pad, and this project will evaluate how well the insertion step works in different tissues.

“Somatic gene therapy has been a goal of medicine for decades. Being able to add new healthy genes will enable us to address treatments of such age-related diseases as atherosclerosis and macular degeneration. Our collaboration with SRF will substantially move us toward finding effective treatments to genetically based age-related diseases,” said Dr. Kennedy.

“Partnering with Brian Kennedy and the Buck enables SRF to continue towards our goal of achieving human clinical trials on rejuvenation biotechnologies in the next five years. Brian’s leadership in moving this technology into mammals is a huge step forward,” said Dr. Aubrey de Grey, CSO, SENS Research Foundation.

This research has been made possible through the generous support of the Forever Healthy Foundation and its founder Michael Greve, as well as the support of our other donors. The Forever Healthy Foundation is a private nonprofit initiative whose mission is to enable people to vastly extend their healthy lifespans and be part of the first generation to cure aging. In order to accelerate the development of therapies to bring aging under full medical control, the Forever Healthy Foundation directly supports cutting-edge research aimed at the molecular and cellular repair of damage caused by the aging process.

About SENS Research Foundation (SRF)SENS Research Foundation is a 501(c)(3) nonprofit that works to research, develop, and promote comprehensive regenerative medicine solutions for the diseases of aging. SRF is focused on a damage repair paradigm for treating the diseases of aging, which it advances through scientific research, advocacy, and education. SENS Research Foundation supports research projects at universities and institutes around the world with the goal of curing such age-related diseases as macular degeneration, heart disease, cancer, and Alzheimer’s disease. Educating the public and training researchers to support a growing regenerative medicine field are also major endeavors of the organization that are being accomplished though advocacy campaigns and educational programs. For more information, visit http://www.sens.org.

About Buck Institute for Research on AgingBuck Institute is the U.S.’s first independent research organization devoted to Geroscience — focused on the connection between normal aging and chronic disease. Based in Novato, California, the Buck is dedicated to extending “healthspan,” the healthy years of human life, and does so by utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and others focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimer’s and Parkinson’s, cancer, cardiovascular disease, macular degeneration, osteoporosis, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics, bioinformatics and stem cell technologies. For more information: http://www.thebuck.org.

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SENS Research Foundation Announces New Research Program on Somatic Gene Therapy With Buck Institute for … – Markets Insider

Sangamo Receives Fast Track Designation From The FDA For SB-525 Investigational Hemophilia A Gene Therapy – PR Newswire (press release)

SB-525 has already received Orphan Drug designation from the FDA. The FDA has cleared an Investigational New Drug application for this program, and a Phase 1/2 clinical trial evaluating SB-525 in adults with hemophilia A is expected to open and begin screening subjects for enrollment by the end of the second quarter 2017. Data from this study are expected in late 2017 or early 2018.

About Hemophilia A

Hemophilia A is a monogenic, rare bleeding disorder in which the blood does not clot normally. It is caused by mutations in the F8 gene which encodes Factor VIII clotting protein that helps the blood clot and stop bleeding when blood vessels are injured. Individuals with this mutation experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. According to the Centers for Disease Control and Prevention, hemophilia occurs in about one of every 5,000 male births, with an estimated 20,000 males in the U.S. living with the disorder.

About Sangamo Therapeutics

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

Forward Looking Statements

Thispressreleasemaycontainforward-looking statements based on Sangamo’s current expectations. Theseforward-looking statements include, without limitation references relating to the benefit of Fast Track designation to accelerate regulatory approval of SB-525, research and development of therapeutic applications of Sangamo’s gene therapy and ZFP technology platforms, the potential of Sangamo’s technology to treat hemophilia and lysosomal storage disorders, and the expected timing of initiating clinical trials of SB-525 and the release of data from these trials. Actual results may differ materially from these forward-looking statements due to a number of factors, includinguncertaintiesrelatingto substantial dependence on the clinical success of lead therapeutic programs,the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo’s ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo’s SEC filings, including the risk factors described in its Annual Report onForm10-K and its most recent QuarterlyReportonForm10-Q. Sangamo Therapeutics, Inc. assumes no obligation to update the forward-looking information contained in this pressrelease.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-receives-fast-track-designation-from-the-fda-for-sb-525-investigational-hemophilia-a-gene-therapy-300458224.html

SOURCE Sangamo Therapeutics, Inc.

http://www.sangamo.com

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Sangamo Receives Fast Track Designation From The FDA For SB-525 Investigational Hemophilia A Gene Therapy – PR Newswire (press release)

Gene therapy infection can prevent blindness, research shows – The Independent

A gene therapy that deliberately infects the eye with a virus can safely preserve vision in people affected by one of the leading causes of blindness, research has shown.

In a small preliminary study, scientists used an altered common cold-type virus to carry a repair gene that combats age-related macular degeneration (AMD).

The disease is marked by abnormal blood vessels that leak fluid into the central part of the retina, or macula.

After being injected into patients’ eyes, the virus penetrated retinal cells and deposited the gene, which manufactured a therapeutic protein called FLT01.

Lead researcher Professor Peter Campochiaro, from Johns Hopkins University in the US, said: This preliminary study is a small but promising step towards a new approach that will not only reduce doctor visits and the anxiety and discomfort associated with repeated injections in the eye, but may improve long-term outcomes.

The Phase I clinical trial involved 19 men and women aged 50 and older with advanced wet AMD.

With the help of the gene, retinal cells were turned into factories making FLT01.

The scientists hope this will eliminate the need to administer repeated injections of the protein, which suppresses a natural growth-driving molecule called VEGF.

Prolonged suppression of VEGF is needed to preserve vision, and that is difficult to achieve with repeated injections because life often gets in the way, said Prof Campochiaro.

For safety and ethical reasons, the patient group consisted of people for whom standard approved treatments were highly unlikely to restore vision.

Only 11 patients stood any chance of fluid reduction. Of those, four showed dramatic improvements after the gene therapy. The amount of fluid in their eyes dropped from a severe level to almost nothing.

Two other patients experienced a partial reduction in the amount of fluid in their eyes.

The findings are reported in the latest issue of The Lancet medical journal.

Press Association

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Gene therapy infection can prevent blindness, research shows – The Independent

Sangamo Biosciences (SGMO) Presents Recent Developments from Research and Clinical Programs at ASGCT – StreetInsider.com

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Sangamo Therapeutics, Inc. (NASDAQ: SGMO) today highlighted data from research and clinical-stage programs presented over the past week at the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT). Research from Sangamo scientists and collaborators was selected for 10 oral presentations and nine poster presentations during the conference.

“This year at ASGCT we showcased several exciting research and clinical programs emerging from Sangamo’s laboratories,” said Dr. Sandy Macrae, Sangamo’s CEO. “Sangamo is known for its leading research in genome editing, and over time we have developed additional expertise in gene therapy, gene regulation and cell therapy. We are also rapidly advancing our viral and non-viral delivery capabilities which have the potential to broaden our applications of genomic therapies. Such range of expertise allows us to be selective as we pair technology platforms with therapeutic applications, and compels us to make strategic choices about our product candidates. We will develop and commercialize certain products ourselves, while others, such as our gene therapy for hemophilia A now in collaboration with Pfizer, or our CNS or oncology programs, we may advance with a partner to leverage the right disease area focus, skills and resources.”

Selected Highlights from ASGCT 2017

Zinc Finger Nuclease Technology ImprovementsEd Rebar, Ph.D., Sangamo’s vice president of technology, presented recent enhancements to the Company’s zinc finger nuclease (ZFN) genome editing technology that substantially improve specificity while maintaining very high levels of on-target modification. These include the removal of positively charged amino acids in the zinc finger beta-sheet that make non-specific contacts with the DNA phosphate backbone, as well as the substitution of key residues within the Fok-1 cleavage domain. Dr. Rebar showed that these refinements could be applied broadly to ZFN reagents to substantially reduce off-target cleavage without sacrificing on-target cutting efficiency.

