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Archive for the ‘Gene Therapy Research’ Category

Gene Therapy – Sickle Cell Anemia News

Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administrationapprovedthe first gene therapy for an inherited disease a genetic form of blindness in December 2017.

Sickle cell anemia is caused by a mutation in the HBB gene which provides the instructions to make part of hemoglobin, the protein in red blood cells that carries oxygen.

Researchers are working on two different strategies to treat sickle cell anemia with gene therapy. Both of these strategies involve genetically altering the patients own hematopoietic stem cells. These are cells in the bone marrow that divide and specialize to produce different types of blood cells, including the red blood cells.

One strategy is to remove some of the patients hematopoietic stem cells, replace the mutated HBB gene in these cells with a healthy copy of the gene, and then transplant those cells back into the patient. The healthy copy of the gene is delivered to the cells using a modified, harmless virus. These genetically corrected cells will then hopefully repopulate the bone marrow and produce healthy, rather than sickled, red blood cells.

The other strategy is to genetically alter another gene in the patients hematopoietic stem cells so they boost production of fetal hemoglobin a form of hemoglobin produced by babies from about seven months before birth to about six months after birth. This type of hemoglobin represses sickling of cells in patients with sickle cell anemia, but most people only produce a tiny amount of it after infancy. Researchers aim to increase production of fetal hemoglobin in stem cells by using a highly specific enzyme to cut the cells DNA in the section containing one of the genes that suppress production of fetal hemoglobin. When the cell repairs its DNA, the gene no longer works and more fetal hemoglobin is produced.

Gene therapy offers an advantage over bone marrow transplant, in that complications associated with a bone marrow donation now the only cure for the disease such as finding the right match are not a concern.

Twelve clinical trials studying gene therapy to treat sickle cell anemia are now ongoing. Nine of the 12 are currently recruiting participants.

Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene. These Phase 2 trials are recruiting both children and adults in the United States and Jamaica.

Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use ofMozobil (plerixafor) in patients with sickle cell anemia to increase the production of stem cells to be used for gene therapy. This medication is already approved to treat certain types of cancer. All three are recruiting U.S. participants.

One trial (NCT00669305) is recruiting sickle cell anemia patients in Tennessee to donate bone marrow to be used in laboratory research to develop gene therapy techniques.

The final study(NCT00012545) is examining the best way to collect, process and store umbilical cord blood from babies with and without sickle cell anemia. Cord blood contains abundant stem cells that could be used in developing gene therapy for sickle cell anemia. This trial is open to pregnant women in Maryland both those who risk having an infant with sickle cell anemia, and those who do not.

One clinical trial (NCT02151526) conducted in France is still active but no longer recruiting participants. It is investigating the efficacy of gene therapy in seven patients. For the trial, a gene producing a therapeutic hemoglobin that functions similarly to fetal hemoglobin is introduced into the patients stem cells. A case studyfrom one of the seven was published in March 2017; it showed that the approach was safe and could be an effective treatment option for sickle cell anemia.


Sickle Cell Anemia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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Gene Therapy - Sickle Cell Anemia News

What are the ethical issues surrounding gene therapy …

Because gene therapy involves making changes to the bodys set of basic instructions, it raises many unique ethical concerns. The ethical questions surrounding gene therapy include:

How can good and bad uses of gene therapy be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

Current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed to a persons children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the inserted gene to be passed to future generations. This approach is known as germline gene therapy.

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they cant choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.

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Gene therapy reverses rare immune disorder | National …

April 30, 2019

Children born with a rare genetic disorder called X-linked severe combined immunodeficiency (X-SCID) dont have a functioning immune system. As a result, they cant fight off infections. Without treatment, an infant with X-SCID will usually die within the first year or two of life.

The best option for treatment of newly diagnosed infants with X-SCID has been stem-cell transplantation from a genetically matched sibling. But less than a quarter of children with X-SCID have a matched donor available. For those without a matched donor, standard treatment has been a half-matched bone marrow transplant from a parent. But most infants receiving this type of transplant only have part of their immune system, called T lymphocytes, restored. These infants will need lifelong injections of protective antibodies. In addition, as they grow into young adulthood, they may have chronic medical problems that affect growth, nutrition, and quality of life.

To develop a better approach to fix the immune systems of children with X-SCID, researchers have used gene therapy to alter patients own blood stem cells. An engineered virus brings a healthy copy of the gene into the stem cells to replace the mutated gene that causes the disease.

Early results from trials of gene therapy for X-SCID resulted in life-saving correction of T lymphocytes. But similar to bone marrow transplant from a parent, the immune restoration was incomplete. In addition, in those first gene therapy studies, almosta third of the children developed leukemia. The approach accidentally stimulated cells to grow uncontrollably. In later studies, improved design of the engineered virus didnt cause cancer, but also didnt fully restore a healthy immune system.

In 2010, Dr. Harry Malech of NIHs National Institute of Allergy and Infectious Diseases (NIAID) and Dr. Brian Sorrentino of St. Jude Childrens Research Hospital reported a new and safer version of gene therapy for X-SCID. They designed a harmless engineered virus (called a lentivector) that could deliver genes into cells without activating other genes that can cause cancer. Before the altered stem cells were returned to their bodies, patients were given low doses of the chemotherapy drug busulfan. This made it easier for the new stem cells to grow in the bone marrow. In young adults and children treated at the NIH Clinical Center, the new therapy proved to be both safe and effective at restoring the full range of immune functions.

Based on this work, a team led by Dr. Ewelina Mamcarz of St. Jude Childrens Research Hospital began treatment in 2015 of newly diagnosed infants with X-SCID using the lentivector and busulfan. The work was funded in part by NHLBI. The team described the treatment of eight infants with the disorder on April 18, 2019, in the New England Journal of Medicine.

By 3 to 4 months after infusion of the repaired stem cells, 7 of the 8 infants had normal levels of multiple types of immune cells in their blood. The last infant required a second stem-cell infusion, after which his immune-cell levels rose to a normal range.

The infants new immune systems were able to fight off infections that the researchers had detected before the gene therapy. Four of the eight discontinued immune-system boosting medications that theyd previously needed. Of those four, three developed antibodies in response to vaccination, indicating a fully functional immune system.

A year and a half after gene therapy, all children were healthy and growing normally.

The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our continuing study at NIH is unprecedented, Malech says.

The researchers will continue to follow the participants over time. They plan to track how the childrens immune systems develop and look for any late side effects.

References:Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. PMID: 30995372.

Funding:NIHs National Institute of Allergy and Infectious Diseases (NIAID); National Heart, Lung, and Blood Institute (NHLBI); and National Cancer Institute (NCI); American Lebanese Syrian Associated Charities; California Institute of Regenerative Medicine; and Assisi Foundation of Memphis.

Gene therapy reverses rare immune disorder | National ...

Gene Therapy Market Emerging Trends, Growth and New …

Global Gene Therapy Market report offers clients the most efficient and dependable insight into the Gene Therapy market, ranging across different major players.

Pune, India April 29, 2019

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Gene Therapy Market Emerging Trends, Growth and New ...

International Cell and Gene therapy Conferences | Gene …

About Conference

About Conference

In continuation to 1st successful past scientific meeting, 2nd EuroSciCon Conference on Cell and Gene Therapy will be held on April 08-09, 2019 on Paris, France.

EuroSciCon suggests every single person to attend "Genetherapy 2019 in the midst of April 08-09, 2019 at Paris, France which merges brief keynote introductions, speaker talks, Exhibitions, Symposia, Workshops.

Genetherapy 2019 will gather world-class educators, researchers, analysts, Molecular Biologist, Gene therapists , Young Researchers working in the related fields to consider, exchange views and their experiences before an extensive worldwide social occasion of individuals. The social gathering warmly welcomes Presidents, CEO's, Delegates and present day experts from the field of Gene therapy and Public wellbeing and other pertinent organization positions to take an interest in these sessions, B2B get together and board talks. The assembly of this event will be revolving around the topic Exploring the possibilities and breakthroughs in cell and gene therapy.

EuroSciCon is the longest running independent life science events company with a predominantly academic client base. Our multi professional and multi-specialty approach creates a unique experience that cannot be found with a specialist society or commercially. EuroSciCon are corporate members of the following organizations: Royal Society of Biology, IBMS Company.

This global meeting gives the chance to Molecular Biologist, Gene Therapists, young researchers, specialists and analysts throughout the world to assemble and take in the most recent advances in the field of Cell and Gene Therapy and to trade innovative thoughts and encounters.

2 days of scientific exchange

100+ abstracts submitted

20+ scientific sessions

50+ worldwide professionals

80+ healthcare experts

Genetherapy 2019 is the yearly gathering directed with the help of the Organizing Committee Members and individuals from the Editorial Board of the supporting cell and Gene therapy related journals.

Reason to attend?

Genetherapy 2019 is relied upon to give young researchers and scientists a platform to present their revolutions in the field of Cell and Gene Therapy. This conference invites Presidents, CEO's, Delegates and present day specialists from the field of Cell and Gene Therapy and Public wellbeing and other pertinent organization positions to take an interest in this sessions, B2B get together and board talks.

About City:

Paris, the world's most popular city destination, has plenty of must-see places but make sure you spend at least a day strolling off the beaten path, as this is the only way to discover the real Paris: a lively cosmopolitan but undeniably French city.

The city is known for its cafe culture and designer boutiques along the Rue du Faubourg Saint-Honor. Paris is the city of love, inspiration, art and fashion. It has a population of more than 2million people and is divided into 20 districts. Paris has a lot of interesting architecture and museums to offer; among them the famous tourist place to visit is the Eiffel Tower. A significant number of the acclaimed roads and city building areas structures where changed by Haussmann and Napoleon III (Charles Louis Napoleon Bonaparte). The lanes where made much wider, places and squares where fabricated and the structures totally modified. Paris has a nickname called La Ville-Lumiere. The famous places to visit in Paris are Notre Dame Cathedralwhich is Roman Catholic Cathedral situated in the eastern half of the city, Louvre Museum which is located at the heart of Paris , Champs Elysees which is a Arc of Triumph, Montmartre which is a hill located at the north of Paris and its height is 130 metre, it is best known White Domed Basilica of the sacred heart at the top, Quartier Latin which is called the famous private garden located on the left bank of the seine around the Sorbonne, Disneyland Paris which is located 32 km from central Paris , it has two theme parks Disneyland and Walt Disney studios.

Track 1: Cell Science Research

Cell Science Research examines cells their physiological properties, their structure, the organelles they contain, interchanges with their condition, their life cycle, division, end and cell work.

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Track 2: Cell & Gene Therapy

Quality treatment is described as a plan of approaches that modify the announcement of a man's characteristics or repair bizarre characteristics. Each system incorporates the association of a specific nucleic destructive (DNA or RNA). Nucleic acids are frequently not taken up by cells, henceforth exceptional transporters; implied 'vectors' are required. Vectors can be of either mainstream or non-viral nature however Cell treatment is portrayed as the association of living whole cells into the patient for the treatment of a disease. The start of the cells can be from a comparable individual (autologous source) or from another individual (allogeneic source). Cells can be gotten from undifferentiated life forms, for instance, bone marrow or induced pluripotent central microorganisms (iPSCs), rethought from skin fibroblasts or adipocytes. Youthful microorganisms are associated with respect to bone marrow transplantation particularly. Distinctive methods incorporate the utilization of basically create cells, isolated in vitro (in a dish) from essential microorganisms.

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Track 3: Regenerative Medicine

Regenerative Medicine implies a social affair of biomedical approaches to manage investigate and clinical applications which are away to supplant or "recouping" human cells, tissues or organs to restore or set up conventional limits which were vexed on account of afflictions. The field of Regenerative medication has pulled in much thought as it holds the assurance of recuperating hurt tissues and organs in the body by supplanting hurt tissue or by strengthening the body's own repair segments to patch hurt tissues or organs. It in like manner may enable analysts to create tissues and organs in the lab and safely install them inside the body. Regenerative courses of action subsequently can be a dynamic progress in the field of therapeutic administrations.

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Track 4: Immunotherapy

Due to rapidly pushing field of tumor immunology as of late, there has been age of a couple of new procedures for treating development called Immunotherapies. Immunotherapy is a sort of treatment that extends the nature of safe response against tumors either by enabling the activities of specific sections of safe structure or by checking signals conveyed by illness cells that cover safe responses. A couple of sorts of immunotherapy are also called as biologic treatment or biotherapy. Late movements in development immunotherapies have given new supportive systems. These consolidate tumor-related macrophages as treatment centers in oncology, in-situ commencement of platelets with checkpoint inhibitors for post-careful development immunotherapy, safe checkpoint blockade and related endocrinopathies and some more.

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Track 5 : Genetics and stem cell biology

An undifferentiated mass of cell in a multicellular animal which is prepared for offering rise to uncertain number of cells of a comparable sort, and from which certain diverse sorts of cell rise by detachment. Undifferentiated life forms can isolate into specific cell creates. The two describing characteristics of an undifferentiated cell are endless self-restoration and the ability to isolate into a specific adult. There are two critical classes of youthful microorganisms: pluripotent that can end up being any cell in the adult body, and multipotent that is kept to transforming into a more limited masses of cells.

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Track 6: Epigenetics

The examination of changes in living creatures caused by alteration of quality verbalization instead of adjustment of the inherited code itself. Epigenetics are unfaltering heritable characteristics that can't be cleared up by changes in DNA progression.

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Track 7: Human Genomics

The human genome is the total arrangement of nucleic corrosive groupings for people, encoded as DNA inside the 23 chromosome combines in cell cores and in a little DNA particle found inside individual mitochondria. Human genomes incorporate both protein-coding DNA genes and noncoding DNA

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Track 8: Next Generation Sequencing

Deoxyribonucleic destructive, for the most part known as DNA, contains the outlines of life. Inside its structures are the codes required for the party of proteins and non-coding RNA these sub-nuclear mechanical assemblies impact all the natural systems that make and care forever. By understanding the game plan of DNA, examiners have had the ability to outline the structure and limit of proteins and what's more RNA and have gotten a cognizance of the essential purposes behind ailment. Front line Sequencing (NGS) is an able stage that has enabled the sequencing of thousands to countless iotas in the meantime. This compelling device is evolving fields, for instance, redid medicine, inherited infections, and clinical diagnostics by offering a high throughput elective with the capacity to progression various individuals meanwhile.