Dr. Rebar concluded with a detailed specificity analysis of a ZFN pair, in which these approaches were combined, which identified no significant off-target modification with an assay sensitivity of approximately 0.1%. Importantly, this study was performed on samples generated using clinically relevant delivery conditions, transfection scales and cell types, and with an on-target modification level of greater than 80%.

Gene Therapy for Fabry DiseaseThomas Wechsler, Ph.D., Sangamo’s director and lead scientist for rare diseases, presented new data from the Company’s preclinical AAV-cDNA gene therapy program for Fabry disease. Earlier in the week, Sangamo announced that it will advance this program toward human clinical development with preclinical studies enabling an Investigational New Drug Application (IND) in the second half 2018.

Fabry is an X-linked lysosomal storage disorder caused by mutations in the GLA gene that encodes for the alpha-galactosidase A enzyme (-Gal A). This mutation results in the buildup of Gb3 and Lyso-Gb3 lipid molecules in the body’s cells, resulting in a range of symptoms and life-threatening complications that affect multiple tissues and organ systems in the body.

Dr. Weschler presented data from GLAKO mouse models of Fabry disease demonstrating that a single infusion of Sangamo’s AAV vector containing an -Gal A transgene and a liver specific promoter successfully transduced the liver, resulting in episomal expression of -Gal A in the plasma and various tissues for the duration of the study, out to 60 days. From a single treatment, the AAV-cDNA vector achieved enzyme activity levels in the plasma of up to 100 fold greater than wildtype and 10 to 100 fold greater than wildtype in tissues including the liver, heart, kidney and spleen. Importantly, -Gal A secreted from the liver led to a significant reduction in the levels of accumulated Gb3 and Lyso-Gb3 lipid substrates, in target tissues such as the kidney and heart.

Gene Regulation Treatment for Reduction of TauSangamo Scientist Bryan Zeitler, Ph.D., presented recent data demonstrating significant reduction of tau expression using Sangamo’s proprietary zinc finger protein transcription factor (ZFP-TF) gene-regulation technology. The research was conducted in conjunction with Dr. Brad Hyman, Director of the Alzheimer’s Disease Research Center at Massachusetts General Hospital. The reduction of tau expression has been shown to help reduce neurofibrillary tangles in the brain and provide neuronal protection and reversal of pathology in Alzheimer’s disease and other tauopathy disease models.

The presentation included data from in vivo studies in wild-type mice demonstrating up to 90% reduction of tau mRNA and protein in the mouse hippocampus, as well as up to 70% tau reduction across all regions of the brain, including the cortex, midbrain, cerebellum, thalamus, hypothalamus and striatum.

In addition, data from in vivo studies in an amyloid mouse model of Alzheimer’s disease suggest that a single administration of ZFP-TFs significantly reduced neuronal dystrophies in mice with established disease pathology. This is the first time that a tau lowering agent has demonstrated a reduction in neuritic dystrophy. Specificity and off-target analysis in ZFP-TF-treated primary neurons revealed that tau was the only gene suppressed out of more than 26,000 coding transcripts analyzed. New data in Dr. Zeitler’s presentation demonstrated that the effect of ZFP-TF treatment in lowering tau was durable out to the last measurement, at 11 months.

These experiments were conducted using Sangamo’s novel, proprietary AAV serotype for improved CNS transduction.

Sangamo intends to seek a partner with disease area expertise for the development and commercialization of its gene regulation approach for certain central nervous system applications including Alzheimer’s disease and other tauopathies.

In Vivo Genome Editing Treatments for MPS I and MPS IISangamo Scientist Russell DeKelver, Ph.D., presented additional preclinical data from the Company’s in vivo genome editing clinical programs in MPS I and MPS II demonstrating phenotypic correction of disease in mouse models following a single administration of Sangamo’s genome editing treatments. Newly presented histopathological analysis demonstrated reduced cellular vacuolation in various secondary tissues, as well as in the bone marrow, and central nervous system tissues such as the spinal cord and pituitary gland in treated MPS I and MPS II mice, four months after dosing. Furthermore, newly presented mass spectrometry analysis confirmed significant reduction of dermatan sulfate, a type of GAG biomarker, in the brains of MPS I and MPS II mice treated with Sangamo’s genome editing treatments.

Sangamo recently initiated two Phase 1/2 clinical trials evaluating SB-318 and SB-913, ZFN-mediated in vivo genome editing treatments for MPS I and MPS II, respectively. Data are expected in late 2017 or early 2018.

Cell TherapyResearch by Brigit Riley, Ph.D., Sangamo’s director of discovery and translational research, was presented demonstrating high levels of homology driven genome editing of human B cells by ZFN mRNA and AAV6 transgene delivery. The data demonstrated robust ZFN-mediated, site-specific modification of B cells at targeted loci, including AAVS1, CCR5 and TRAC locus. The data also demonstrated high levels of targeted transgene insertion, driven by homology directed repair, using a B cell specific promoter. Analysis of AAV serotype transduction showed the superiority of AAV6 in transducing B cells compared to several other serotypes.

The data demonstrate the potential for using genome editing to genetically modify B cells ex vivo and harness their natural ability to produce large amounts of antibodies to generate protein production reservoirs. This novel approach for using genome editing to harness the protein production capacity of B cells could be relevant for multiple indications, including immune disorders, cancer immunotherapies and other monogenic disorders.

DeliverySangamo Scientist Anthony Conway, Ph.D., presented new data from the Company’s research into a next-generation delivery platform using lipid nanoparticles (LNPs). ZFN mRNA delivery via LNPs allowed for accumulation of genome modification within the mouse liver following repeat administration, with progressive increases in genomic modification out to six repeat doses tested. LNP delivery of new ZFN architectures led to greater than 85% on-target modification in vitro and greater than 60% on-target modification in vivo, resulting in greater than 90% protein knockdown of TTR and PCSK9 in wildtype mice. Repeat dosing of ZFNs using LNP-mRNA in combination with a single human AAV-IDS donor vector resulted in efficient targeted insertion of the IDS gene into the albumin locus and accumulative enzymatic activity levels in mouse plasma after each subsequent dose.

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Sangamo Biosciences (SGMO) Presents Recent Developments from Research and Clinical Programs at ASGCT – StreetInsider.com

SENS Research Foundation Announces New Research Program on … – Marketwired (press release)

MOUNTAIN VIEW, CA–(Marketwired – May 15, 2017) – SENS Research Foundation (SRF) has launched a new research program focused on somatic gene therapy in collaboration with the Buck Institute for Research on Aging. Brian Kennedy, PhD, a leading expert on the biology of aging, will be running the project in his lab at the Buck.

Many potential treatments of age-related diseases require the addition of new genes to the genome of cells in the body, a technology known as somatic gene therapy. The technology has been hampered, up until now, by the inability to control where the gene is inserted. That lack of control resulted in a significant risk of insertion in a location that encourages the cell to become malignant.

SRF has devised a new method for inserting genes into a pre-defined location. In this program, this will be done as a two-step process, in which first CRISPR is used to create a “landing pad” for the gene, and then the gene is inserted using an enzyme that only recognizes the landing pad. SRF has created “maximally modifiable mice” that already have the landing pad, and this project will evaluate how well the insertion step works in different tissues.

“Somatic gene therapy has been a goal of medicine for decades. Being able to add new healthy genes will enable us to address treatments of such age-related diseases as atherosclerosis and macular degeneration. Our collaboration with SRF will substantially move us toward finding effective treatments to genetically based age-related diseases,” said Dr. Kennedy.

“Partnering with Brian Kennedy and the Buck enables SRF to continue towards our goal of achieving human clinical trials on rejuvenation biotechnologies in the next five years. Brian’s leadership in moving this technology into mammals is a huge step forward,” said Dr. Aubrey de Grey, CSO, SENS Research Foundation.

This research has been made possible through the generous support of the Forever Healthy Foundation and its founder Michael Greve, as well as the support of our other donors. The Forever Healthy Foundation is a private nonprofit initiative whose mission is to enable people to vastly extend their healthy lifespans and be part of the first generation to cure aging. In order to accelerate the development of therapies to bring aging under full medical control, the Forever Healthy Foundation directly supports cutting-edge research aimed at the molecular and cellular repair of damage caused by the aging process.

About SENS Research Foundation (SRF)SENS Research Foundation is a 501(c)(3) nonprofit that works to research, develop, and promote comprehensive regenerative medicine solutions for the diseases of aging. SRF is focused on a damage repair paradigm for treating the diseases of aging, which it advances through scientific research, advocacy, and education. SENS Research Foundation supports research projects at universities and institutes around the world with the goal of curing such age-related diseases as macular degeneration, heart disease, cancer, and Alzheimer’s disease. Educating the public and training researchers to support a growing regenerative medicine field are also major endeavors of the organization that are being accomplished though advocacy campaigns and educational programs. For more information, visit http://www.sens.org.

About Buck Institute for Research on AgingBuck Institute is the U.S.’s first independent research organization devoted to Geroscience — focused on the connection between normal aging and chronic disease. Based in Novato, California, the Buck is dedicated to extending “healthspan,” the healthy years of human life, and does so by utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and others focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimer’s and Parkinson’s, cancer, cardiovascular disease, macular degeneration, osteoporosis, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics, bioinformatics and stem cell technologies. For more information: http://www.thebuck.org.

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SENS Research Foundation Announces New Research Program on … – Marketwired (press release)

Sangamo Therapeutics Presents Recent Developments from Research and Clinical Programs at Annual Meeting of the … – PR Newswire (press release)

Selected Highlights from ASGCT 2017

Zinc Finger Nuclease Technology ImprovementsEd Rebar, Ph.D., Sangamo’s vice president of technology, presented recent enhancements to the Company’s zinc finger nuclease (ZFN) genome editing technology that substantially improve specificity while maintaining very high levels of on-target modification. These include the removal of positively charged amino acids in the zinc finger beta-sheet that make non-specific contacts with the DNA phosphate backbone, as well as the substitution of key residues within the Fok-1 cleavage domain. Dr. Rebar showed that these refinements could be applied broadly to ZFN reagents to substantially reduce off-target cleavage without sacrificing on-target cutting efficiency.

Dr. Rebar concluded with a detailed specificity analysis of a ZFN pair, in which these approaches were combined, which identified no significant off-target modification with an assay sensitivity of approximately 0.1%. Importantly, this study was performed on samples generated using clinically relevant delivery conditions, transfection scales and cell types, and with an on-target modification level of greater than 80%.

Gene Therapy for Fabry DiseaseThomas Wechsler, Ph.D., Sangamo’s director and lead scientist for rare diseases, presented new data from the Company’s preclinical AAV-cDNA gene therapy program for Fabry disease. Earlier in the week, Sangamo announced that it will advance this program toward human clinical development with preclinical studies enabling an Investigational New Drug Application (IND) in the second half 2018.

Fabry is an X-linked lysosomal storage disorder caused by mutations in the GLA gene that encodes for the alpha-galactosidase A enzyme (-Gal A). This mutation results in the buildup of Gb3 and Lyso-Gb3 lipid molecules in the body’s cells, resulting in a range of symptoms and life-threatening complications that affect multiple tissues and organ systems in the body.

Dr. Weschler presented data from GLAKO mouse models of Fabry disease demonstrating that a single infusion of Sangamo’s AAV vector containing an -Gal A transgene and a liver specific promoter successfully transduced the liver, resulting in episomal expression of -Gal A in the plasma and various tissues for the duration of the study, out to 60 days. From a single treatment, the AAV-cDNA vector achieved enzyme activity levels in the plasma of up to 100 fold greater than wildtype and 10 to 100 fold greater than wildtype in tissues including the liver, heart, kidney and spleen. Importantly, -Gal A secreted from the liver led to a significant reduction in the levels of accumulated Gb3 and Lyso-Gb3 lipid substrates, in target tissues such as the kidney and heart.

Gene Regulation Treatment for Reduction of TauSangamo Scientist Bryan Zeitler, Ph.D., presented recent data demonstrating significant reduction of tau expression using Sangamo’s proprietary zinc finger protein transcription factor (ZFP-TF) gene-regulation technology. The research was conducted in conjunction with Dr. Brad Hyman, Director of the Alzheimer’s Disease Research Center at Massachusetts General Hospital. The reduction of tau expression has been shown to help reduce neurofibrillary tangles in the brain and provide neuronal protection and reversal of pathology in Alzheimer’s disease and other tauopathy disease models.

The presentation included data from in vivo studies in wild-type mice demonstrating up to 90% reduction of tau mRNA and protein in the mouse hippocampus, as well as up to 70% tau reduction across all regions of the brain, including the cortex, midbrain, cerebellum, thalamus, hypothalamus and striatum.

In addition, data from in vivo studies in an amyloid mouse model of Alzheimer’s disease suggest that a single administration of ZFP-TFs significantly reduced neuronal dystrophies in mice with established disease pathology. This is the first time that a tau lowering agent has demonstrated a reduction in neuritic dystrophy. Specificity and off-target analysis in ZFP-TF-treated primary neurons revealed that tauwas the only gene suppressed out of more than 26,000 coding transcripts analyzed. New data in Dr. Zeitler’s presentation demonstrated that the effect of ZFP-TF treatment in lowering tau was durable out to the last measurement, at 11 months.

These experiments were conducted using Sangamo’s novel, proprietary AAV serotype for improved CNS transduction.

Sangamo intends to seek a partner with disease area expertise for the development and commercialization of its gene regulation approach for certain central nervous system applications including Alzheimer’s disease and other tauopathies.

In Vivo Genome Editing Treatments for MPS I and MPS IISangamo Scientist Russell DeKelver, Ph.D., presented additional preclinical data from the Company’s in vivo genome editing clinical programs in MPS I and MPS II demonstrating phenotypic correction of disease in mouse models following a single administration of Sangamo’s genome editing treatments. Newly presented histopathological analysis demonstrated reduced cellular vacuolation in various secondary tissues, as well as in the bone marrow, and central nervous system tissues such as the spinal cord and pituitary gland in treated MPS I and MPS II mice, four months after dosing. Furthermore, newly presented mass spectrometry analysis confirmed significant reduction of dermatan sulfate, a type of GAG biomarker, in the brains of MPS I and MPS II mice treated with Sangamo’s genome editing treatments.

Sangamo recently initiated two Phase 1/2 clinical trials evaluating SB-318 and SB-913, ZFN-mediated in vivo genome editing treatments for MPS I and MPS II, respectively. Data are expected in late 2017 or early 2018.

Cell TherapyResearch by Brigit Riley, Ph.D.,Sangamo’s director of discovery and translational research, was presented demonstrating high levels of homology driven genome editing of human B cells by ZFN mRNA and AAV6 transgene delivery. The data demonstrated robust ZFN-mediated, site-specific modification of B cells at targeted loci, including AAVS1, CCR5 and TRAC locus. The data also demonstrated high levels of targeted transgene insertion, driven by homology directed repair, using a B cell specific promoter. Analysis of AAV serotype transduction showed the superiority of AAV6 in transducing B cells compared to several other serotypes.