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Track 9: Gene Editing and CRISPR Based Technologies

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) Technology is a champion among the most fit yet clear mechanical assembly for genome changing. It urges and empowers investigators to easily change DNA groupings and modify quality limits. It has various potential applications that join helping innate disseminates, treating and keeping the spread of diseases and improving yields. CRISPR broadly used as CRISPR-Cas9 where CRISPRs are particular stretches out of DNA and Cas9 is the protein which is an aggravate that exhibitions like a few nuclear scissors, fit for cutting DNA strands. The assurance of CRISPR advancement anyway raises moral stresses as it isn't 100% compelling. Regardless, the progression of CRISPR-Cas9 has disturbed the designed science industry these days, being a clear and great quality changing device.

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Track 10: Proteomics

Proteomics is the broad scale examination of proteomes. A proteome is a course of arrangement of proteins made in a living being, structure, or regular setting. We may imply, for instance, the proteome of a creature composes (for example, Homo sapiens) or an organ (for example, the liver). The proteome isn't relentless; it fluctuates from cell to cell and changes after some time. To some degree, the proteome reflects the key transcriptome. Regardless, protein activity (regularly reviewed by the reaction rate of the systems in which the protein is incorporated) is similarly changed by various components despite the verbalization level of the appropriate quality.

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Track 11: Viral Gene Therapy

Customary strategies for quality treatment fuse transfection. It twisted up clearly inefficient and confined fundamentally in view of movement of value into right now duplicating cells invitro. Quality treatment utilizes the transport of DNA into cells by techniques for vectors, for instance, natural nanoparticles or viral vectors and non-viral systems. The Several sorts of contaminations vectors used as a piece of value treatment are retrovirus, adenovirus, disease adeno-related and herpes simplex contamination. While other recombinant viral vector structures have been delivered, retroviral vectors remain the most surely understood vector system for quality treatment traditions and most prominent application on account of their certain significance as the essential vectors made for compelling quality treatment application and the soonest phases of the field of value treatment.

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Track 12: Cell Therapy of Cardiovascular Disorders

Cardiovascular contaminations have transformed into a growing clinical issue all around. the other test in the treatment of the cardiovascular disease is cell transplantation or cell cardiomyoplasty. Exceptional ischaemic harm and relentless cardiomyopathies incite unending loss of cardiovascular tissue and in the end heart disillusionment. Force medications wide mean to tighten the over the top changes that happen when harm and to cut back shot segments of vas diseases. Regardless, they don't improve the patient's close to home fulfillment or the figure more than coordinate. Unmistakable sorts of undifferentiated living beings have been used for primary microorganism treatment.

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Track 13: Regulatory and Safety Aspects of Cell and Gene Therapy

Cell treatment things require a combination of prosperity examinations. Comparable living being and quality things are heterogeneous substances. There are a few zones that particularly ought to be tended to as it is extremely not the same as that of pharmaceuticals. These range from making group consistency, thing soundness to thing prosperity, quality and sufficiency through pre-clinical, clinical examinations and displaying endorsement. This review plots the present headings/administers in US, EU, India, and the related challenges in making SCBP with highlight on clinical point.

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Track 14: Markets & Future Prospects for Cell & Gene Therapy

The immense number of associations related with cell treatment has extended development incredibly in the midst of the past couple of years. More than 500 associations have been recognized to be locked in with cell treatment and 305 of these are profiled 291 co-tasks. Of these associations, 170 are related with fundamental microorganisms. The Profiles of 72 academic establishments in the US related with cell treatment close by their business facilitated efforts. Allogeneic development with in excess of 350 clinical preliminaries is prepared to order the commercialization of cell medicines in publicize. Advance R&D in cell and quality treatment is depended upon to bloom given the normally based purposes of intrigue.

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Track 15: Gene therapy for Diseases

Gene therapy is the addition of particular genes at some particular locales into a person's cells or tissues to treat an illness, in which the inadequate or non-working quality is then supplanted with the working quality.

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Track 16: Stem Cell therapy

Stem Cell therapyis the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use. Recent studies are going on for the treatment ofSpinal cordinjury as well. Thus,Stem cell therapyhas a great scope in future as well.

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Track 17: Gene Editing

Gene Editing is where the defective gene is being expelled or supplanted from the genome, in order to change the imperfect type of quality to a working structure. Different methods, for example, gene substitution, gene knock out, gene knock down are utilized for this reason. Additionally, site coordinated mutagenesis has been broadly utilized for gene altering purposes.

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International Cell and Gene therapy Conferences | Gene ...

Highmark Health Blog | Gene Therapy Research: Dr. Passineau

Michael Passineau, PhD, is a man who speaks in metaphors. For good reason he works within a realm of medicine not many people understand. Director of the Gene Therapy Program at Allegheny Health Network (AHN) and a leading force behind AHNs scientific research to address clinical needs, Passineau leans into the way a good metaphor can bring clarity to challenging conceptsincluding the nature of his work.

I think of clinicians as chefs, he says. At the end of each day, theyve done something tangible. Theyve made a meal. Researchers, on the other hand, are a bit like sculptors. We can work for years on something, but once its complete, its permanent.

For over a decade, his research has revolved around gene therapy more specifically, the use of ultrasound technology, instead of viral administration, to deliver therapeutic DNA into the cells of salivary glands. The goal: restore saliva flow to patients who suffer from radiation-induced dry mouth, or xerostomia.

Supported by grants from the National Institutes of Health (NIH), Passineau, radiation oncologist Dr. Mark Trombetta, and their research team are on track to petition the U.S Food and Drug Administration for Investigational New Drug (IND) status, which would allow their work to move out of the lab and into Phase 1 clinical trials with humans.

Xerostomia is an iatrogenic complication, meaning it is caused by treatment in this case, head and neck radiation to treat cancer. When the beam of radiation passes through the head, it damages the salivary glands, resulting in chronic dry mouth. This can lead to permanent loss of function in the salivary glands, difficulty eating, and loss of teeth.

Its anything but trivial, says Passineau. To illustrate how this condition impacts a persons quality of life, I often have donors and executives take a piece of surgical gauze and chew on it while I describe my research. After about five minutes, they understand how difficult it really is.

While there are a few existing medications used to treat xerostomia, they are difficult to administer, and their effects are not long lasting. Most people deal with the condition by carrying a water bottle at all times or by taking saliva substitutes. Unfortunately, these options dont work particularly well.

Were all made of trillions of cells, says Passineau, beginning an attempt to explain gene therapy in a nutshell.

Each cell has a role to play, whether its beating heart muscle, growing hair and nails, or perceiving light signals in your retina, he continues. The way those biochemical machines are engineered is dictated by your genomic DNA, which is DNA you get from your parents. Those genes code for proteins, which are the gears and springs that make cells function as biochemical machines.

Gene therapy means reprogramming the cell changing the machine code telling the cell how to work. That requires getting new DNA to travel inside the cell. Passineau says this is one of the most difficult tasks in the world since our cells are designed to repel foreign DNA.

In nature, foreign DNA gets into human cells only through viral infection or during conception. Virally administered gene therapy approaches have been developed, but one drawback is that after a viral vector is introduced into the body, the immune system fights back and will also react to the vector on subsequent treatments, making them ineffective.

Thats where Passineaus research comes in. Weve developed a method of delivering genes that doesnt require viral administration, he explains. Instead, we use soundwaves.

To understand how ultrasonic administration, or sonoporation, works, Passineau turns again to metaphor.

Picture an agricultural pond, with a thick layer of algae on it. If you throw a ping-pong ball into the middle, it will just sit on top, he says. That is very much what a cell membrane is like the outer covering is rather rigid. So, to deliver the genes, we have to get through the cell membrane. It is only seven nanometers thick but its the longest seven nanometers in nature for someone like me.

Passineaus ground-breaking research uses soundwaves to temporarily alter the permeability of the cell membrane, allowing for the transfer of therapeutic DNA into the cell.

Lets understand how this works in our pond metaphor before getting into what that means for gene therapy.

Imagine we explode a grenade above the pond, Passineau says. For a moment, the layer of slime would open up, and youd see down to the bottom of the pond. Then, it would close again.

In Passineaus lab, the grenade is a mix of a gene drug for xerostomia known as Aquaporin-1 and a solution of microbubbles. Used routinely in cardiac imaging and other medical applications, microbubbles have a resonant frequency that can be used to create the desired explosion.

The classic example is a crystal glass if an opera singer hits the right frequency for that glass, it will vibrate. If she really turns up the volume, the glass will shatter, because it cant absorb the energy, Passineau says. That same thing happens with the microbubbles.

So after administering the microbubble and Aquaporin-1 solution to the treatment site, a low-frequency ultrasound beam is used to create an ultrasonic acoustic field in which the bubbles vibrate. Turn up the power, and the bubbles implode. That opens up the cell membrane long enough for the gene drug to get in, before it closes back up.

For gene therapy researchers like Passineau, the membrane around our cells is the longest seven nanometers in nature.

Sonoporation works well for what were doing in the salivary glands, but not so well for the heart, and certainly not for the brain, Passineau points out. However, we have other applications we intend to use this research for.

He explains that one promising use involves Sjogrens syndrome, an autoimmune disease affecting nearly 4 million Americans (90 percent of whom are women). The diseases debilitating symptoms include severe dry mouth, which may be treatable with Passineaus gene therapy technique.

Another research area he says he is excited about is the use of gene therapy to combat obesity and overeating. Do you remember when you were a kid and youd eat too fast and your mom would tell you to slow down because your brain didnt know whether or not your stomach was full yet? he asks. Well, that was absolutely true.

He explains that, when we eat, our intestines stretch and release a protein called peptide YY (PYY), which circulates through the blood, eventually entering the saliva and interacting with receptors on your tongue.

Think of your appetite as a glass of water, he says. To feel full, you have to fill the glass with PYY. Some people have bigger glasses than others, but if we can use gene therapy to modify saliva and make the glass start half full, then a person would feel full without needing to eat as much.

Passineau adds that poor health outcomes and high costs associated with obesity make this an attractive target for research investment. Obesity adds billions of dollars to the cost of medical care in the U.S. each year, and some studies estimate the cost as high as $190 billion per year.

If gene therapy was this easy, everyone would be doing it. Instead, as Passineau points out, it is one of the most difficult tasks in the world.

At AHN, research is a small but important piece of the operation, Passineau says. Its important to note that everything we do in research is driven by physicians who have recognized clinical needs, and who have partnered with us to develop novel solutions.

Similarly, looking at the value that research can deliver, and its potential impact on both health and overall health care costs, Passineau says that federal funding has an essential role in advancing further discoveries in areas like gene therapy and sonoporation. Government investment really is the lifeblood of what drives research, he says.

Another impact on the success and pace of advances in medical research is whether talented, driven young people decide to take this path. Passineau admits that, like the process of research itself, the path to becoming a successful researcher can be long and sometimes feels like three steps forward, two steps back. But if the work feels meaningful, its all worth it.

I landed where I am today because I figured out what I was good at, he says. Inventing, solving big picture problems and helping people.

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Highmark Health Blog | Gene Therapy Research: Dr. Passineau

Gene therapy restores immunity in infants with rare …

News Release

Wednesday, April 17, 2019

NIH scientists and funding contributed to development of experimental treatment

A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. Eight infants with the disorder, called X-linked severe combined immunodeficiency (X-SCID), received an experimental gene therapy co-developed by National Institutes of Health scientists. They experienced substantial improvements in immune system function and were growing normally up to two years after treatment. The new approach appears safer and more effective than previously tested gene-therapy strategies for X-SCID.

These interim results from the clinical trial, supported in part by NIH, were published today in The New England Journal of Medicine.

Infants with X-SCID, caused by mutations in the IL2RG gene, are highly susceptible to severe infections. If untreated, the disease is fatal, usually within the first year or two of life. Infants with X-SCID typically are treated with transplants of blood-forming stem cells, ideally from a genetically matched sibling. However, less than 20% of infants with the disease have such a donor. Those without a matched sibling typically receive transplants from a parent or other donor, which are lifesaving, but often only partially restore immunity. These patients require lifelong treatment and may continue to experience complex medical problems, including chronic infections.

"A diagnosis of X-linked severe combined immunodeficiency can be traumatic for families," said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID). These exciting new results suggest that gene therapy may be an effective treatment option for infants with this extremely serious condition, particularly those who lack an optimal donor for stem cell transplant. This advance offers them the hope of developing a wholly functional immune system and the chance to live a full, healthy life.

To restore immune function to those with X-SCID, scientists at NIAID and St. Jude Childrens Research Hospital in Memphis, Tennessee, developed an experimental gene therapy that involves inserting a normal copy of the IL2RG gene into the patients own blood-forming stem cells. The Phase 1/2 trial reported today enrolled eight infants aged 2 to 14 months who were newly diagnosed with X-SCID and lacked a genetically matched sibling donor. The study was conducted at St. Jude and the Benioff Childrens Hospital of the University of California, San Francisco. Encouraging early results from a separate NIAID-led study at the NIH Clinical Center informed the design of the study in infants. The NIH study is evaluating the gene therapy in older children and young adults with X-SCID who previously had received stem cell transplants.

The gene therapy approach involves first obtaining blood-forming stem cells from a patients bone marrow. Then, an engineered lentivirus that cannot cause illness is used as a carrier, or vector, to deliver the normal IL2RGgene to the cells. Finally, the stem cells are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to help the genetically corrected stem cells establish themselves in the bone marrow and begin producing new blood cells.

Normal numbers of multiple types of immune cells, including T cells, B cells and natural killer (NK) cells, developed within three to four months after gene therapy in seven of the eight infants. While the eighth participant initially had low numbers of T cells, the numbers greatly increased following a second infusion of the genetically modified stem cells. Viral and bacterial infections that participants had prior to treatment resolved afterwards. The experimental gene therapy was safe overall, according to the researchers, although some participants experienced expected side effects such as a low platelet count following chemotherapy.