The data demonstrate the potential for using genome editing to genetically modify B cells ex vivo and harness their natural ability to produce large amounts of antibodies to generate protein production reservoirs. This novel approach for using genome editing to harness the protein production capacity of B cells could be relevant for multiple indications, including immune disorders, cancer immunotherapies and other monogenic disorders.

DeliverySangamo Scientist Anthony Conway, Ph.D., presented new data from the Company’s research into a next-generation delivery platform using lipid nanoparticles (LNPs). ZFN mRNA delivery via LNPs allowed for accumulation of genome modification within the mouse liver following repeat administration, with progressive increases in genomic modification out to six repeat doses tested. LNP delivery of new ZFN architectures led to greater than 85% on-target modification in vitro and greater than 60% on-target modification in vivo, resulting in greater than 90% protein knockdown of TTR and PCSK9 in wildtype mice. Repeat dosing of ZFNs using LNP-mRNA in combination with a single human AAV-IDS donor vector resulted in efficient targeted insertion of the IDS gene into the albumin locus and accumulative enzymatic activity levels in mouse plasma after each subsequent dose.

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

Forward Looking StatementsThis press release contains forward-looking statements regarding Sangamo’s current expectations. These forward looking statements include, without limitation, references to the potential of novel delivery systems to broaden applications of genomic therapies,the ability to bring research and preclinical studies to clinical development, the expected timing of filing INDs and releasing data from ongoing clinical programs, the intent to seek partners and collaborators to develop and commercialize gene regulation treatment, and the research and development of ZFNs and ZFP-TFs, clinical trials and therapeutic applications of Sangamo’s ZFP technology. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the dependence on the success of clinical trials of lead programs, the lengthy and uncertain regulatory approval process, uncertainties related to the timing of initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of Sangamo’s therapeutics, and the ability to establish strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable gene-based therapeutics. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo’s operations and business environments. These risks and uncertainties are described more fully in Sangamo’s Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q as filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

ContactSangamo Therapeutics, Inc. McDavid Stilwell (510) 970-6000, x219 mstilwell@sangamo.com

Varant Shirvanian (510) 970-6000, x205 vshirvanian@sangamo.com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-presents-recent-developments-from-research-and-clinical-programs-at-annual-meeting-of-the-american-society-of-gene–cell-therapy-300457323.html

SOURCE Sangamo Therapeutics, Inc.

http://www.sangamo.com

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Sangamo Therapeutics Presents Recent Developments from Research and Clinical Programs at Annual Meeting of the … – PR Newswire (press release)

Selecta Biosciences Announces New Preclinical Gene Therapy Data at the Annual Meeting of the American Society of … – GlobeNewswire (press release)

May 15, 2017 08:00 ET | Source: Selecta Biosciences

WATERTOWN, Mass., May 15, 2017 (GLOBE NEWSWIRE) — Selecta Biosciences, Inc. (NASDAQ:SELB), a clinical-stage biopharmaceutical company focused on unlocking the full potential of biologic therapies by avoiding unwanted immune responses, today announced new preclinical data regarding non-immunogenic gene therapies that were presented at the American Society of Gene & Cell Therapy (ASGCT) 2017 Annual Meeting, which took place last week in Washington, D.C.

Immunogenicity is a key challenge in gene therapy, limiting the number of diseases and patients that can be effectively treated and presenting a safety hurdle, said Werner Cautreels, Ph.D., CEO and Chairman of Selecta. Together with various collaborators, we have again demonstrated the potential of Selectas proprietary immune tolerance Synthetic Vaccine Particles (SVP) technology, which is designed to improve the clinical benefits and transform the development of gene therapy. We also were pleased with the presentation of preclinical proof-of-concept data for our proprietary product candidate in MMA, a life-threatening rare disease that can only be treated today by diet or organ transplantation.

A team led by Charles Venditti, M.D., Ph.D., Senior Investigator and Head, Organic Acid Research Section in the National Human Genome Research Institute at the National Institutes of Health, and Luk Vandenberghe, Ph.D., Director of the Grousbeck Gene Therapy Center at Mass. Eye and Ear and an Assistant Professor at Harvard Medical School, delivered a presentation entitled Anc80 Mediates Hepatic Correction of Methylmalonyl-CoA Mutase Deficiency in Murine Models of Methymalonic Acidemia. This presentation featured data from mouse models of MMA, a rare inborn error of metabolism most frequently caused by mutations in the enzyme methylmalonyl-CoA mutase (MUT). In this study, MUT-deficient mice were treated with Selectas Anc80-synMUT product candidate to express the human MUT gene. The gene therapy induced a robust biochemical and clinical response as plasma methylmalonic acid levels dropped precipitously, substantial weight gain ensued and survival was sustained. Further, presented data indicate that the combination of SVP and Anc80 could effectively overcome the immunogenicity that has limited other gene therapy programs by enabling enrollment of patients with pre-existing antibodies to AAV and keeping patients eligible for repeat administration.

A team led by Federico Mingozzi, Ph.D., Head of Immunology and Liver Gene Therapy at Genethon, delivered a presentation entitled Modulation of AAV Vector Dosing and Avoidance of Capsid Immune Responses via Repeated Co-Administration of Vector with Rapamycin Tolerogenic Nanoparticles. This presentation featured data from both mouse and non-human primate studies demonstrating how the co-administration of SVP-Rapamycin completely blocked anti-AAV immune responses in an antigen-specific manner and allowed for vector re-administration and gene therapy dose titration. The ability to dose titrate could provide for more effective development and administration of gene therapies.

Click here to view these presentations.

About Selecta’s MMA Program

MMA is an inborn error of metabolism that, according to the U.S. National Institutes of Health (NIH), affects an estimated one in 25,000 to 48,000 individuals globally. MMA patients are unable to process certain proteins and fats, leading to the accumulation of toxic metabolites. Symptoms of this life-threatening disease start to develop in early childhood and, despite strict diet, patients suffer from a wide range of disease-related complications such as pancreatitis, strokes and chronic kidney failure. Selecta exclusively licensed Anc80 for MMA from Massachusetts Eye and Ear (MEE) in May 2016. Under the license agreement, Selecta also has the exclusive option to develop gene therapies using Anc80 for additional pre-defined lysosomal storage, genetic muscular and genetic metabolic diseases. In early 2017, Selecta entered into a strategic manufacturing agreement with Lonza Houston, Inc. under which Lonzawill produce an Anc80-AAV-based gene therapy product for Selecta’s MMA program.

Selecta intends to combine Anc80 with recently discovered transgenes and Selectas SVP-Rapamycin to create a novel gene therapy for MMA. This therapy is intended to a) enable the treatment of patients with and without pre-existing anti-AAV antibodies; b) prevent cellular immune responses that often reduce the expression levels of gene therapies; and c) provide the ability to administer repeat gene therapy doses to achieve sufficient levels of methylmalonyl-CoA mutase (MUT), the enzyme that MMA patients are lacking.

To advance the MMA program, Selecta entered into a Collaborative Research and Development Agreement (CRADA) with MEE and the National Human Genome Research Institute, NIH, in 2016. Principal investigators in this CRADA initiative are Charles Venditti, M.D., Ph.D., Senior Investigator and Head, Organic Acid Research Section in the National Human Genome Research Institute at the National Institutes of Health, and Luk Vandenberghe, Ph.D., Director of the Grousbeck Gene Therapy Center at MEE and an Assistant Professor at Harvard Medical School. A physician-scientist specializing in the study of inborn errors of metabolism including MMA, Dr. Venditti and his group have published several studies showing the effectiveness of gene therapy as a treatment for MMA in mice. Dr. Vandenberghe from MEE is the inventor of Anc80.

About Selecta Biosciences, Inc.

Selecta Biosciences, Inc. is a clinical-stage biopharmaceutical company that is focused on unlocking the full potential of biologic therapies by avoiding unwanted immune responses. Selecta plans to combine its tolerogenic Synthetic Vaccine Particles (SVP) to a range of biologics for rare and serious diseases that require new treatment options. The companys current proprietary pipeline includes SVP-enabled enzyme, oncology and gene therapies. SEL-212, the companys lead candidate in Phase 2, is being developed to treat severe gout patients and resolve their debilitating symptoms, including flares and gouty arthritis. Selectas clinical oncology candidate, LMB-100, is in a Phase 1 program targeting pancreatic cancer and mesothelioma. Its two proprietary gene therapy product candidates are being developed for rare inborn errors of metabolism and have the potential to enable repeat administration. The use of SVP is also being explored in the development of vaccines and treatments for allergies and autoimmune diseases. Selecta is based in Watertown, Massachusetts. For more information, please visit http://selectabio.com and follow @SelectaBio on Twitter.

Forward-Looking Statements

Any statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (the company), including without limitation, whether the companys MMA product candidate will prevent cellular immune responses, enable repeat administration or allow for the treatment of patients with and without pre-existing anti-AAV antibodies, the companys ability to unlock the full potential of biologic therapies, the companys plan to apply its SVP platform to a range of biologics for rare and serious diseases, the potential of SEL-212 to treat severe gout patients and resolve their debilitating symptoms, the potential of the companys two gene therapy product candidates to enable repeat administration, the potential treatment applications for products utilizing the SVP platform in areas such as gene therapy, immuno-oncology, allergies, autoimmune diseases and vaccines, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors discussed in the Risk Factors section of the companys Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, or SEC, on May 11, 2017, and in other filings that the company makes with the SEC. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any obligation to update any forward-looking statements included in this press release.

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Selecta Biosciences Announces New Preclinical Gene Therapy Data at the Annual Meeting of the American Society of … – GlobeNewswire (press release)

CRISPR kills HIV and eats Zika ‘like Pac-man’. Its next target? Cancer – Wired.co.uk

HIV has no cure. Its not quite the implacable scourge it was throughout the 1980s and 1990s, thanks to education, prophylactics, and drugs like PrEP. But still, no cure. Genetically-modified humans: what is CRISPR and how does it work?

Part of the problem is HIVs ability to squirrel itself away inside a cells DNA including the DNA of the immune cells that are supposed to be killing it. The same ability, though, could be HIVs undoing. All because of CRISPR. You know, CRIPSR: the gene-editing technique that got everyone really excited, then really sceptical, and now cautiously optimistic about curing a bunch of intractable diseases.

Last week, a group of biologists published research detailing how they hid an anti-HIV CRISPR system inside another type of virus capable of sneaking past a hosts immune system. Whats more, the virus replicated and snipped HIV from infected cells along the way. At this stage, it works in mice and rats, not people. But as a proof of concept, it means similar systems could be developed to fight a huge range of diseasesherpes, cystic fibrosis, and all sorts of cancers.

Those diseases are all treatable, to varying degrees. But the problem with treatments is you have to keep doing them in order for them to work. The current anti-retroviral therapy for HIV is very successful in suppressing replication of the virus, says Kamel Khalili, a neurovirologist at Temple University in Philadelphia and lead author of the recent research, published in Molecular Therapy. But that does not eliminate the copies of the virus that have been integrated into the gene, so any time the patient doesnt take their medication the virus can rebound. Plus treatments can and often do fail.

Gene therapy has promised to revolutionise medicine since the 1970s, when a pair of researchers introduced the concept of using viruses to replace bad DNA with good DNA. The first working model was tested on mice in the 1980s, and by the 1990s researchers were using gene therapies with limited success to treat immune and nutrition deficiencies. Then, in 1999, a patient in a University of Pennsylvania gene therapy trial named Jesse Gelsinger died from complications. The tragedy temporarily skid-stopped the whole field. Gene therapy had been steadily getting its groove back, but the 2012 discovery that CRISPR could make easy, and accurate, cuts on human genes, added more vigor.

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CRISPR as an agent for curing HIV has its own problems. For one, it has to be able to snip away the HIV from an infected cell without damaging any of the surrounding DNA. HIV mutates and evolves, so Khalili and his co-authors couldnt just program their CRISPR system with a single genetic mugshot. Instead, they had to target enough unchanging sections that were also critical to the virus survival.

Their next challenge was delivering the system to a critical mass of infected cells. First, you have to get it past the immune system which is programmed to attack any non-foreign object entering the body. They did this by packing their CRISPR system inside another type of virus called AAV (short for adeno associated virus). AAVs are a very small helper virus, they cant actually replicate in a cell on their own unless they have another virus there to help it along, says Keith Jerome, a microbiologist at the Fred Hutchinson Cancer Research Centre in Seattle. The great thing about AAVs is they cause essentially no immune system response in humans. Although thats not always true. Jesse Gelsinger died in 1999 because his immune system overreacted to the AAVs hed been given in his gene therapy trial. So doctors hoping to prescribe AAV-based gene therapy have to be aware of patients prior exposure.

In order to get approved for human use, this type of CRISPR-borne cure would have to be both safe and effective. This study got part of the way but was going strictly for efficacy: Does this work? Khalili and his co-authors treated mice and rat model with strains of HIV that were latent; hiding away in cellular DNAand others where the HIV was actively replicating. Then they used it on mice grafted with human cells. In all three cases, HIV rates went down significantly.

Other good news on the safety front: theres no evidence their trial made any off-target cuts. Ageing is a disease. Gene therapy could be the ‘cure’

Theyll now need to run more experiments to make sure thats absolutely the case, probably using primate models since their DNA is closer to humans. They also have to make sure the treatment gets rid of enough HIV, so it doesnt just replicate itself back to harmful levels. In actual human patients theres no way that a CRISPR gene therapy will ever get 100 per cent of HIV, says Paul Knoepfler, a stem cell biologist at UC Davis. How highly efficient will be efficient enough to make a clinically meaningful impact?

Khalili believes he can get close enough. According to him, the CRISPR system doesnt need to eliminate all the HIV-infected cells, just enough so an HIV-patients immune system can get strong enough to take care of the rest on its own. I strongly believe in the gene-editing strategy, and with my 30 years in HIV research, I think this is the one that is going to take us to the end.

Hes not the only optimist. The advantage of using a virus as your delivery system is it can infect virtually every cell, says Jianhua Luo, a pathologist at the University of Pittsburgh. Luo is using a similar CRISPR-in-a-virus system to target cancerous DNA in cells.

And curing HIV could be a proof-of-concept for other diseases even genetic diseases people are born with. Although the virus starts as a simple infection, once it becomes part of a persons chromosome, it essentially becomes a genetic disease.

Since the HIV research was published, a team of biologists at University of California, Berkeley, described 10 new CRISPR enzymes that, once activated, are said to “behave like Pac-Man” to chew through RNA in a way that could be used as sensitive detectors of infectious viruses.

These new enzymes are variants of a CRISPR protein, Cas13a, which the UC Berkeley researchers reported last September in Nature, and could be used to detect specific sequences of RNA, such as from a virus. The team showed that once CRISPR-Cas13a binds to its target RNA, it begins to indiscriminately cut up all RNA making it “glow” to allow signal detection.

Two teams of researchers at the Broad Institute subsequently paired CRISPR-Cas13a with the process of RNA amplification to showed that the system, dubbed Sherlock, could detect viral RNA at extremely low concentrations, such as the presence of dengue and Zika viral RNA, for example. Such a system could be used to detect any type of RNA, including RNA distinctive of cancer cells.

Imagine a world where, instead of removing her breasts, Angelina Jolie could instead have taken a dose of genes that snip away the BRCA2 genes that threatened her with cancer. Thats the difference between a treatment and a cure.