"The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our study at NIH is unprecedented," said Harry Malech, M.D., chief of the Genetic Immunotherapy Section in NIAIDs Laboratory of Clinical Immunology and Microbiology. Dr. Malech co-led the development of the lentiviral gene therapy approach with St. Judes Brian Sorrentino, M.D., who died in late 2018. These encouraging results would not have been possible without the efforts of my good friend and collaborator, the late Brian Sorrentino, who was instrumental in developing this treatment and bringing it into clinical trials, said Dr. Malech.

Compared with previously tested gene-therapy strategies for X-SCID, which used other vectors and chemotherapy regimens, the current approach appears safer and more effective. In these earlier studies, gene therapy restored T cell function but did not fully restore the function of other key immune cells, including B cells and NK cells. In the current study, not only did participants develop NK cells and B cells, but four infants were able to discontinue treatment with intravenous immunoglobulins infusions of antibodies to boost immunity. Three of the four developed antibody responses to childhood vaccinations an indication of robust B-cell function.

Moreover, some participants in certain early gene therapy studies later developed leukemia, which scientists suspect was because the vector activated genes that control cell growth. The lentiviral vector used in the study reported today is designed to avoid this outcome.

Researchers are continuing to monitor the infants who received the lentiviral gene therapy to evaluate the durability of immune reconstitution and assess potential long-term side effects of the treatment. They also are enrolling additional infants into the trial. The companion NIH trial evaluating the gene therapy in older children and young adults also is continuing to enroll participants.

The gene therapy trial in infants is funded by the American Lebanese Syrian Associated Charities (ALSAC), and by grants from the California Institute of Regenerative Medicine and the National Heart, Lung, and Blood Institute, part of NIH, under award number HL053749. The work also is supported by NIAID under award numbers AI00988 and AI082973, and by the Assisi Foundation of Memphis. More information about the trial in infants is available on using identifier NCT01512888. More information about the companion trial evaluating the treatment in older children and young adults is available using identifier NCT01306019.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit

NIHTurning Discovery Into Health

E Mamcarz et al. Lentiviral gene therapy with low dose busulfan for infants with X-SCID. The New England Journal of Medicine DOI: 10.1056/NEJMoa1815408 (2019).


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Gene therapy restores immunity in infants with rare ...

Bubble boy disease: Doctors successfully treat SCID-X1 …

Researchers from St. Jude Childrens Research Hospital have cured babies with bubble boy disease through gene therapy. Angela Gosnell, Knoxville News Sentinel

MEMPHIS, Tenn. Researchers from St. Jude Childrens Research Hospital have cured babies with bubble boy disease through gene therapy involving a re-engineeredvirus, according to a newly published study.

St. Jude performed the therapy oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1) a genetic condition also known as "bubble boy" disease according to a study published in the New England Journal of Medicine's April 18 issue.

The diseaseprevents babies from developing an immune system to fight even routine infections.In January 2018, St. Jude researchers reported that babies in the trial developed fully functioning immune systems but would be monitored further to confirm its long-term benefits.

Corresponding authors Dr. Ewelina Mamcarz and Dr. Stephen Gottschalk from St. Jude Children's Research Hospital. St. Jude performed a new therapy oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1), a genetic condition called "bubble boy" disease, according to a study published in the New England Journal of Medicine's April 18 issue.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

Previous infections cleared in all infants, and all continued to grow normally, the study said of the results.

St. Jude and UCSF Benioff Childrens Hospital San Francisco treated the children enrolled in the clinical trial with gene therapy developed by St. Judes Brian Sorrentino, the studys senior author,who led groundbreaking gene therapy research before his death in November at 60 years old.

Brian Sorrentino(Photo: Courtesy of Memorial Park Funeral Home)

James Downing, CEO of St. Jude Children's Research Hospital, said it was the lifelong ambition of Sorrentino, a survivor of pediatric cancer, to develop a cure.

Were comfortable, I think, at this point stating this is a cure, Downing said. Only time will say this will be a durable, lifelong cure.

After the therapy, the babies received their standard vaccinations and are now living a normal life with fully functioning immune systems, St. Jude says. Ten infants have received the therapy so far.

Study co-author Stephen Gottschalk, chair of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, said the researchers hope the therapy will be a template for treating other blood disorders.

Newborns with bubble boy disease, caused by a mutation inside a specific gene,must be placed inprotective isolation because they lack a proper immune system. Contact with the outside world is a major infection risk.

Perhaps the most well-known person with the disease was David Vetter, who died in 1984 at 12 years old. He helped inspire the 1976 movie "The Boy in the Plastic Bubble."

David Vetter had to stay inside a bubble in Houston on Dec. 17, 1976. Vetter was born with a genetic disorder leaving him no natural immunity against disease. Vetter died in 1984.(Photo: AP)

Most with the disease die by age 2 without treatment.

This disease is called bubble boy disease because babies had to be kept in special plastic chambers to protect them from infections, said first and corresponding author Ewelina Mamcarzof the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy. We dont have these chambers now, we are more advanced, but we need to protect them from infections as simple as a common cold virus (that) can kill them.

The patients came to researchers between 2 and 14 months of age, Mamcarz said, with severe life-threatening infections.

The gene therapy works like this: A deactivated virus is inserted into the patients bone marrow, which deliversthe correct gene copy into blood stem cells, replacing the defective one. These cells are then frozen and undergo testing.

This virus is able to effectively deliver a healthy copy of the gene into a stem cell in a way that was not possible before, Mamcarz said.

The patient then receives two days of low-dose busulfan, a chemotherapy drug that makes space in the marrow for the stem cells to grow, and the cells are then infused back into the patient.

Dr. Ewelina Mamcarz, first and corresponding author of a study published in the New England Journal of Medicine about a therapy performed at St. Jude Children's Research Hospital oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1), a genetic condition called "bubble boy" disease.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

It takes about 10 days from the time the cells are taken outto when they are infused into the patient, Mamcarz said.

The proper immune cells were found within three months of the treatment in all but one patient, who needed a second dose of gene therapy, St. Jude says.

This novel approach has shown really outstanding results for the infants, Downing said. The treatment has fully restored the immune system in these patients, which wasnt possible before, and has no immediate side effects.

The gene therapy developed and produced at St. Jude differs from previous gene replacement efforts in part by not activating adjacent genes that could cause leukemia. The viruses are equipped with insulators to block that accidental activation.

Past gene therapy did not have insulators, which inadvertently caused leukemia, Gottschalk said.

Gael Jesus Pino Alva, 2, and his mother, Giannina Alva. Gael was treated with a new therapy designed to fight X-linked severe combined immunodeficiency (SCID-X1), a genetic condition known as "bubble boy" disease, at St. Jude Children's Research Hospital.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

Current treatments for bubble boydisease are limited. Bone marrow transplants from compatible sibling donors are the best bet, but most patientslack a properdonor.

Mamcarz said researchers would like to treat more patients and follow them for longer periods of time to see if the gene therapy performed in the clinicaltrial can truly be used as an upfront treatment, and it's still too early to determine costs.

But the results from the research are a first, and their approach could be used to eventually treatother disorders like sickle cell disease, she said.

The kids are cured because for the first time, we are able to restore all three types of cells that constitute a full immune system: T cells, B cells and NK cells, Mamcarz said. Our patients are able to generate a healthy, fully functioning immune system. That is the first for gene therapy.


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Downingsaid the search for a cure has been a journey spanning more than a decade. Early gene therapy studies with the viral vectorsled to leukemia, he said, causing the work to stall. But Sorrentino pushed on.

Brian Sorrentino decided we really needed to produce vectors we could trust in not inducing leukemia, Downingsaid.

The patients' quality of life following the treatmentshows theyindeed found a cure, Downing said.

The question will become, Will it be a durable cure? Will it last 10, 20, 50 years for these children? And only time will tell," he said.

Follow Max Garland on Twitter:@MaxGarlandTypes.


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Bubble boy disease: Doctors successfully treat SCID-X1 ...

Gene Therapy 2019 Global Market Outlook,Research,Trends …

WiseGuyReports.Com Publish a New Market Research Report On Gene Therapy 2019 Global Market Outlook,Research,Trends and Forecast to 2026.

Pune, India April 15, 2019

Gene Therapy Industry 2019


The global gene therapy market is anticipated to reach USD 4,300 million by 2021. The demand for gene therapy is primarily driven by continuous technological advancements and successful progression of several clinical trials targeting treatments with strong unmet need. Moreover, rising R&D spend on platform technologies by large and emerging biopharmaceutical companies and favorable regulatory environment will accelerate the clinical development and the commercial approval of gene therapies in the foreseeable future. Despite promise, the high cost of gene therapy represents a significant challenge for commercial adoption in the forecast period.

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Gene therapy involves inactivating a mutated gene that is not functioning properly and introducing a new gene to assist in fighting a disease. Overall, the field of gene therapy continues to mature and advance with many products in development and nearing commercialization. For instance, Spark Therapeutics received approval of Luxturna, a rare form inherited blindness in December 2017. Gene therapy market in late 2017 also witnessed the approvals of Gilead/Kite Pharmas Yescarta and Novartis Kymriah in the cancer therapeutic area.

Gene therapy offers promise in the treatment of range of indications in cancer and genetic disorders. Large Pharmaceuticals and Biotechnology companies exhibit strong interest in this field and key among them include Allergan, Shire, Biomarin, Pfizer and GSK. The gene therapy space is witnessing a wave of partnerships and alliances. Pfizer has recently expanded its presence in gene therapy with the acquisition of Bamboo Therapeutics and Allergan entered the field, with the acquisition of RetroSense and its Phase I/II optogenetic program.

North America holds a dominating position in the global gene therapy market which is followed by Europe and the Asia Pacific. The U.S. has maximum number of clinical trials ongoing followed by Europe. Moreover, the field of gene therapy in the U.S. and Europe continues to gain investor attention driven by success of high visible clinical programs and the potential of gene therapy to address strong unmet need with meaningful commercial opportunity. Moreover, the increasing partnerships and alliances and the disruptive potential of gene therapy bodes well for the sector through the forecast period.Key Findings from the study suggest products accessible in the market are much competitive and manufacturers are progressively concentrating on advancements to pick up an aggressive edge. Companies are in a stage of development of new items in order to guarantee simple implementation and connection with the current gene. The hospatility segment is anticipated to grow at a high growth rate over the forecast period with the expanding utilization of smart locks inferable from expanding security-related worries among clients amid their stay at the hotels. North America is presumed to dominate the global smart locks market over the forecast years and Asia Pacific region shows signs of high growth owing to the booming economies of India, and China.

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Table Of Contents Major Key Points

1. Gene Therapy Overview1.1. History and Evolution of Gene Therapies1.2. What is Gene Therapy1.3. Types of Gene Therapy1.4. Ex vivo and in vivo Approaches of Gene Therapy1.5. RNAi Therapeutics1.6. CAR-T Technology based Gene Therapy1.7. Types of Vectors used for Gene Therapy1.7.1. Viral1.7.2. Non-Viral

2. Historical Marketed Gene Therapies [2003-2012]2.1. Rexin-G (Epeius Biotechnologies Corporation)2.2. Gendicine (SiBiono GeneTech Co., Ltd)2.3. Neovasculgen [Human Stem Cells Institute (HSCI))2.4. Glybera (UniQure Biopharma B.V.)

3. First Countries to get an access to Gene Therapies3.1. Philippines for Rexin-G [2003]3.2. China for Gendicine [2003]3.3. Russia for Neovasculgen [2011]3.4. Selected European Countries for Glybera [2012]

4. Marketed Gene Therapies [Approved in Recent Years]4.1. KYMRIAH (tisagenlecleucel)4.1.1. Therapy Description4.1.2. Therapy Profile4.1.2.1. Company4.1.2.2. Approval Date4.1.2.3. Mechanism of Action4.1.2.4. Researched Indication4.1.2.5. Vector Used4.1.2.6. Vector Type4.1.2.7. Technology4.1.2.8. Others Development Activities4.1.3. KYMRIAH Revenue Forecasted till 20214.2. YESCARTA (axicabtagene ciloleucel)4.2.1. Therapy Description4.2.2. Therapy Profile4.2.2.1. Company4.2.2.2. Approval Date4.2.2.3. Mechanism of Action4.2.2.4. Researched Indication4.2.2.5. Vector Used4.2.2.6. Vector Type4.2.2.7. Technology4.2.2.8. Others Development Activities4.2.3. YESCARTA Revenue Forecasted till 20214.3. LUXTURNA (voretigene neparvovec-rzyl)4.3.1. Therapy Description4.3.2. Therapy Profile4.3.2.1. Company4.3.2.2. Approval Date4.3.2.3. Mechanism of Action4.3.2.4. Researched Indication4.3.2.5. Vector Used4.3.2.6. Vector Type4.3.2.7. Technology4.3.2.8. Others Development Activities4.3.3. LUXTURNA Revenue Forecasted till 20214.4. STRIMVELIS4.4.1. Therapy Description4.4.2. Therapy Profile4.4.2.1. Company4.4.2.2. Approval Date4.4.2.3. Mechanism of Action4.4.2.4. Researched Indication4.4.2.5. Vector Used4.4.2.6. Vector Type4.4.2.7. Technology4.4.2.8. Others Development Activities4.4.3. STRIMVELIS Revenue Forecasted till 2021

5. Comparison of current Regulatory Status for Gene Therapy Products5.1. U.S5.2. Europe5.3. Japan

6. Emerging Gene Therapies [Phase III]6.1. Gene Based Therapeutics under Development6.2. Therapy Description

7. Indication of Focus in Gene Therapy7.1. Cancer7.2. Neurodegenerative Disorders7.3. Lysosomal Storage Disorders (LSDs)7.4. Ocular Diseases7.5. Muscle Disorders7.6. Anemia7.7. Hemophilia7.8. Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency


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Gene Therapy 2019 Global Market Outlook,Research,Trends ...

Hemophilia Gene Therapy Market 2019 to Showing Impressive …

Apr 12, 2019 (The Expresswire via COMTEX) -- Hemophilia Gene Therapy Market report is a complete study of current trends in the Market, industry growth drivers, and restraints. It provides Market forecasts for the coming years

Global Hemophilia Gene Therapy Market report observes different predilections, obstructions, and difficulties looked by the best makers/Economy by Business Leaders contenders of Complete Reports Hemophilia Gene Therapy market. Our specialists' group has considered every single angle directly from the piece of the pie, size, status, and development.

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Synopsis of the Hemophilia Gene Therapy Market: -

Hemophilia Gene Therapymarket competition by top manufacturers/ Key player/ Economy by Business Leaders: Spark Therapeutics, Ultragenyx, Shire PLC, Sangamo Therapeutics, Bioverativ, BioMarin, uniQure, Freeline Therapeutics,. And More

Hemophilia is a rare bleeding disorder in which the blood does not clot normally. Hemophilia is a monogenic disease (a disease that is caused by a genetic defect in a single gene). There are two types of hemophilia caused by mutations in genes that encode protein factors which help the blood clot and stop bleeding when blood vessels are injured. Individuals with hemophilia experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. The most frequent forms of hemophilia affect males.

Hemophilia Gene Therapy Market Segment by Type covers:

Hemophilia Gene Therapy Market Segment by Applications can be divided into:

Scope of theHemophilia Gene Therapy MarketReport:

Hemophilia Gene Therapy Market Segment by Regions, regional analysis covers

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The report deeply displays the global Hemophilia Gene Therapy Market.

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The next part also sheds light on the gap between supply and consumption. Apart from the mentioned information,growth rateof Hemophilia Gene Therapy market in 2024is also explained.Additionally, type wise and application wise consumptiontables andfiguresof Hemophilia Gene Therapy marketare also given.

Table of Contents

Market Overview 1.1 Hemophilia Gene Therapy Introduction 1.2 Market Analysis by Type 1.3 Market Analysis by Applications 1.4 Market Analysis by Regions 1.4.1 North America (United States, Canada and Mexico) United States Market States and Outlook (2013-2023) Canada Market States and Outlook (2013-2023) Mexico Market States and Outlook (2013-2023) 1.4.2 Europe (Germany, France, UK, Russia and Italy) Germany Market States and Outlook (2013-2023) France Market States and Outlook (2013-2023) UK Market States and Outlook (2013-2023) Russia Market States and Outlook (2013-2023) Italy Market States and Outlook (2013-2023) 1.4.3 Asia-Pacific (China, Japan, Korea, India and Southeast Asia) China Market States and Outlook (2013-2023) Japan Market States and Outlook (2013-2023) Korea Market States and Outlook (2013-2023) India Market States and Outlook (2013-2023) Southeast Asia Market States and Outlook (2013-2023) 1.4.4 South America, Middle East and Africa Brazil Market States and Outlook (2013-2023) Egypt Market States and Outlook (2013-2023) Saudi Arabia Market States and Outlook (2013-2023) South Africa Market States and Outlook (2013-2023) Nigeria Market States and Outlook (2013-2023) 1.5 Market Dynamics 1.5.1 Market Opportunities 1.5.2 Market Risk 1.5.3 Market Driving Force 2 Manufacturers Profiles

3 Global Hemophilia Gene Therapy Market Analysis by Regions

4 Global Hemophilia Gene Therapy Market Competition, by Manufacturer

5 Sales Channel, Distributors, Traders and Dealers


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Hemophilia Gene Therapy Market 2019 to Showing Impressive ...

What is Gene Therapy? | Pfizer: One of the world’s premier …

Gene therapy is a technology aimed at correcting or fixing a gene that may be defective. This exciting and potentially transformative area of research is focused on the development of potential treatments for monogenic diseases, or diseases that are caused by a defect in one gene.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patients cells to compensate for a defective one, using a viral vector.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patients cells to compensate for a defective one, using a viral vector.

Viral vectors can be developed using adeno-associated virus (AAV), a naturally occurring virus which has been adapted for gene therapy use. Its ability to deliver genetic material to a wide range of tissues makes AAV vectors useful for transferring therapeutic genes into target cells. Gene therapy research holds tremendous promise in leading to the possible development of highly-specialized, potentially one-time delivery treatments for patients suffering from rare, monogenic diseases.

Pfizer aims to build an industry-leading gene therapy platform with a strategy focused on establishing a transformational portfolio through in-house capabilities, and enhancing those capabilities through strategic collaborations, as well as potential licensing and M&A activities.

We're working to access the most effective vector designs available to build a robust clinical stage portfolio, and employing a scalable manufacturing approach, proprietary cell lines and sophisticated analytics to support clinical development.

In addition, we're collaborating with some of the foremost experts in this field, through collaborations with Spark Therapeutics, Inc., on a potentially transformative gene therapy treatment for hemophilia B, which received Breakthrough Therapy designation from the US Food and Drug Administration, and 4D Molecular Therapeutics to discover and develop targeted next-generation AAV vectors for cardiac disease.

Gene therapy holds the promise of bringing true disease modification for patients suffering from devastating diseases, a promise were working to seeing become a reality in the years to come.

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What is Gene Therapy? | Pfizer: One of the world's premier ...

STAR Gene Therapy | Charcot-Marie-Tooth Association

The CMTA Is Accelerating Research Through Gene Therapy

The CMTA looks forward to a time when doctors are able to use genetic therapies to treat the root cause of CMT rather than prescribing medications or recommending surgery. We are already envisioning the possibilities that gene therapy holds for our community of 2.8 million people worldwide living with CMT. In fact, were leading the pursuit to explore gene therapy in CMT by expanding our Strategy to Accelerate Research (STAR) program and our STAR Advisory Board.

At the CMTA, we are already envisioning the possibilities that gene therapy holds for our community of 2.8 million people worldwide living with CMT. John Svaren, PhD, Chair, CMTA Scientific Advisory Board

Given the increased feasibility and applicability of gene therapy to CMT, the CMTA hosted a Gene Therapy Workshop in 2018. In response to invitations from CMTA board member Dr. Steven Scherer, more than 20 of the top gene therapy experts gathered for the inaugural CMT-centered workshop on gene therapy. This meeting included experts who have worked in related genetic and neuromuscular disease areas, as well as clinicians and scientists spearheading efforts toward gene therapy for CMT2D and CMT4J.

Building on this meeting, the CMTA is assembling the best experts to formulate gene therapy strategies for CMT2 and CMT1 subtypes. Four gene therapy experts, Beverly Davidson, PhD, at the University of Pennsylvania, Kleopas Kleopa, MD, at the Cyprus Institute of Neurology & Genetics, Scott Harper, PhD, at the Ohio State University School of Medicine, and Steven Gray, PhD, at the University of Texas Southwestern Medical Center have now joined the Scientific Advisory Board of the CMTA. Dr. Davidson is an acknowledged leader in the gene therapy field, and her extensive experience includes both academic research and commercial translation gene therapy approaches. Dr. Kleopa has shown proof of concept that gene therapy works in two mouse models of CMT: CMT1X and CMT4C. This strategy can capitalize on the CMT animal models that have been developed and characterized with CMTA support. Dr. Harper is collaborating with Robert Burgess, PhD, at the Jackson Laboratory to develop a gene therapy vector to be used in a treatment for CMT2D. Dr. Grays core expertise is in Adeno-Associated Virus (AAV) gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system, with application to CMT4J.

Our genes dictate many of our personal characteristics; however, mutations in genes cause genetic diseases, such as CMT. Scientists have been working for decades to modify or replace faulty genes with healthy ones to treat, cure or prevent disease. Fortunately, we are seeing significant progress on these efforts to provide gene therapy options for CMT. In fact, recent studies have provided an effective gene therapy for spinal muscular atrophy (SMA), a devastating disorder that affects the same motor neurons that are affected by CMT.

Sometimes the whole gene is duplicated, as in CMT1A, where a chromosome segment around the PMP22 gene is present in three copies instead of two. Alternatively, a part of a gene is defective or missing from birth, causing many of the other known forms of CMT. Any of these variations can disrupt the structure of the protein that is encoded by the affected gene, causing cellular problems that ultimately lead to disease.

In gene therapy, scientists can do one of several things depending on the problem with the gene. The simplest form of gene therapy is to simply provide a correct copy of the gene, which is the basis of the gene therapy for SMA. In variations of this approach, genes that are causing problems can be suppressed. One example of this was the recent demonstration that antisense oligonucleotides can be used to improve the neuropathy in rodent models of CMT1A. In addition, the exciting new field of genome editing using CRISPR technology has now made it possible to correct disease-causing mutations, and collaborative projects have already been initiated with leaders in this field

In order to insert new genes directly into cells, scientists use a vehicle called a vector that is genetically engineered to deliver the correct version of the gene. For example, viruses have a natural ability to deliver genetic material into cells, and therefore, can be used as vectors. While some viruses cause disease, virus vectors are highly modified to remove their ability to cause disease so that they can be safely used to carry therapeutic genes into human cells.

Gene therapy can be used to modify cells inside or outside the body. When its done inside the body, a doctor will inject the vector carrying the gene directly into the part of the body that has defective cells.

Before a company can market a gene therapy product for use in humans, the gene therapy product has to be tested for safety and effectiveness so that the Food and Drug Administration (FDA) can evaluate whether the risks of the therapy are acceptable in light of its potential benefits. Gene therapies have begun to receive FDA approval, and many gene therapies are in clinical trials.

At the CMTA, we believe gene therapy holds the promise to provide effective therapies for people living with CMT. As we continue to make great strides in this area, the CMTA is committed to helping speed the development of gene therapy approaches by investing in the most promising and groundbreaking gene therapy treatments that have the potential to benefit our community.

We are members of the National Organization for Rare Disorders (NORD), and they have put together a six-minute video to help answer questions frequently asked about gene therapy. We think this video will help you better understand the basics of gene therapy.

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STAR Gene Therapy | Charcot-Marie-Tooth Association

Gene Therapy to Treat Macular Degeneration – AMDF

In Boston, scientists are working at the frontier of genetic research in an attempt to cure Macular Degeneration, the leading cause of blindness in the U.S., an enormous task.

Rajendra Kumar-Singh: There are about 3 billion nucleotides in the human genome and just 1 small mistake is sufficient to cause a problem. And when that problem occurs it can lead to inherited retinal degeneration.

Dean Bok: The promises of gene therapy at this point in time are tremendous. In principal, one can replace a bad gene with a good one. Its easier to replace a gene thats recessive, where you need two bad ones in order to produce the disease, and thats where weve had success. The challenge is for genes that are dominant. You need to get rid of the bad guys before the good guys can do their work.

Rajendra Kumar-Singh: Because the source of inherited retinal degeneration is DNA, it makes sense to be able to deliver normal DNA to correct the defect and hence gene therapy is going to be a key player in trying to develop novel therapies for these inherited retinal degeneration.

Narrator: (Animation) An imbalance in the complement system, which helps to fight many diseases, can cause holes or, macs, to form in the macula. A protein called cd59 normally helps prevent this from occurring. At Tufts University they are seeking a way to increase this protein in people with macular degeneration.

Rajendra Kumar-Singh: We plan to express the same protein but at higher levels on the cells that are normally getting damaged in AMD and theoretically we hope to be able to prevent the formation of these macs on these cells. When we use gene therapy we are in fact putting back in a normal version of the gene, such as the protein that is produced from that is now normal and allows the cell to revert to a normal, healthy looking or healthy functioning cell. We can potentially inject just once directly into the eye and that may serve as a therapeutic for the lifetime of the patient whether it be dry AMD or wet AMD. Science is all about solving problems and I would love to be the one to be able to solve this problem and provide some sort of therapies to people who otherwise might potentially go blind. And I think Ill have fulfilled my role as a scientist if I can achieve that.

Rajendra Kumar-Singh, PhD, Professor of Ophthalmology and NeuroscienceTufts University

Dean Bok, Phd, Distinguished Professor of Neurobiology and OphthalmologyUCLA

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Gene Therapy to Treat Macular Degeneration - AMDF

Amicus Establishes Global Research and Gene Therapy Center …

New 75,000 sq. ft. State of the Art Facility in uCity Square Adjacent to Penn Campus

Strengthens Amicus Capabilities as a Leading Global Rare Disease Biotechnology Company

CRANBURY, N.J. and PHILADELPHIA, Feb. 26, 2019 (GLOBE NEWSWIRE) -- Amicus Therapeutics (FOLD) today announced it is establishing a new Global Research and Gene Therapy Center of Excellence in uCity Square in Philadelphia, PA, to advance its commitment to world-class science that makes a meaningful difference in the lives of people living with rare metabolic diseases. Philadelphia is a well-regarded ecosystem for biotechnology and gene therapy research and offers an ideal environment for Amicus to advance its pipeline, attract and retain top talent and foster external collaborations within the rare diseases.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, This Amicus Global Research and Gene Therapy Center of Excellence is an important next step in the evolution of our science, research and gene therapy capabilities. In considering locations, Philadelphia became the clear choice as a burgeoning hub for medical breakthroughs. The proximity to our collaborators at the University of Pennsylvania and other major academic centers and hospitals in the area also provides a tremendous opportunity to advance our commitment to gene therapies. Philadelphia is easily accessible to New Jersey, which has been a strong contributor to our success and will remain the location of our global headquarters. As Amicus continues to expand globally, my hope is that the great science to come from our research in Philadelphia will one day soon lead to medicines with the potential to alleviate an enormous amount of suffering. This is our mission at Amicus and we are honored to be a part of the exciting Philadelphia research community.

Under the leadership of Jeff Castelli, PhD, Chief Portfolio Officer and newly appointed Head of Gene Therapy, and Hung Do, PhD, Chief Science Officer, the new facility will be located at 3675 Market Street in uCity Square, a 6.5 million square-foot, mixed-use knowledge community consisting of office, laboratory, clinical, residential and retail space designed to enable university and corporate research, entrepreneurial activity and community engagement.