Nick Stockton is a staff writer for WIRED US. This article originally appeared on WIRED. It has been updated to reference the new University of California, Berkeley research.

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CRISPR kills HIV and eats Zika ‘like Pac-man’. Its next target? Cancer – Wired.co.uk

AveXis Reports First Quarter 2017 Financial and Operating Results … – GlobeNewswire (press release)

May 11, 2017 16:00 ET | Source: AveXis

CHICAGO, May 11, 2017 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported financial results for the first quarter ended March 31, 2017, recent corporate highlights and upcoming milestones.

We are very pleased with our progress during the first quarter and recent weeks, including the recently reported encouraging results from the closeout of the Phase 1 trial of AVXS-101 in SMA Type 1, said Sean Nolan, President and Chief Executive Officer of AveXis. Our team is focused on executing our plan to bring AVXS-101 to patients suffering from SMA Type 1 as quickly and safely as possible.

Recent Highlights

Results from the Phase 1 Trial of AVXS-101 in SMA Type 1: The Phase 1, open-label, dose-escalating study was designed to evaluate the safety and tolerability of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1. The key measures of efficacy were the time from birth to an event, which was defined as either death or at least 16 hours per day of required ventilation support for breathing for 14 consecutive days in the absence of acute reversible illness or perioperatively, and video confirmed achievement of ability to sit unassisted. Additionally, several exploratory objective measures were assessed, including a standard motor milestone development survey and Childrens Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND).

Presented AVXS-101 research at the Annual Meeting of the American Academy of Neurology: Jerry Mendell, M.D., director of the Center for Gene Therapy at The Research Institute at Nationwide Childrens Hospital, presented results from the Phase 1 study of AVXS-101 in SMA Type 1 during a plenary session, including video evidence of children achieving motor milestones.

Completed Type B chemistry manufacturing and controls (CMC) meeting with the U.S. Food and Drug Administration (FDA): On May 1, 2017, AveXis participated in a Type B CMC meeting with the FDA. The purpose of the meeting was to present to the agency AveXis proposed process for producing the intended commercial scale GMP-derived gene therapy product, to gain alignment with the agency on the proposed assay qualification plan, and to gain alignment on the proposed protocol for demonstrating comparability of the intended commercial scale GMP-derived product with the material administered to patients in the Phase 1 trial of AVXS-101 in SMA Type 1.

The company expects to provide an update on its further plans and development timelines following receipt of the minutes of the CMC Type B meeting, currently anticipated in early June 2017.

Joao Siffert Appointed to Board of Directors: On April 19, 2017, AveXis announced the appointment of Joao Siffert to its Board of Directors, effective upon the completion of the annual meeting of stockholders. Dr. Siffert brings important knowledge to the Board based on his experience in central nervous system drug development and regulatory expertise in both the U.S. and Europe, as well as his experience working with global health care companies.

First Quarter 2017 Financial Results

Selected Financial Information

Operating Results:

Balance Sheet Information:

Conference Call Information The AveXis conference call and webcast of April 25, 2017 was conducted in lieu of a first quarter 2017 financial and operating results conference call. AveXis will not host a conference call and webcast related to its first quarter 2017 financial and operating results. The Company expects to host its next conference call and webcast following receipt of the minutes from the CMC meeting with the FDA, expected approximately 30 days following the meeting, which took place May 1, 2017.

About SMA SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births.

The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 1 is the leading genetic cause of infant mortality.

About AVXS-101 AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons providing rapid onset of effect, and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.

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, is in an ongoing Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit http://www.avexis.com.

Forward-Looking Statements This press release contains “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, AveXis research, development and regulatory plans for AVXS-101, including the potential of AVXS-101 to positively impact quality of life and alter the course of disease in children with SMA Type 1 and statements about the effects of SMA Type 1 on developmental milestones and timing of regulatory feedback. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the scope, progress, expansion, and costs of developing and commercializing AveXis product candidates; regulatory developments in the U.S. and EU, as well as other factors discussed in the “Risk Factors” and the “Management’s Discussion and Analysis of Financial Condition and Results of Operations” section of AveXis Annual Report on Form 10-K for the year ended December 31, 2016, filed with the SEC on March 16, 2017. In addition to the risks described above and in the Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect AveXis results. There can be no assurance that the actual results or developments anticipated by AveXis will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, AveXis. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved.

All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. AveXis cautions investors not to rely too heavily on the forward-looking statements AveXis makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). AveXis undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Non-GAAP Financial Measure In addition to disclosing financial results that are determined in accordance with GAAP, in order to understand and evaluate our operating performance, and provide a more complete understanding of factors and trends affecting our business, we also measure the increase in research and development expenses and general and administrative expenses excluding non-cash stock-based compensation expense. We believe that excluding this expense better reflects the increase in research and development and general and administrative expenses during the period, as compared to the prior period. This non-GAAP financial metric should be considered supplemental to and not a substitute for financial information prepared in accordance with GAAP. Because non-GAAP financial metrics exclude the effect of items that will increase or decrease the companys reported results of operations, we strongly encourage investors to review our consolidated financial statements and periodic reports in their entirety.

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AveXis Reports First Quarter 2017 Financial and Operating Results … – GlobeNewswire (press release)

Pfizer expands hemophilia gene therapy pipeline with $720m Sangamo deal – BioPharma-Reporter.com

Pfizer has upped its presence in the gene therapy space through collaboration with Sangamo Therapeutics on its programmes worth up to $720m.

The agreement sees Pharma giant Pfizer pay Sangamo $70m upfront, but the Californian genome editing firm could receive up to $475m in milestone payments for its lead haemophilia A gene therapy SB-525, and a further $175m for other candidates developed under the collaboration.

Sangamos gene therapies are based on its multi-platform technology, including its zinc finger nuclease (ZFN) knock-out gene tool, and both adeno associated virus (AAV) and messenger RNA delivery tech.

Sangamo brings deep scientific and technical expertise across multiple genomic platforms, and we look forward to working together to advance this potentially transformative treatment for patients living with Hemophilia A, Pfizers president of Worldwide R&D Mikael Dolsten said in a statement.

The deal is the latest venture for Pfizer in the haemophilia gene therapy sector. The firm has a long-standing haemophilia B collaboration with Spark Therapuetics, inked in December 2014 when Pfizer also established its gene therapy research unit.

Pfizer has since acquired Bamboo Therapeutics for $150m, adding an advanced recombinant AAV vector design and production technology and several pipeline therapies, and begun assessing sites in North Carolina to expand its gene therapy manufacturing capabilities.

Pfizer has made significant investments in gene therapy over the last few years and we are building an industry-leading expertise in recombinant adeno-associated virus (rAAV) vector design and manufacturing, said Dolsten.

We believe SB-525 has the potential to be a best-in-class therapy that may provide patients with stable and durable levels of Factor VIII protein with a single administration treatment.

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Pfizer expands hemophilia gene therapy pipeline with $720m Sangamo deal – BioPharma-Reporter.com

Pfizer doubles down on gene therapy pipeline with $70M Sangamo buy-in – FierceBiotech

After deals and acquisitions with Spark Therapeutics and Bamboo, Pfizer is once again looking to bolster its rare and gene therapy pipeline as it outlines a new drug pact with Sangamo.

The collaborationlicense agreement focuses on the development and eventual sale of the biotechs gene therapy programs for hemophilia A, including SB-525, one of Sangamos four lead product candidates.

This early candidate is slated to enter the clinic this quarter, centering on testing safety as well as blood levels of Factor VIII protein, and other efficacy endpoints.

Sangamo gets $70 million upfront from the Big Pharma, and could gain $475 million in biobucks and sales royalties on any medications from the collaborationthat gain approval.

Under the deal, Sangamo will take the lead on the SB-525 phase 1/2 test as well as unspecified manufacturing activities.