An initial group of Amicus research employees has moved into temporary space in the building at BioLabs@CIC Philadelphia during construction of the permanent space. The new 75,000 sq. ft. Center will be completed in the second half of 2019 and will serve as the headquarters for the global Amicus science organization and the gene therapy leadership team. Amicus expects up to 200 employees to eventually be based at the new Philadelphia facility. The Company is maintaining global business operations in Cranbury, NJ, and international headquarters in Marlow, UK.

J. Larry Jameson, MD, PhD, Executive Vice President for the Health System and Dean of the Raymond and Ruth Perelman School of Medicine stated, On behalf of Penn Medicine, I would like to welcome Amicus Therapeutics to Philadelphia. Amicus is working to pioneer significant advancements in gene therapy, which includes a collaboration with Dr. James Wilson and his team at our Orphan Disease Center. This relationship reflects how the innovation ecosystem at Penn brings together researchers, innovators, and entrepreneurs to accelerate research discoveries to patients as quickly as possible. The close proximity between the Amicus Center of Excellence and our campus will further strengthen this relationship and create additional opportunities to work together.

Jim Kenney, Mayor of Philadelphia, commented, The City of Philadelphia is committed to fostering innovative companies, academic institutions, and hospitals that are focused on the latest advancements in research and development, while also elevating the patient experience within our healthcare systems. Amicus Therapeutics is an established leader in biotechnology with a unique and intense patient-dedicated mission. The Companys presence and investment in Philadelphia will create additional opportunities that will be highly influential as our city continues its transformation into a major global biotech hub.

About Amicus Therapeutics Amicus Therapeutics (FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at, and follow us on Twitter and LinkedIn.

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Forward-Looking StatementsThis press release contains "forward- looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 Words such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, "confidence," "encouraged," potential, plan, targets, likely, may, will, would, should and could, and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2017 and Quarterly Report on 10-Q for the Quarter ended September 30, 2018. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.


Investors/Media:Amicus TherapeuticsSara Pellegrino, IRCVice President, Investor Relations & Corporate (609) 662-5044

Media:Amicus TherapeuticsMarco WinklerDirector, Corporate 662-2798


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Amicus Establishes Global Research and Gene Therapy Center ...

Journal of Stem Cell Research and Therapy- Open Access …

PUBMED NLM ID: 101586297 | Index Copernicus Value: 84.95 The Journal of Stem Cell Research & Therapy is an open access journal that showcases seminal research in the field of stem cell therapy. As stem-cells are flag-bearers of translational research, the field has an interdisciplinary feel by including oncology, clinical research, medicine and healthcare under the aegis of stem-cell therapy. It also includes scientific research related to the auxiliary areas of Biology by prioritizing scholarly communication milieu and transfers expert knowledge synthesized from the ever burgeoning stem-cell literature. In order to create such impactful content, the Journal of Stem Cell Research & Therapy brings together an expert Editorial Board, which comprises of noted scholars in the field of Cell Biology. Every single article is subjected to rigorous peer review by illustrious scientists. In addition to Research Articles, the Journal also publishes high quality Commentaries, Reviews, and Perspectives aimed at synthesizing the latest developments in the field, and putting forward new theories in order to provoke debates amongst the scholars in the field. The journal thus maintains the highest standards in terms of quality and comprehensive in its approach.The journal aims to provide the authors with an efficient and courteous editorial platform. The authors can be assured of an expeditious publishing process. In this regard, the journal also provides advance online posting of the accepted articles. The Journal of Stem Cell Research & Therapy ensures barrier-free, open access distribution of its content online and thus, helps in improving the citations for authors and attaining a good impact factor.

Scholarly Journal of Stem Cell Research & Therapy is using online manuscript submission, review and tracking systems of Editorial Manager for quality and quick review processing. Review processing is performed by the editorial board members of Journal of Stem Cell Research and Therapy or outside experts; at least two independent reviewers approval followed by editor approval is required for acceptance of any citable manuscript.

It is an undifferentiated cell which is capable of transforming into more cells of same type or multiple other types. They are found in multicellular organisms. They can differentiate into cells of blood, skin, heart, muscles, brain etc. In adult human being, they replenish the dead cells of various organs. Stem cells are being used for treatment of various diseases like diabetes, arthritis, few cancers, bone marrow failure etc.

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They can develop into any cell type or organ in the body. A single totipotent stem cell can give rise to an entire organism. Fertilized egg or a zygote is the best example. Zygote divides and produces more totipotent cells. After 4 days the cells lose totipotency and become pluripotent.

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They can differentiate into any cell type in the human body. Embryonic stem cells are mostly pluripotent stem cells. They have the ability to differentiate into any of three germ layers: endoderm, mesoderm, or ectoderm.

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Cancer Science & Therapy, Cervical Cancer: Open Access, Reproductive Medicine, Genetics & Stem Cell Biology, Stem Cell Reports, Hematology/ Oncology and Stem Cell Therapy, Journal of Stem Cells, International Journal of Stem Cells

These are multipotent stem cells normally found in the bone marrow and are derived from mesenchyme. They differentiate into adipocytes, chondrocytes, osteoblasts, myocytes and tendon. MSCs can also be extracted from blood, fallopian tube, fetal liver and lungs.

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They are the multipotent stem cells derived from mesoderm and located in red bone marrow. They are responsible for production of red blood cells, white blood cells and platelets. HSCs give rise to myeloid lineage (which forms erythrocytes, eosinophils, basophils, neutrophils, macrophages, mast cells and platelets) and lymphoid lineage (which forms T-lymphocytes, plasma cells and NK cells).

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They can differentiate into more than one cell type, but only into a limited number of cell types. Hematopoietic stem cells are considered multipotent as they can differentite into red blood cells, platelets, white blood cells but they cannot differentiate into hepatocytes or brain cells.

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Cells with stem cell like abilities have been observed breast cancer, colon cancer, leukemia, melanoma, prostate cancer which can form new cells and lead to tumorigenesis. They cause relapse and metastasis by giving rise to new tumors. Scientists are developing methods to destroy CSCs in place of traditional methods which focus on bulk of cancer cells.

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Head and Neck Cancer Research, Lung Cancer Diagnosis & Treatment, Genetics & Stem Cell Biology, Cancer Research, Nature Reviews Cancer, Journal of the National Cancer Institute, Clinical Cancer Research, Cancer Cell, Cancer, International Journal of Cancer, British Journal of Cancer

They are derived from Hematopoietic stem cells. They differentiate into Erythrocyte progenitor cell (forms erythrocytes), Thrombocyte progenitor cell (forms platelets) and Granulocyte-Monocyte progenitor cell (forms monocytes, macrophages, neutrophils, basophils, eosinophils, dendritic cells).

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They are the self-renewing, multipotent stem cells in the nervous system that differentiate into neurons, astrocytes and oligodendrocytes. They repair the nervous system after damage or an injury. They have potential clinical use the management of Parkinsons disease, Huntingtons disease and multiple sclerosis.

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They are derived from embryo in the blastocyst stage. They are pluripotent stem cells. They give rise to all derivatives of the three primary germ layers: endoderm (stomach, colon, liver, pancreas, intestines etc.), mesoderm (muscle, bone, cartilage, connective tissue, lymphatic system, circulatory system, genitourinary system etc.) and ectoderm (brain, spinal cord, epidermis etc.).

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Embryonic stem cells are derived from the fetus are used in treatment of various diseases. As ESCs are pluripotent, they can differentiate into any cell type. Researchers are able to grow ESCs into complex cells types like pancreatic -cells and cardiocytes. Fetal cell therapy is generating lot of controversy from religious groups and ethics committees.

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Research is being done to use stem cells for the treatment of diabetes mellitus. Human embryonic stem cells may be grown in vivo and stimulated to produce pancreatic -cells and later transplanted to the patient. Its success depends on response of the patients immune system and ability of the transplanted cells to proliferate, differentiate and integrate with the target tissue.

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The procedure to replace damaged cells (in cancers, aplastic anemia etc.) with healthy stem cells of the same person or in another compatible person to restore the normal production of cells. It can either be autologous or allogeneic. Bone marrow HSCs are generally used for the transplantation.

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They are the totipotent, undifferentiated cells present in the meristems (shoot and root apices) of a plant. They never undergo aging process and can grow into any cell in the plant throughout its lifetime. They have numerous applications in production of cosmetics, perfumes, pigments, insecticides and antimicrobials.

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Plant Pathology & Microbiology, Plant Biochemistry & Physiology, Plant Physiology & Pathology, Genetics & Stem Cell Biology, Plant Cell, Plant Physiology, Plant Journal, Trends in Plant Science, Current Opinion in Plant Biology, Plant, Cell and Environment, American Journal of Transplantation, Plant Molecular Biology

Several types of dental stem cells have been isolated from mature and immature teeth, exfoliated deciduous teeth and apical papilla, MSCS from tooth germs and from human periodontal ligament. They are found to be multipotent and can give rise to osteogenic, adipogenic, myogenic and neurogenic cell lineages.

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Oral Health and Dental Management, Research & Reviews: Journal of Dental Sciences, Dental Implants and Dentures: open access, Genetics & Stem Cell Biology, International Endodontic Journal, Dental Materials, Caries Research, Journal of Endodontics, Monographs in Oral Science, Molecular Oral Microbiology, Journal of Dentistry,International journal of oral science

Adipose tissue is a huge source of mesenchymal stem cells which differentiate into various cell types. They can be easily extracted in large numbers by a simple lipo-aspiration. They have good application potential in regenerative medicine. ASCs are found to have the ability to differentiate into bone cells, cartilage cells, nerve cells, adipocytes etc.

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Childhood Obesity, Obesity and Eating Disorders, Reproductive Medicine, Genetics & Stem Cell Biology, International Journal of Obesity, Obesity, Obesity Surgery, Obesity Reviews, Diabetes, Obesity and Metabolism, Diabetes, Obesity and Metabolism, Surgery for Obesity and Related Diseases, Pediatric obesity

Preservation of stem cells is critical for both research and clinical application of stem-cell based therapies. Properly preserved stem cells can be later used in the field of regenerative medicine for treating congenital disorders, heart defects etc. Currently there is no universal method for preserving stem cells and the existing methods are expensive.

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MSCs can be applied in osteoarthritis treatment through implantation and microfracture as well as intra-articular injections. Single injection studies have showed improvement from pain which decreased overtime. Multiple, regular MSC injections into joints may be necessary.

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Osteoporosis and Physical Activity, Osteoarthritis, Fertilization: In Vitro - IVF-Worldwide, Reproductive Medicine, Genetics & Stem Cell Biology, Osteoarthritis and Cartilage, Arthritis and Rheumatism, Arthritis Care and Research, Arthritis Research and Therapy, Seminars in Arthritis and Rheumatism

OMICS International through its Open Access Initiative is committed to make genuine and reliable contributions to the scientific community. OMICS International hosts over 700 leading-edge peer reviewed Open Access Journals and organizes over 1000 International Conferences annually all over the world. OMICS International journals have over 10 million readers and the fame and success of the same can be attributed to the strong editorial board which contains over 50000 eminent personalities that ensure a rapid, quality and quick review process. OMICS International signed an agreement with more than 1000 International Societies to make healthcare information Open Access. OMICS International Conferences make the perfect platform for global networking as it brings together renowned speakers and scientists across the globe to a most exciting and memorable scientific event filled with much enlightening interactive sessions, world class exhibitions and poster presentations.

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Journal of Stem Cell Research and Therapy- Open Access ...

Gene Therapy Initiative –

Exploring Nonsense Suppressionas a Treatment for NF1

This project aims to find compounds that suppress the effects of nonsense mutations in the NF1 gene, thus restoring neurofibromin protein expression and function in NF1 patients.

David Bedwell, PhDUniversity of Alabama, Birmingham

Bruce Korf, MD, PhDUniversity of Alabama, Brimingham

Mark Suto, PhDSouthern Research

This project will resolve two primary challenges applying gene therapy approaches to NF1 by using an innovative strategy to engineer new viruses that targets tumor initiating cells and CRISPR-based genome editing to restore the mutated NF1 gene. Using a unique team with complimentary expertise, this venture applies some of the most exciting modern biotechnologies to NF1.

Charles Gersbach, PhDDuke University

David V. Schaffer, PhDUniversity of California, Berkeley

David G. Kirsch, MD, PhDDuke University

Ataluren is a drug that can suppress protein synthesis termination at premature nonsense codons to produce essential proteins in patients with Duchenne muscular dystrophy. This project aims to evaluate its effect on mouse cells with an NF1 gene that harbors nonsense mutations.

Allan Jacobson, PhDUniversity of Massachusetts

This project proposes using nanoparticles to deliver 1) key coding regions of NF1 gene (cDNA) that will make neurofibromin protein, and 2) gene-editing regents to directly correct the mutation that causes NF1 in a patient derived NF1 rat model. If successful, the new system will provide essential pre-clinical data and lay the foundation for clinical trials using nanomedicine to treat NF1 disease.

Robert Kesterson, PhDUniversity of Alabama, Birmingham

Jiangbing Zhou, PhDYale University

This project will bioengineer trans-acting ribozymes, RNA molecules with catalytic properties similar to protein enzymes, to target faulty transcripts of the NF1 gene that fail to translate functional neurofibromin. NF1 mouse models with patient specific mutations that are amenable to ribozyme-mediated correction will be developed for subsequent animal studies.

Andr Leier, PhD University of Alabama, Birmingham

Ulrich Muller, PhDUniversity of California, San Diego

The mutation of one gene, e.g. NF1, often makes other genes that are not normally required for cell survival vulnerable to inactivation. This project aims to kill cells that have inactivated both copies of the NF1 gene. Using CRISPR/CAS9 technology, genes that become essential for the survival of cells with inactivated both copies of the NF1 gene will be identified, particularly those for which an FDA-approved drug is already available.

Eric Pasmant, PharmD, PhDUniversity Paris Descartes

Raphal Margueron, PhDInstitut Curie

This project seeks to develop two new NF1 drug candidates by developing and characterizing multiple potential therapeutics in parallel within fourteen research laboratories. AAV vectors for delivery and zinc finger protein and antisense oligonucleotides to upregulate NF1 expression will also be used when evaluating the efficacy of different therapeutic modalities.

Miguel Sena-Esteves, PhDUniversity of Massachusetts

Scot Wolfe, PhDUniversity of Massachusetts

Matthew Gounis, PhDUniversity of Massachusetts

Jonathan Watts, PhDUniversity of Massachusetts

Xandra Breakefield, PhDMassachusetts General Hospital

Casey Maguire, PhDMassachusetts General Hospital

Antisense directed gene therapy, or more specifically exon skipping, causes cells to skip over faulty pieces of the genetic code, leading to a truncated, but still functional, protein. This project aims to identify exons within the NF1 gene that may be skipped while still maintaining gene function and then develop antisense oligonucleotides to enable modulation of expression.

Deeann Wallis, PhDUniversity of Alabama, Birmingham

Linda Popplewell, PhDRoyal Holloway University of London

Gene Therapy Initiative -

AveXis Research & Development

The U.S. Food and Drug Administration (FDA) has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation, for the treatment of SMA Type 1.

The European Medicines Agency (EMA) also granted AveXis access into its PRIority Medicines (PRIME) program for AVXS-101 for the treatment of SMA Type 1.

The open-label, single-arm, single-dose, multi-center trial known as STR1VE is designed to evaluate the efficacy and safety of a one-time IV infusion of AVXS-101 in patients with SMA Type 1. The co-primary efficacy outcome measures of the trial include the achievement of independent sitting for at least 30 seconds at 18 months of age; and, event-free survival at 14 months of age. Co-secondary outcome measures include the ability to thrive, and the ability to remain independent of ventilatory support at 18 months of age.

The open-label, dose-comparison, multi-center Phase 1 trial known as STRONG is designed to evaluate the safety, optimal dosing, and proof of concept for efficacy of AVXS-101 in two distinct age groups of patients with SMA Type 2, utilizing a one-time IT route of administration. The primary outcome measure for patients less than 24 months of age at the time of dosing is the achievement of the ability to stand without support for at least three seconds. The primary outcome measure for patients between 24 months and 60 months of age at the time of dosing is the achievement of change in Hammersmith Functional Motor Scale Expanded from baseline. The secondary outcome measure for both age groups is the proportion of patients that achieve the ability to walk without assistance, defined as taking at least five steps independently while displaying coordination and balance. Developmental abilities, including motor function, will also be evaluated as exploratory objectives.

Learn more about clinical trials

We have exclusive worldwide license agreements to develop and commercialize gene therapy using the AAV9 vector to treat two rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

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AveXis Research & Development

Cell and Gene Therapy | Alliance for Cancer Gene Therapy …

What is Cell and Gene Therapy for Cancer?Gene therapy is a technique that uses genes to treat or prevent disease such as cancer by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: replacing a mutated or abnormal gene that causes disease with a healthy copy of the gene; inactivating, or knocking out, a mutated gene that is functioning improperly; and introducing a new gene into the body to help fight a disease.

Cell Therapy is the infusion or transplantation of whole cells into a patient for treatment of an inherited or acquired disease like cancer.

Primary Forms of Cell and Gene Therapies for Cancer Treatment

The long-term goal of cancer cell and gene therapy is to develop treatments that attack only cancer cells, eliminating adverse effects on the body. Furthermore, these therapies have potential for treating other diseases such as cardiovascular, disorders, cystic fibrosis, hemophilia, sickle-cell anemia, muscular dystrophy, diabetes, and Parkinsons. All research in this area, therefore, makes a difference.

About Molecular Medicine

Molecular medicine uses the bodys own cells and genes as both the source and medicine for diseases of all types the basis for all cell and gene therapies.

Molecular medicine began with the identification of DNA in the early 1900s. Progress was slow until the mapping of the human genome in the new millennium and the rapid technological advances that made it possible to isolate and target specific cells and genes.

This field of study explains the fundamental genetic errors that cause diseases like cancer and helps establish a blueprint for good health.

Molecular medicine and advanced technology make it possible to target cancers directly without damage to other parts of the body.

Molecular medicine is also referred to as genetic medicine, gene therapy, targeted therapeutics, genetic epidemiology or individualized medicine.

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Cell and Gene Therapy | Alliance for Cancer Gene Therapy ...

Gene Therapy Market Share Insights – Grand View Research

Industry Outlook

The global gene therapy market sizewas valued at USD 7.6 million in 2017. It is estimated to expand at a CAGR of over 19.0% during the forecast period. Increasing number of molecules in the development phase is expected to stoke market growth. It was projected that in 2016, more than 900 molecules are in the development phase that can prove to be an effective treatment for several incurable diseases, which are generally caused by an error in a single gene.

Increasing gene therapy innovations for cardiovascular and rare diseases treatment is one of the key trends driving the market. Rising focus on development of gene therapy treatment for rare diseases is a result of intensifying competition among market players to consolidate their position in the industry.

Gene therapy involves incorporation of an artificial or a modified gene using modified viral vectors that help deliver the gene at intended site of action or even kill the cell that may cause the disease. This treatment is mostly a one-time treatment or requires very few doses of medication to completely cure the disease.

The method of treatment, which was once considered impossible, has now become a trend among big and small companies. A consequence of this has been an upsurge in the number of successful startups, backed by investors in line with big companies. The trends is poised to continue and boost the growth of the market during the forecast period.

Some novel molecules to be used for gene therapy are set to reach the commercialization stage. The growth of the market is largely dependent on key decisions made by manufacturers such as pricing, regulations, and reimbursement for treatment along with payers who help cover treatment costs. However, concerns regarding unethical use of the therapy can hamper growth prospects, especially in developing countries.

The gene therapy market is witnessing an upswing in innovations in various therapeutic fields of medicine. However, oncology is at the forefront in terms of innovation. On the basis of indication, the cancer segment accounted for the leading share of the overall market revenue in 2017. This is due to the high number of pipeline molecules that were registered over the last three years. Rising prevalence of cancer caused due to genetic mutations is also contributing to the growth of the segment. The genetic disorders segment, however, is anticipated to register the highest CAGR during the forecast period.

There are very few drugs in the market that have been approved by various regulatory bodies across the globe. These drugs are considered to change the treatment methods and regimen for rare and orphan diseases, however, their sky high pricing is limiting their commercial success.

For instance, Glybera, the first drug approved for the treatment of LPLD, was a breakthrough in the medical history. However, the drug couldnt be a commercial success due to high pricing at 1.6 million USD at the time of launch and very low prevalence of the disease (1-2 per a million of population).

In 2016, GlaxoSmithKline got another drug Strimvelis approved by European drug regulatory authority for the treatment of ADA-SCID. Other techniques in R&D are likely to witness a significant growth rate in the forecast years due to proven success of approved drugs.

The key element of gene therapy lies in the delivery of modified gene or functioning gene. Delivery systems used should be able to deliver functioning gene to intended cell target through modified viruses, which are considered the best vectors by scientists as viruses are highly evolved in delivering nucleic acid, bypassing the immune system of the host.

Several viruses such as Adeno-associated virus, retrovirus, lentivirus, and herpes simplex virus are modified in labs and are used as carriers for gene therapy drugs. Adenovirus is the most used viral vector followed by Retrovirus due to their reduced immunogenicity. Each of the viruses has its own disadvantage such as toxicity, limited DNA carrying capacity, etc.

Non-Viral vectors are being developed lately that can reduce or eliminate viral toxicity completely, however, none of the non-viral vectors possess ideal vector properties as of now.

Europehas seen two efficient gene therapy molecules after 2010, one was released in 2012 by UniQure N.V and other by GlaxoSmithKline, which were groundbreaking and were approved by the European regulatory body.

The U.S. is estimated to become a leader in terms of revenue as the country more than 64.0% of clinical trials by various big and small companies in the overall clinial trials. Among emerging economies, Russia and China are expected to be at the forefront of the market by a significant margin as they have two approved drugs in the market that can be used for cancer treatment.

Some of the key players are UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics, Voyager Therapeutics, Human Stem Cell Institute, Bristol Myers Squibb, and Chiesi Farmaceutici S.p.A.

Due to a large number of pipeline molecules in development and intense competition among companies to augment their revenue growth, the market is projected to tread along a healthy growth track. Most of the startups are attracting capital investments to support their research for new molecules and initiate new product development.



Base year for estimation


Actual estimates/Historical data

2014 - 2016

Forecast period

2017 - 2025

Market representation

Revenue in USD Million and CAGR from 2017 to 2025

Regional scope

North America, Europe, Asia Pacific, Latin America, Middle East & Africa

Country scope

U.S., Canada, Germany, U.K., China, Japan, Brazil, South Africa

Report coverage

Revenue forecast, company share, competitive landscape, growth factors and trends

15% free customization scope (equivalent to 5 analyst working days)

If you need specific information, which is not currently within the scope of the report, we will provide it to you as a part of customization

This report forecasts revenue growth and provides an analysis of themarket trends in each of the sub-markets from 2014 to 2026. For the purpose of this report, Grand View Research has segmented the global gene therapy market report on the basis of indication, vector type, and region:

Indication Outlook (Revenue, USD Million, 2014 - 2026)


Cardio Vascular Diseases

Infectious Diseases

Genetic Disorders

Neuro Disorders


Vector Type Outlook (Revenue, USD Million, 2014 - 2026)

Viral Vectors



Adeno-associated virus

Vaccinia virus

Herpes simplex virus


Non-Viral Vectors

Injection of Naked DNA



Regional Outlook (Revenue, USD Million, 2014 - 2026)

North America


Asia Pacific

Latin America


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Gene Therapy Market Share Insights - Grand View Research

What is gene therapy? – Genetics Home Reference – NIH

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

Replacing a mutated gene that causes disease with a healthy copy of the gene.

Inactivating, or knocking out, a mutated gene that is functioning improperly.

Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.

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What is gene therapy? - Genetics Home Reference - NIH

Gene Therapy Manufacturing – The Bioprocessing Summit

Cambridge Healthtech Institute s 3rd AnnualAugust 16-17, 2018

It is an exciting time for gene therapy therapies on the market, encouraging clinical data and a long list of pharma collaborations. Pricing and reimbursement takes a majority of the headlines but equally important is producing these therapies in a scalable, cost-effective and robust way, all the while developing a clear CMC and characterization profile that satisfies the regulators.

Cambridge Healthtech Institutes Gene Therapy Manufacturing meeting takes a practical, case study driven approach to the process development, scale-up and production of gene therapies, tackling key topics such as AAV, lentivirus and retrovirus process development and scale-up, CMO management from early to late-stage development.

Final Agenda

Day 1 | Day 2 | Speaker Biographies

Thursday, August 16

11:30 am Registration Open (Grand Ballroom Foyer)

12:15 pm Enjoy Lunch on Your Own

1:15 10th Anniversary Cake Break in the Exhibit Hall with Last Chance for Poster Viewing (Grand Ballroom)

1:55 Chairpersons Remarks

John Pieracci, PhD, Director, Purification, Biogen

2:00 KEYNOTE PRESENTATION: Challenges and Strategies for the Development of a Robust, Scalable, Cost-Effective Biomanufacturing Process

Sadettin Ozturk, PhD, Senior Vice President, Process and Analytical Development, MassBiologics

The use of viral vectors has increased in recent years, both as gene therapies and as vectors for ex vivo cell therapy products. Industrialization of viral vector manufacturing is maturing as companies tackle problems in process control, scale-up, facility design, characterization and quality, and regulatory considerations. This presentation will examine the current state of the art, emerging technologies and challenges.

2:45 Enabling Industrial Scale Production of Lentiviral Vectors for Gene Therapy

Kelly Kral, PhD, Associate Director, Vector Process Development and Manufacturing, bluebird bio

Lentiviral vectors are an ideal platform for indications requiring long-term, stable expression, but the production processes have historically been limited by scale. As the field has now entered commercialization, there is demand for larger quantities of vector, driving the need for more scalable processes. This presentation will review the development, scale-up, and tech transfer of our suspension-based lentiviral vector process.

3:15 Strategies to Deliver Scalable and Reliable Lentiviral Vector Biomanufacturing

Jeffrey Bartlett, PhD, CSO, Calimmune, Inc.

Large-scale clinical production of lentiviral vectors (LV) using current good manufacturing practice (cGMP) methods comes with significant challenges. We have established the Cytegrity stable cell line system for LV bioproduction and have defined key process, quality and regulatory parameters needed to achieve desired productivity and quality across multiples scales and different bioproduction systems. This approach has allowed the production of LV required for Phase I and II clinical trials, while paving the way for future commercialization.

3:45Evolving Process-Centric Facility Design

Mike Sheehan, MSc, MBA, PMP, Senior Project Manager, DPS Group

Increasingly gene therapy products transitioning from clinical phase to commercial manufacture is driving demand for companies to provide additional capacity. Bringing products to market requires exploring opportunities for leading edge facility design, implementing new & evolving technologies, responding to scalability, speed to market and financial considerations.

4:00 Refreshment Break (Foyer)

4:15 Scalable Lentiviral Vector Production Using HEK293 Suspension Cells

Parminder S Chahal, Research Officer, Human Health Therapeutics Research Centre, National Research Council Canada

We have developed expertise in the production of lentiviral vectors (LV) using packaging cell lines and stable producers. Both grow in suspension and in serum-free conditions. Using a stable producer cell line that produces LV expressing GFP, we have compared different modes of operation in bench-scale bioreactors (batch, fed-batch and perfusion). Next, a battery of filters and supplements were evaluated for clarification. A maximal recovery of 78% was obtained.

4:45 Development and Characterization of Novel Micro-RNA Attenuated Oncolytic Herpes Simplex Viruses

Jonathan Platt, PhD., Senior Research Scientist, CMC Operations, Oncorus

Oncorus is developing next generation HSV-based oncolytic virus with enhanced potency for tumor cell killing and recruitment of the immune system. Our innovative miR-attenuation strategy enables robust viral replication in tumor cells, while preventing replication in healthy tissue. The development and characterization of therapeutic oHSV requires thorough product understanding gained through process characterization. Strategies for development and characterization of manufacturing processes centered around a strong organizational infrastructure will be presented.