Pfizer, meanwhile, will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for the therapy, as well as any additional products, if any.

Sangamo will also work with Pfizer on manufacturing and technical ops using viral delivery vectors.

SB-525 works as a AAV vector carrying a Factor VIII gene construct driven by a synthetic, liver-specific promoter. The FDA has already cleared the start of human trials for SB-525, and given it an orphan drug tag.

The deal has proved powerful for Sangamo, with its shares jumping 44% after hours on the news last night.

This marks another step into the new world of gene therapies for Pfizer, coming less than a year after its $700 million buy of Bamboo Therapeutics, adding advanced recombinant adeno-associated virus (rAAV)-based gene therapies to its pipeline.

It also has a long-standing deal with Spark Therapeutics, in hemophilia, penned in 2014. Back in January, Pfizer in fact paid a $15 million milestone bonus to Spark for hitting its marks in the ongoing hemophilia B phase 1/2 trial FDA breakthrough-tagged SPK-9001.

Pfizer also has a series of preclinical gene therapies, including a neuromuscular candidate for Duchenne muscular dystrophy (DMD), as well as preclinical candidates to treat Friedreichs ataxia and Canavan disease, and a phase I candidate for giant axonal neuropathy.

Pfizer also gained an operating gene therapy manufacturing facility that Bamboo bought from the University of North Carolina last year.

The pharma also has several academic research agreements, including one with Kings College London to develop a series of rAAV gene therapy vectors and another with the University of Iowa Research Foundation to develop a potential gene therapy for cystic fibrosis.

And its partnered with Emeryville, CA-based Molecular Therapeutics (4DMT) to discover and develop targeted next-generation rAAV vectors for cardiac disease; it made an investment in the company a few years back.

Once seen as the next big thing in research, gene therapies have however come under pressure in recent months about just how viable they are on the market. After struggling for years to make a commercial success out of Glybera, the worlds first approved gene therapy, uniQure recently called it quits on the treatment.

The drugmaker said it wouldnt bother asking European authorities to renew the $1-million-plus gene therapys marketing authorization when it expires in October, and comes after it abandoned plans to gain an approval in the U.S. Reports from MIT Technology Review suggest only one patient ever used the med.

GlaxoSmithKline has also been struggling in Europe with its bubble boy syndrome gene therapy Strimvelis. Mindful of Glyberas cost, GSK put its price tag at half that of Glybera, at $665,000, and also offered a money-back guarantee.

Its been approved in Europe for nearly a year, but it only treated its first patient this month, according to Business Insider.

Treatment is tough as the drug is not so much manufactured as it is created for each individual patient, with a site in Italy currently the only approved site in the world for this type of manufacture, and thus the only place where patients can be treated. Only around 15 patients in Europe are believed to have the condition.

Other biotechs are however working on the manufacturing side in order to try and make these therapies more available for patients, and thus open up their viability.

There are already a number of medications on the market for hemophilia, such as from Biogen spin-off Bioverativ and Sobi, with gene therapy predicted by some also working in the space, including uniQure and BioMarin, to be the next class for treating the blood disorder.

But speaking to FierceBiotech at the start of the year, Bioverativs new chief and former Biogen exec John Cox told me that while they are to working on gene therapy approaches to hemophilia, there are reasons to be cautious: There are of course question marks over gene therapy: The obvious one is safety, because of the history here, and this is a risk-averse population, for good reason, and the other question is naturally over efficacy, and how long does it last, as well as manufacturing, scale and so on.

Were all hoping for a cure, and of course were doing work on gene therapy now, but I dont think people are looking at these now as a permanent cure; the questions are over durability, rather than cure.

He said that investors and even doctors talk a lot about gene therapy in the hemophilia space, but that if you talk to hemophilia A patients about what they really want, being able to dose, once a week [which is the target with its candidate, or even just less frequently, is what they want.

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Pfizer doubles down on gene therapy pipeline with $70M Sangamo buy-in – FierceBiotech

Gene Therapy Advanced by Structural Study of Potential Viral Vectors – Genetic Engineering & Biotechnology News

Scientists led by Associate Professor Vijay Reddy, Ph.D., at The Scripps Research Institute (TSRI) say they have discovered the structural details that make one virus a better tool for future therapies than a closely related virus. The team wrote inScience Advances(“Cryo-EM structure of human adenovirus D26 reveals the conservation of structural organization among human adenoviruses”) thatthe structure of a less prevalent species D adenovirus may work well as a gene-delivery vector because its structure doesn’t allow it to wind up in the liver, thus minimizing liver toxicity. The Reddy Lab’s study reportedly is the first to show the structural details on species D’s surface that set it apart from another common subtype of adenovirus, called species C, which does travel to the liver.

“Greater understanding of the structures of adenoviruses from different species will help generate better gene therapies and/or vaccine vectors,” said Dr. Reddy.

Using cryo-electron microscopy, the researchers discovered that while these two species of adenoviruses share the same shell-like core, they have different surface structures called decorations or loops, whichare key to a virus’s behavior. They determine which receptors on human cells the virus can bind to. For species C adenoviruses, specific loops help the virus attach to blood coagulation factors (adaptor proteins) and get targeted to the human liver.Species D adenoviruses display distinctly different loop decorations. For use in gene and vaccine therapies, the virus would deliver helpful genes instead.

Species D also has one more important advantage over species C: Humans are constantly exposed to species C adenoviruses, so most people have developed antibodies to fight them off. These same antibodies would fight off the species C viruses even if they were designed for beneficial therapies. On the flip side, many of the species D adenoviruses are rare, and it’s unlikely that a patient would have antibodies to fight them off, according to the investigators. That makes species D viruses better for delivering therapies, explains Dr. Reddy, who adds that scientists are already testing ways to use it to generate malaria and Ebola virus vaccines.

The researchers next plan to look at members of the other five species of adenoviruses to see if they would have useful traits as viral therapy vectors.

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Gene Therapy Advanced by Structural Study of Potential Viral Vectors – Genetic Engineering & Biotechnology News

Global Gene Therapy Technologies, Markets and Companies Research Report 2017-2026 – Research and Markets – PR Newswire (press release)

Gene therapy can now combine with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 187 companies involved in developing gene therapy are presented along with 232 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade.

Key Topics Covered:

Part I: Technologies & Markets

Executive Summary

1. Introduction

2. Gene Therapy Technologies

3. Clinical Applications of Gene Therapy

4. Gene Therapy of Genetic Disorders

5. Gene Therapy of Cancer

6. Gene Therapy of Neurological Disorders

7. Gene Therapy of Cardiovascular Disorders

8. Gene therapy of viral infections

9. Research, Development and Future of Gene Therapy

10. Regulatory, Safety, Ethical Patent Issues of Gene Therapy

11. Markets for Gene Therapy

12. References

Part II: Companies

13. Companies involved in Gene Therapy

For more information about this report visit http://www.researchandmarkets.com/research/9jzl3f/gene_therapy

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-gene-therapy-technologies-markets-and-companies-research-report-2017-2026—research-and-markets-300455149.html

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Global Gene Therapy Technologies, Markets and Companies Research Report 2017-2026 – Research and Markets – PR Newswire (press release)

Sangamo Therapeutics And Pfizer Announce Collaboration For Hemophilia A Gene Therapy – PR Newswire (press release)

“With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program,” said Dr. Sandy Macrae, Sangamo’s Chief Executive Officer. “We believe Pfizer’s end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases.”

Under the terms of the collaboration agreement, Sangamo will receive a $70 million upfront payment from Pfizer. Sangamo will be responsible for conducting the SB-525 Phase 1/2 clinical study and certain manufacturing activities. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for SB-525 and additional products, if any. Sangamo is eligible to receive potential milestone payments of up to $475 million, including up to $300 million for the development and commercialization of SB-525 and up to $175 million for additional Hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will also receive tiered double-digit royalties on net sales. Additionally, Sangamo will be collaborating with Pfizer on manufacturing and technical operations utilizing viral delivery vectors.

Gene therapy is a potentially transformational technology for patients, focused on highly specialized, one-time, treatments that address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patient’s cells to compensate for a defective one. The genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector such as rAAV. There have been no gene therapy products approved in the U.S. to date.

Hemophilia A is a rare blood disorder caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the U.S. and more than 150,000 worldwide with Hemophilia A. SB-525 is comprised of a rAAV vector carrying a Factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter. The U.S. Food and Drug Administration has cleared initiation of human clinical trials for SB-525, which also has been granted orphan drug designation. Sangamo is on track this quarter to start a Phase 1/2 clinical trial to evaluate safety and to measure blood levels of Factor VIII protein and other efficacy endpoints.

Conference CallSangamo will host a conference call today, May 10, 2017 at 5:00 p.m. ET, which will be open to the public, to discuss the details of the collaboration and the Company’s first quarter business and financial results. The call will also be webcast live and can be accessed via a link the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A replay of the webcast will also be available for one week after the call.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 15225000. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on May 10, 2017 to 11:59 p.m. ET on May 17, 2017. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 15225000.

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

Forward Looking Statements This press release may contain forward-looking statements based on Sangamo’s current expectations. These forward-looking statements include, without limitation references relating to the collaboration agreement with Pfizer, potential milestone payments and royalties under the collaboration agreement, ability of the collaboration to advance and commercialize SB-525 as a treatment for Hemophilia A, research and development of therapeutic applications of Sangamo’s genomic therapy platforms, the expected timing of clinical trials of lead programs, including SB-525 and the release of data from these trials, the impact of Sangamo’s clinical trials on the field of genetic medicine and the benefit of orphan drug status. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to substantial dependence on the clinical success of lead therapeutic programs, the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo’s ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo’s SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo Therapeutics, Inc. assumes no obligation to update the forward-looking information contained in this press release.

Pfizer and Rare DiseaseRare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide,i representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including hematology, neuroscience, and inherited metabolic disorders.ii

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness and meet the needs of patient families.

Pfizer Inc: Working together for a healthier worldAt Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world’s best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us. For more information, please visit us at http://www.pfizer.com. In addition, to learn more, follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Pfizer Disclosure Notice: The information contained in this release is as of May 10, 2017. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about an investigational Hemophilia A agent, SB-525, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical study commencement and completion dates as well as the possibility of unfavorable study results, including unfavorable new clinical data and additional analyses of existing clinical data; risks associated with initial data, including the risk that the final results of the Phase I/2 study for SB-525 and/or additional clinical trials may be different from (including less favorable than) the initial data results and may not support further clinical development; whether and when any applications may be filed with regulatory authorities for SB-525; whether and when regulatory authorities may approve any such applications, which will depend on the assessment by such regulatory authorities of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of SB-525; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2016 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

i Rare Disease: Facts and Statistics. http://globalgenes.org/rare-diseases-facts-statistics. Accessed September 7, 2016. ii Pfizer Inc. Rare Disease. http://www.pfizer.com/health-and-wellness/health-topics/rare-diseases/areas-of-focus. Accessed December 20, 2016.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-and-pfizer-announce-collaboration-for-hemophilia-a-gene-therapy-300455555.html

SOURCE Sangamo Therapeutics, Inc.

http://www.sangamo.com

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Sangamo Therapeutics And Pfizer Announce Collaboration For Hemophilia A Gene Therapy – PR Newswire (press release)

Brammer invests in commercial-ready gene therapies – BioPharma-Reporter.com

Brammer Bio says its commercial-scale gene therapy manufacturing facility in Cambridge, Massachusetts, US, will open in the second half of 2017.

The contract development and manufacturing organization (CDMO) said the site offers process development, clinical phase, and current Good Manufacturing Practices (cGMP) services for cell and gene therapies.

We are delighted to add an experienced commercial biologics team and facilities to help meet the needs of this transformative industry, said Mark Bamforth, CEO of Brammer.

Brammer has completed a Type-C meeting with the US Food and Drug Administration (FDA) to assess the plans for the Massachusetts-based site. A Type-C meeting regards the development and review of drugs or biological drug products regulated by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).

A Site Master File will be submitted later this year to support Brammers clients product applications.

The site originally housed Biogens clinical and commercial biologics manufacturing facility.

Brammer purchased it along with Biogens distribution center in Somerville, Massachusetts, on January 1, 2017.

The Somerville site offers Brammer nearby storage and distribution capabilities.

The announcement follows Brammers 2016 plans to renovate its 50,000 square-foot facility in Lexington, Massachusetts, to cater for late stage and commercial therapy supply.

Brammers facilities now offer 230,000 square feet of development, distribution and cGMP manufacturing capabilities across Florida and Massachusetts, US.

More:
Brammer invests in commercial-ready gene therapies – BioPharma-Reporter.com

Brammer invests in commercial-ready gene therapies – OutSourcing-Pharma.com

Brammer Bio says its commercial-scale gene therapy manufacturing facility in Cambridge, Massachusetts, US, will open in the second half of 2017.

The contract development and manufacturing organization (CDMO) said the site offers process development, clinical phase, and current Good Manufacturing Practices (cGMP) services for cell and gene therapies.

We are delighted to add an experienced commercial biologics team and facilities to help meet the needs of this transformative industry, said Mark Bamforth, CEO of Brammer.

Brammer has completed a Type-C meeting with the US Food and Drug Administration (FDA) to assess the plans for the Massachusetts-based site. A Type-C meeting regards the development and review of drugs or biological drug products regulated by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).

A Site Master File will be submitted later this year to support Brammers clients product applications.

The site originally housed Biogens clinical and commercial biologics manufacturing facility.

Brammer purchased it along with Biogens distribution center in Somerville, Massachusetts, on January 1, 2017.

The Somerville site offers Brammer nearby storage and distribution capabilities.

The announcement follows Brammers 2016 plans to renovate its 50,000 square-foot facility in Lexington, Massachusetts, to cater for late stage and commercial therapy supply.

Brammers facilities now offer 230,000 square feet of development, distribution and cGMP manufacturing capabilities across Florida and Massachusetts, US.

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Brammer invests in commercial-ready gene therapies – OutSourcing-Pharma.com

Clinical trial on the horizon for RP genetic therapy – AOP

Researchers hope to use advanced gene editing technology to reverse the course of retinitis pigmentosa

09 May 2017 by Selina Powell

American researchers have used the gene-editing tool CRISPR/Cas9 to reverse the course of retinitis pigmentosa (RP) and restore vision in mice.

The study, published in Cell Research, involved using the genetic tool to prevent the degeneration of the light-sensing cells.

CRISPR/Cas9 allows scientists to target specific sections of genetic code and edit DNA at precise locations.

By deactivating certain genes, researchers were able to transform rod photoreceptors into cone photoreceptors cells that are less vulnerable to the genetic mutations that cause RP.

Senior author, Dr Kang Zhang, explained to OT that traditional RP gene therapy approaches targeted a single gene or mutation.

However, he emphasised that since there were many mutations and genes causing RP, many patients were left without treatment options.

Our gene therapy approach will create a universal, cost-effective gene therapy treatment for a majority, if not all, RP patients, Dr Zhang elaborated.

Dr Zhang emphasised that a common cold virus was being used as a gene therapy vector and had a good safety profile in clinical trials.

It was expected that a preclinical study would be conducted over the coming months, and a human trial would be undertaken early next year.

Read more:
Clinical trial on the horizon for RP genetic therapy – AOP

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