5:15 End of Day

Day 1 | Day 2 | Speaker Biographies


8:00 am Registration Open and Morning Coffee (Grand Ballroom Foyer)

8:25 Chairpersons Remarks

Nathalie Clment, PhD, Associate Director and Associate Professor, Powell Gene Therapy Center, Pediatrics, University of Florida

8:30 FEATURED PRESENTATION: rAAV Vector Design, Capsid Directed Evolution and Scale Up Activities Using the BEVS System

Jacek Lubelski, PhD., VP, Global Pharmaceutical Development, uniQure

9:00 Towards a Pivotal Process for AAV Manufacture with HSV

David Knop, PhD, Executive Director, Process Development, AGTC

9:30 Large-Scale Manufacturing of Clinical Grade AAV in the Academic Setting

Nathalie Clment, PhD, Associate Director and Associate Professor, Powell Gene Therapy Center, Pediatrics, University of Florida

The talk will present our current methods for the production of research and clinical-grade rAAV with a special emphasis on the HSV-based suspension method capable of generating high titers of improved rAAV quality. Up-to-date in vitro, in vivo, and clinical data will be shown, and pros and cons of the method will be discussed in comparison to the two other most common methods, transfection and the baculovirus system.

10:00 Networking Coffee Break (Foyer)

10:30 Scale-Up Approach to AAV Manufacturing

Johannes C.M. van der Loo, PhD, Director, Clinical Vector Core, The Raymond G. Perelman Center for Molecular and Cellular Therapies, Childrens Hospital of Philadelphia

The Clinical Vector Core at the Childrens Hospital of Philadelphia manufactures preclinical- and clinical-grade AAV for academia and industry-sponsored clinical trials. With the field of gene therapy maturing, there is a growing need for larger scale products. We will discuss a strategy for scale-up that builds on our existing mammalian adherent cell-based manufacturing platform.

11:00 Virus-Like Particles and Other Extracellular Particles from Insect and Mammalian Cells

Alois Jungbauer, PhD, Professor, Institute of Biotechnology, University of Natural Resources and Life Sciences (BOKU)

Virus-like particles and other extra cellular particles are a next generation of biopharmaceuticals. They can be produced by a wide variety of host cells. The challenge is the production of high titers and downstream processing. The particle of interest are contaminated with other particles with similar biophysical properties and therefore difficult to separate. Examples will be given for 3 different cell types.

11:30 Considerations for the Purification Process Characterization of an AAV from Recovery to Drug Substance

Ratish Krishnan, PhD, Scientist, Bioprocessing Research & Development, Pfizer

Smart and efficient approaches for lab-scale characterization are required to ensure a robust adeno-associated manufacturing process. Specific challenges related to the uniqueness of characterizing an AAV manufacturing process will be discussed. Focus will be given to working with limited quantities of material and employing assays that are still being defined.

12:00 pm Next Generation AAV Viral Vector Manufacturing: Proven Technologies with a Modern Twist

Sandhya Buchanan, Director, Upstream Process Development, FUJIFILM Diosynth Biotechnologies

Current approaches to commercial-scale manufacture of viral vectors have been successful for many early phase trials and some late phase trials. Unique challenges/limitations arising for AAV manufacturing include quantities sufficient for patient needs and consumables for manufacturing. We discuss proven technologies blended with modern advancements to meet the needs of the advancing field of gene therapy.

12:30 Enjoy Lunch on Your Own

1:25 Chairpersons Remarks

Chia Chu, Senior Principal Scientist, Bioprocess Research & Development, Pfizer

1:30 FEATURED PRESENTATION: Separation of Full and Empty AAV Particles Using Scalable Isocratic Elution Chromatography

Meisam Bakhshayeshi, PhD, Head, Purification Development, Gene Therapy, Biogen

Robust and efficient removal of AAV empty particles is a critical part of the AAV manufacturing process. In this study, we present a scalable ion exchange chromatography process with isocratic wash and elution to separate full and empty particles. A combination of mono- and di-valent salts were used as eluents to achieve the high degree of resolution required for this separation. High product purity and recovery was achieved from this process.

2:00 Lyophilisation of AAV Gene Therapy Product

Tanvir Tabish, PhD, Head, Drug Product Development for Gene Therapy, Device and Combination Products, Shire

The gene therapy adeno-associated virus (AAV) subtype 8 containing Factor IX (FIX)(BAX335) was formulated in a new proprietary buffer and lyophilized. A stability study was established with the lyophilized material to determine its stability profile at the accelerated temperature of +5C over a 10 month period. The freeze-dried product displayed an improved stability profile when stored at a temperature of +5C. We demonstrated the feasibility of lyophilisation of the AAV viral drug product in the formulation buffer.

2:30 AAV Manufacturing at 2,000L Scale

Alex Fotopoulos, PhD., Senior Vice President, Technical Operations, Ultragenyx.

Changing the manufacturing site (tech transfer) should always include an assessment of comparability, however the ability to demonstrate this varies between early and late development. This talk will discuss common pitfalls and mistakes and highlight key aspects of the comparability exercise.

3:00 CMO Selection for Cell & Gene Therapy

Chad Green, PhD, Principal & Senior Consultant, Dark Horse

As the diversity of CMOs available for cell and gene therapies continues to grow worldwide, identifying the most suitable to engage is becoming an increasingly complex challenge. This presentation will address fundamental questions, such as whether a CMO is even the best choice for manufacturing before progressing to provide concrete guidance on the critical questions to ask prospective CMOs (and yourself), how to ask them and how to analyze the answers and make an optimal, rational choice.

3:30 Close of Conference

Day 1 | Day 2 | Speaker Biographies

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Gene Therapy Manufacturing - The Bioprocessing Summit

GeoGene: Gene Therapy, What it is, The process and Vectors …

What is Gene Therapy?

Certain diseases are caused byfaulty genes which produce defective proteins. The symptoms of genetic disease are the result of subsequent disrupted vital cell processes caused by missing or defective proteins. In theBio Building Blockssection of this web-site, protein synthesisis outlined as the process whereby,genesultimately give rise toproteinswhich are responsible for important cell processes. If a particular gene is defective, its protein product may not be made at all, may work poorly or may behave too aggressively.

For example:Cystic Fibrosis(CF) is caused by amissing or mutated genethat results in adefective cell membrane transport protein. This ultimately results in a build-up of thick mucus in the lungs and the body's airways.As another example,cancersare caused by cells that divide and grow uncontrollably.Particular genes can cause such cell growth to occur if they are defective. Such defective genes are calledoncogenes.

Are we treating the symptom or treating the cause? Historically, genetic disorders have been treated byaddressing the biological eventsthat result from the genetic mutation, as opposed tofixing a defective gene(or genes) the ultimate source of the problem.For example, the treatment of diabetes has historically involved the administration of insulin (a protein), instead of fixing the defective genes in pancreatic cells that actually prevent these cells from producing insulin in the proper amounts, on their own.

Gene therapy is an alternative approach whereby a genetic disorder is treated by inserting or integrating new genes into human cells. Many attempts at gene therapy aim to add a useful gene into a selected cell type to compensate for a missing or defective version. Other efforts aim to instill new properties in the target cell. This latter method is often employed in the treatment of cancer, where toxic genes are added to cancer cells in an effort to eliminate them.For an overview of how a specific gene is located and isolated from its source (so that it can be introduced into the patient) see ourGenetic Engineeringsection.

It should be noted that even the most advanced somatic cell therapy techniques are still in clinical trials, and are not yet approved for general application. Much more research is required to develop safe, reliable gene therapy techniques.

Depending on the cell types affected, gene therapy can be classified into two broad categories: germ-line gene therapy and somatic cell gene therapy.Germ-line therapyoccurs when germ cells (reproductive cells) are altered, meaning that the resultinggenetic changes will be passed on to the patient's offspring. Alternatively,somatic cell gene therapyinvolves the alteration of somatic cells (non-reproductive body cells, like skin, brain or muscle cells). This genetic manipulation willonly affect the individualto which the changes were made. Somatic cell gene therapy is the only type presently being considered in humans.

Suppose a patient is afflicted with a genetic disorder that affected only certain cells in her or his brain. How could she or he be treated using gene therapy so that the therapeutic gene targets only those cells affected by the disorder? One solution is through the use of avector. A vector is simply a "transporter" for the genetic material that allows it to enter the target cell and, depending on the vector type, can cause new genes to be integrated into the host cell genome. Vectors must be administered totarget specific cell types.

There are three principal ways in which vectors can be administered to carry new genes into target cells. The first is calledex vivosomatic gene therapy, wherethe target cells are removed from the body, cultured in the laboratory with a vector, and re-inserted into the body. This process is usually carried out using blood cells because they are the easiest to remove and return.

The second option,in situsomatic gene therapy, occurs when thevector is placed directly into the affected tissue. This process is being developed for the treatment of cystic fibrosis (by direct infusion of the vector into the bronchi of the lungs), to destroy tumours (eg: brain cancer), and for the treatment of muscular dystrophy.

The third option isin vivosomatic gene therapy, where thevector is injected into the bloodstream, and is able to find and insert new genes only into the cells for which it was specifically designed. Although there are presently noin vivotreatments available, a breakthrough in this area will make gene therapy a very attractive option for treatment.In this case the vector designed to treat our hypothetical patient could be injected into a blood vessel in her or his arm and would find its way to the affected brain cells!

Vectors used in gene therapy can be classified as eitherviralornon-viral.

BothDNAandRNAviruses are being developed as vectors for use in gene therapy. Viruses are an excellent choice for use as vectors, because they have gained, through long periods of evolution, the ability to avoid destruction by the human immune system, and the capacity to get their own genetic material inside human cells. As discussed in theBio Building Blockssection, viruses consist of genetic material (DNA or RNA) surrounded by a protective coat made of proteins and occasionally other molecule types as well.

Normally, a virus infects a cell when its genetic material enters it. Once the viral genetic material is inside, it "hijacks" the cell's DNA- and protein-making machinery, causing it to produce new viruses. Some viruses are even capable of integrating their own genetic material into the host cell's genome.

It is the outer protective viral coat that allows the inner genetic material to penetrate the cell. This outer coat also determines the type of cell that a given virus will infect. Once inside, it is the harmful viral genes that actually hijack the cell and eventually cause it to die.

To trick the virus, scientists retain the outer viral coat, but modify the inner genetic material. They remove the harmful genes and replace them with therapeutic ones. Now the virus ispathogenically disabled(it is no longer harmful to the cell it infects) and incapable of reproducing itself. However, it retains its capability to transfer its genetic material to the cells for which its outer coat was designed.The transfer of genetic material by way of a viral vector is calledtransduction.

The structure and mode of infection of retroviruses is discussed in theBio Building Blockssection. Briefly, retroviruses have RNA as their genetic material. These viruses also carry a specialenzymethat, once inside a cell, makes double-stranded DNA from the virus' RNA template. The new DNA becomes incorporated into the host cell's genome. When the "new" chromosomal genes are transcribed, new virus particles are made, which will leave the cell to infect other cells.

Most types of retroviruses are not very harmful to the cell. Even though allviruses to be used as vectors are deactivated,' meaning that their harmful genes are removed, the fact that the types of retroviruses presently being used as vectors are not very harmful in their natural forms means that their use poses less risk than the use of some other viruses. Even if something goes wrong and some of the original retrovirus particles are administered to the patient, they will not cause serious problems.

Themurine leukaemia virus(MuLV) is one of the more popular retroviruses used as a retroviral vector. The reproductive genes in the retrovirus are replaced with the therapeutic gene. When the virus infects the cell,the therapeutic gene gets incorporated into the cell chromosomes. The new gene causes a protein to be produced which is hoped to have some positive therapeutic effects, either providing an otherwise missing protein, or causing the destruction of harmful cells.

There are several challenges that scientists must overcome for effectivein vivotreatment of disease using retroviral vectors. For example, theviruses must be capable of targeting only those cells affected by the disorder. If this were the case, they could be injected directly into the bloodstream (in vivogene therapy) where they would become dispersed throughout the body, but would only transduce those cells for which they were designed. Presently, retroviral vectors are not terribly specific, meaning that many cells not intended for the transfer of the gene are transduced by the virus, which reduces the transfer to the targeted cell population.

To understand how viruses can be made to be more specific, we should considerhow viruses "choose" the cells they infect. A virus must bind to specific surface receptor molecules to gain entry into a cell. To this end, retroviruses have outer envelope proteins that fit perfectly into certain receptors on specific cells. The MuLV virus binds to cells containing a receptor called theamphotropic receptor. The problem is that a broad range of cell types possess the amphotropic receptor. This means that the MuLV virus, in its natural form, can infect all of these cell types, most of which are likely not the target of the therapy!

To make retroviral vectors more specific about the cells they invade, scientists are experimenting with ways ofreplacing or modifying the outer viral proteins, so that they fit into more rare receptors that appear only on specific cell types being targeted for therapy.Another approach has been toadd new proteinsto the outer viral envelope which either better recognize the target cell, or better recognize the region of the body where the target cells are located.

Another challenge is toengineer retroviral vectors to transducenon-dividingcells. Most retroviruses target actively dividing cells, which makes them ideal for the treatment of rapidly dividing tumour cells, but not in situations where a therapeutic gene is to be introduced into a non-dividing cell, like in the treatment of cystic fibrosis mentioned above. Those few retroviruses that have the ability to infect non-dividing cells are the harmful ones (HIV, the virus that results in AIDS, is one of them). HIV viruses (with their harmful genes removed) cannot be used as vectors, because even with the removal of these genes, there is still a possibility that the virus might become harmful again through a process called recombination. To virtually eliminate the possibility that harmful viruses are produced in this way, while still harnessing the capability of HIV to transduce non-dividing cells, scientists are experimenting with the development of hybrid vectors, made up mostly of other retroviruses and which contain very small and harmless parts of the HIV virus.

As of April, 1998, there was only one vector-based therapeutic technique in the final clinical trial stage(called Phase III). This technique employs a retroviral vector called G1TkSvNa for the treatment ofglioblastoma multiforma, a malignant brain tumour. The treatment is an in situ therapeutic technique, where mouse cells capable of producing and secreting the vector are injected into the tumour.The secreted vectors infect only those cells that are rapidly dividing, meaning only the tumour cells and the vessels supplying blood to the tumour are transduced. The gene transduced into the tumour cells gives rise to a protein (calledHerpes Simplex Thymidine Kinaseor HSTk).Fourteen days later, a drug called ganciclovir is injected into the patient, which is toxic to any cell that incorporates it into its DNA. Only the cells containing HSTk (the tumour cells) are capable of incorporating ganciclovir into their DNA and these cells are therefore selectively killed off.

Adenoviruses are DNA viruses that are able to transduce a large number of cell types, including non-dividing cells. Adenoviruses also have the capacity to carry long segments of added genetic information. In addition, it is fairly easy to produce large amounts of adenoviruses in culture. Adenoviruses, in their natural form, are not very harmful, typically causing nothing more serious than a chest cold in otherwise healthy people. This means that their use as vectors is quite safe. For all these reasons, adenoviruses are currently the most widely used DNA vectors for experiments inin situgene therapy.Research is currently under way using adenoviral vectors for the treatment of several cancers and cystic fibrosis.

The size of the adenovirus protein coat is just large enough to fit the original viral DNA inside. As a result, for every new therapeutic gene to be inserted into the viral genome, a corresponding piece of the old viral DNA must be removed.To make room for the new therapeutic DNA, a region of the old viral DNA called E3 is sometimes removed. However, removing the E3 region has drawbacks, because it codes for a protein that suppresses the human immune response against the vector. Without the E3 region, the virus is more susceptible to the immune system and is more likely to be destroyed before it has served its purpose.

Adenoviral vectors send their DNA to the nucleus, butthe DNA does not get incorporated into the host cell's chromosomes. For this reason, the viral DNA has a finite lifetime within the cell before it is degraded, meaning that the added genes are effective only temporarily. Treatments for chronic conditions like cystic fibrosis, therefore, would need to be repeated periodically, perhaps on a monthly or yearly basis. On the other hand, the transient nature of therapeutic gene expression is useful when the added genes are needed temporarily to induce an immune response to a cancer or pathogen.

Among the other virus types being explored as vectors are theadeno-associated virus(AAV) and theherpes simplex virus(HSV). Both are DNA-based viruses. AAV integrates its genetic material into a host chromosome and cause no diseases in humans. However, because AAV are small, they cannot accommodate large genes. HSV vectors do not integrate their genes into the host genome. They tend to target neurons and thus have the potential for use in the treatment of neurological disorders.

The use of non-viral vectors can involve a direct injection ofplasmid DNAor mixing plasmid DNA with compounds that allow it to cross the cell membrane and protect the DNA from degradation. These methods are currently less efficient than the use of viral vectors. However, unlike disabled viruses which have the possibility of changing spontaneously and causing disease, non-viral vectors possess no viral genes and therefore cannot cause disease.

Liposomes are small, hollow spheres of fatty molecules that are capable of carrying DNA inside of them.A liposome can fuse with the cell membrane, releasing its contents into the cell interior.

Plasmid DNA containing the therapeutic gene is incubated with the empty liposomes under specific conditions. The negatively charged DNA binds to the positively charged (calledcationic) liposomes and the plasmids are absorbed. Liposomes containing plasmid DNA are calledlipoplexes.The lipoplexes can subsequently enter the cells of interest, and thus introduce the therapeutic DNA into the cells.

Experiments have been carried out where lipoplexes have been injected into tumours. The lipolexes contained a gene that gives rise to a protein that is recognized by the human immune system. Theoretically, thesegenes should cause the tumour cells to express the recognizable protein on their surface, which will mark the cells for destructionby the immune system.

The use of lipoplexes for the treatment of cystic fibrosis is currently being studied as well. The cause of the illness is a defective gene which causes a particular protein in the patient's lung cells to be defective. The lipoplexes that are administered using an aerosol spray into the patient's lungs, contain the gene for a functional version of the protein.

Lipoplexes are not as efficient as viral vectors in introducing genes into cells. To improve their efficiency, scientists are attempting to incorporate some viral proteins into the outer surfaces of lipoplexes. In particular, the viral proteins that recognize and bind to specific molecules on the host cell's surface, are being incorporated.

Muscle cells have been shown to be capable of taking up and expressing plasmid DNA. This raises the possibility that plasmid DNA injected into muscles could stimulate the production by muscle cells of a therapeutic protein. This protein could then be secreted into the bloodstream and to the rest of the body. For example, the gene coding for erythropoietin (a protein which helps stimulate the production of red blood cells) has been experimentally injected into animal muscles with some success. Such a treatment would be useful to patients after chemotherapy or radiation therapy.

In addition,plasmid DNA shows promise for use in vaccines, stimulating protective immune responses against diseases like herpes, AIDS or malaria. When the plasmid DNA is injected into muscles, it enters muscle cells and as a result, causes the cells to produce the proteins that correspond to the genes the plasmids contain. The immune system will then learn to recognize the new proteins and will destroy them if they are encountered in the future. Experiments are currently under way where plasmids containing genes for viral coat proteins are injected, in attempt to make the immune system recognize these viruses, so that it will attack and destroy them if they are ever encountered.

As discussed in theBio Building Blockssection, viruses hijack cellular machinery to produce their own proteins and to replicate their genetic material, which results in the production of new viruses.One of the potential uses of antisense technology is to prevent viruses that infect a host cell from producing their own proteins. This would, in turn, prevent their replication.

Recall that proteins are constructed through atwo step process. In the first step,DNA is transcribed to produce messenger RNA(mRNA). The second step involves thetranslation of the mRNA to make a protein. Antisense drugs interact with mRNA, preventing them from being translated into their corresponding protein.

An mRNA molecule is a chain of nucleotides, that gets "read" by a ribosome in the synthesis of a protein. An antisense drug is anoligonucleotide(a relatively small, single stranded chain of nucleotides) that iscomplementaryto a small segment of a target mRNA molecule. When the drug comes into contact with its complementary mRNA, it binds to the mRNA in the same way as the two strands of a DNA molecule bind together.This makes the mRNA "unreadable" by the ribosome, and so no protein is produced.

Because an antisense drug is designed to be complementary to a particular mRNA sequence that is specific to a particular virus' mRNA, it will not interfere with any of the host cell's naturally produced mRNA, meaning that the side effects of the drug are minimal.

At the end of August, 1998, the US Food and Drug Administration (FDA) approved a drug calledformivirsenfor the treatment of cytomegalovirus (CMV) retinitis in patients with AIDS.This makes formivirsen the first antisense drug on the market.Formivirsen blocks the replication ofcytomegalovirus(CMV) which causesretinitis, an eye infection leading to blindness that mainly affects AIDS patients. The drug is periodically injected into the patient's eye, and is claimed to cause only mild side-effects as compared to some other antiviral drugs.

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GeoGene: Gene Therapy, What it is, The process and Vectors ...

The Gene Therapy Plan: Taking Control of Your Genetic …

Praise for The Gene Therapy PlanA guide to harnessing the power hidden in food to subvert a genetic predisposition for disease. . . . Gaynors informative tome is worth reading. Publishers Weekly

The Gene Therapy Plan identifies how the lives we lead, and in particular, the foods and nutritional supplements we ingest, are a key determining factor in whether latent disease (which most people have to some degree) materialize or stay dormant. By identifying researched nutritional protocols that target specific conditions, and by providing a range of rich case studies from his practice as a leading oncologist and internist, Dr. Gaynor provides insight and an action plan into how the body operates that will benefit medical practitioners and patients alike. Deepak Chopra, M.D.The Human Genome Project promised to create a new era of genetic medicine, new drugs, and therapies to advance human health. But the real awakening has been the understanding of foodreal whole foods, herbs, phytonutrientsas medicine and how it can literally upgrade your biologic software by improving the expression of your genes.In The Gene Therapy Plan Dr. Gaynor makes the healthcare of the future available to you today. If you want to learn how to use food and nutrients to prevent and even reverse most chronic disease, read this book! Mark Hyman, M.D., Director of the Cleveland Clinic Center for Functional Medicine and author of the #1 New York Times bestseller The Blood Sugar SolutionThe Gene Therapy Plan is a comprehensive and practical approach to the science of epigeneticsand how to apply it to your life right now. This book is a godsend that could save your life. Christiane Northrup, M.D., author of the New York Times bestseller Womens Bodies, Womens WisdomA brilliant and important piece of work from one of our most distinguished and creative medical thinkers. Do yourself and your family a huge favor: Read this phenomenally important book and learn why and how you can live a healthier life. Devra Davis, Ph.D., M.P.H., founder and president of the Environmental Health Trust, author of The Secret History of the War on CancerDr. Gaynor is a visionary healer. This is a comprehensive, coherent, practical, and easily digestible resource for all who wish to tip the balance away from disease toward health and wellness. Sheldon Marc Feldman, M.D., Vivian L. Milstein Associate Professor of Clinical Surgery, Columbia University College of Physicians and SurgeonsDr. Gaynor presents a comprehensive strategy for readers to re-orient their diet and lifestyle using everyday activities that can help one live longer, and live better. With The Gene Therapy Plan, Dr. Gaynor brings his own integrative philosophy and practice to readers in an engaging and actionable way. William Li, M.D., president and medical director of The Angiogenesis FoundationDr. Gaynor has and always will be at the forefront of integrative medicine. The Gene Therapy Plan empowers you to take control of your health and life. Mimi Guarneri, M.D., president of the Academy of Integrative Health and Medicine

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The Gene Therapy Plan: Taking Control of Your Genetic ...

Antiviral Gene Therapy Research Unit – Wits University

Welcome to the Antiviral Gene Therapy Research Unit (AGTRU) of the University of the Witwatersrand and South African Medical Research Council (SAMRC)

Investigation by the AGTRU team is focused on countering viral infections that cause serious health problems in South Africa. The long term objectives of AGTRU are to

Discovery of the RNA interference (RNAi) pathway and advances in the engineering of sequence-specific nucleases have provided the means for powerful and specific disabling of genes. These advances led to considerable enthusiasm for use of gene therapy to counter viral infections, such as are caused by persistence of hepatitis B virus (HBV) and human immunodeficiency virus type 1 (HIV-1). The focus of the AGTRU has been on optimising use of RNAi activators and transcription activator-like effector nucleases (TALENs) to inhibit viral proliferation. Development of suitable vectors for delivery of antiviral sequences to infected cells is also an active field of investigation within the unit.

Research activities are generously supported by South African and International funding agencies. South African and international partnerships have been established, and these are an important contributor to the groups resource base.

The unit currently has approximately 20 members and these include molecular biologists, clinicians and postgraduate students. There are four tenured university appointees in the unit and the director is Professor Patrick Arbuthnot. AGTRU is equipped as a modern molecular biology research laboratory and has expertise in a range of techniques. These are advanced methods of nucleic acid manipulation, gene transfer to mammalian cells, use of lipoplex and recombinant viral vectors. AGTRU is set up to investigate efficacy of antiviral compounds in vivo in murine (e.g. HBV transgenic mice) and cell culture models of viral replication.

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Antiviral Gene Therapy Research Unit - Wits University

Gene Therapy Research | Ophthotech

Gene Therapy Research Programs

Ophthotech initiated an innovative gene therapy program focused on applying novel gene therapy technology to discover and develop new therapies for ocular diseases. We intend to investigate promising gene therapy product candidates and other technologies through collaborations with leading companies and academic institutions in the United States and internationally.

As we evaluate the unmet medical need for the treatment of orphan ophthalmic diseases, we have considered that many of these diseases are caused by one or more genetic mutations and currently have no approved treatment options available. Further, the potential to achieve an extended treatment effect and possibly a cure through a single gene therapy administration is particularly appealing to patients who do not have any treatment options, as well as for patients with age-related retinal diseases who currently require chronic therapy over years, if not decades.

Gene therapy consists of delivering DNA encoding for a functional protein to a target tissue to facilitate protein synthesis using a recipients existing cellular machinery. Gene therapy can be used to replace a non-functional protein produced innately by the subject as a result of a genetic mutation or simply as a means of producing and delivering a therapeutic protein that would not otherwise be produced within the body. The DNA, which is generally delivered by a viral vector, is governed by a promoter sequence which controls transcription of the gene of interest, or transgene, into RNA to initiate protein synthesis. Some of the challenges that gene therapy faces are producing vectors that transfect, or deposit the transgene, in only specific cell types, producing the desired protein at the therapeutic dose levels, and avoiding inducing an inflammatory response that leads to tissue damage. We are particularly interested in adeno-associated virus, or AAV, gene therapy delivery vehicles, as AAV vectors are relatively specific to retinal cells and their safety profile in humans is relatively well-documented as compared to other delivery vehicles and gene therapy technologies currently in development.

University of Massachusetts Medical School and its Horae Gene Therapy Center

For our first gene therapy collaboration, we entered into a series of sponsored research agreements with the University of Massachusetts Medical School (UMMS) and its Horae Gene Therapy Center to utilize their minigene therapy approach and other novel gene delivery technologies to target retinal diseases. As a condition of each research agreement, UMMS has granted the Company an option to obtain an exclusive license to any patent or patent applications that result from this research.

The use of minigenes as a novel therapeutic strategy seeks to deliver a shortened but still functional form of a large gene packaged into a standard-size AAV delivery vector commonly used in gene therapy. The minigene strategy may offer an innovative solution for diseases that would otherwise be difficult to address through conventional AAV gene replacement therapy where the size of the gene of interest exceeds the transgene packaging capacity of conventional AAV vectors. Research in this newly evolving area of gene therapy is led by Prof. Hemant Khanna and colleagues in the Horae Gene Therapy Center and was described in a recent journal article in Human Gene Therapy, Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis by Wei Zhang, Linjing Li, Qin Su, Guangping Gao, and Hemant Khanna, all at the University of Massachusetts Medical School.

The collaboration with UMass Medical School will also focus on developing the next generation of gene therapy vectors to allow novel delivery approaches for treatment of retinal diseases.

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Gene Therapy Research | Ophthotech