PUBLIC RELEASE DATE:

9-Sep-2014

Contact: Karen Kreeger karen.kreeger@uphs.upenn.edu 215-349-5658 University of Pennsylvania School of Medicine http://www.twitter.com/PennMedNews

PHILADELPHIA - Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this month's issue of Nature Biotechnology. They suggest annuity payments over a defined period of time and contingent on evidence that the treatment remains effective. The approach would replace the current practice of single, usually large, at-point-of-service payments.

"Unlike most rare disease treatments that can continue for decades, gene therapy is frequently administered only once, providing many years, even a lifetime, of benefit," says James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. "Under current reimbursement policies, private insurers and the government typically pay for this therapy once: when it is administered. But these individual payments could reach several million dollars each under current market conditions. We're proposing a different approach that spreads payments out and only keep coming if the patient continues to do well."

Wilson and co-author Troyen A. Brennan, MD, JD, MPH, chief medical officer of CVS Health, note that while large single payments for gene therapy may be the simplest approach, they carry substantial encumbrances. For example, approval of gene therapy treatments is unavoidably based on data derived from trials carried out over several years at most -- considerably shorter than the expected duration of the therapy. Payers may therefore be unwilling to pay large up-front sums for treatments whose long-term benefit has not been established. Additionally, large payments for medications, such as the $84,000-a-patient cost of the hepatitis C treatment Sovaldi, have been criticized in the prevailing climate of curbing health care costs. This, despite the fact that effective gene therapy may reduce the overall financial burden to the health care system.

Wilson and Brennan further note that while a liver transplant, for example, can cost up to $300,000, physicians and hospitals that "transplant livers know they will be compensated at market rates through existing contracts -- gene developers lack that assurance." Annuity payments, they say, could help address these problems.

An example of an annuity-type disbursement could be a hypothetical payment of $150,000 per year for a certain number of years for gene-therapy-based protein replacement for patients with hemophilia B -- so long as the therapy continues to work. According to the authors, the cumulative amount should be less than the cost of a one-time payment of $4-6 million, which would be the expected rate for a gene-based therapy to be comparatively priced to existing, conventional therapies for hemophilia B. "One would presume," they write, "that gene therapy will have to represent a discount in order for insurers to approve its use."

"The annuity model that we're proposing would eliminate the misguided incentive to invest in drugs and treatments with ongoing revenue streams but which require continuing, perhaps lifetime daily administration, with all the attendant inconveniences and burdens to patients and their families, as well as direct and indirect costs to the nation's health system," says Wilson.

The authors point out that gene therapy differs substantially from the case of "orphan" drugs. Development of the latter, which target rare diseases affecting small patient populations, is supported by the Orphan Drug Act of 1983, which provides pharmaceutical manufacturers with grants, tax credits, and an extended period of market exclusivity for their medications. What's more, in virtually all of these cases, the business costs of developing the drugs are further attenuated by ongoing administration of -- and payment for -- the medication over the lifetime of the patient. "The contrast with gene therapy, especially that which produces a durable cure with one administration," the authors write, "is clear."

Excerpt from:

Penn researcher and CVS Health physician urge new payment model for gene therapy

PUBLIC RELEASE DATE:

9-Sep-2014

Contact: Evy Vierstraete info@vib.be 32-924-46611 VIB (the Flanders Institute for Biotechnology)

For the first time the Jeffrey Modell Foundation is giving a research grant to a Belgian laboratory. The team of Adrian Liston from VIB-KU Leuven will use the grant to develop a gene therapy to cure children that suffer from IPEX syndrome, a rare and fatal autoimmune disorder in which the immune system attacks the body's own tissues and organs. At the moment, the only successful therapy to treat the syndrome is a bone marrow transplantation, which is not available for all children.

"This is a real chance for a cure", said group leader Adrian Liston. "The gene responsible for this disease was identified 13 years ago, but for the first time we may have learned enough about the basic biology to solve it. We should know within a year whether the gene therapy works in mice, after which we can move to patients at top speed."

The Jeffrey Modell Foundation (JMF)

JMF is a global non-profit organization for patients who suffer from Primary Immunodeficiency (PI) and their relatives. The organization is devoted to early and precise diagnosis, meaningful treatments and, ultimately, cures. Through clinical and basic research, physician education, patient support, advocacy, public awareness and new-born screening they want to make a difference in the lives of patients with PI.

Vicki and Fred Modell established the Foundation in 1987, in memory of their son Jeffrey, who died at the age of fifteen from complications of PI. During the years, the foundation has created a network of the world's leading expert immunologists. Two years ago the Child Immune Deficiencies Department of UZ Leuven was given the first certification as a "Jeffrey Modell Foundation Diagnostic and Research Center for Primary Immunodeficiencies" in Belgium.

IPEX and primary immunodeficiency (PI)

IPEX is an acronym for immune dysregulation, polyendocrinopathy (diseases affecting multiple endocrine glands), enteropathy (disorder of the intestines), and X-linked (pattern of inheritance).

Original post:

Jeffrey Modell Foundation supports Belgian research on primary immunodeficiency

Contact Information

Available for logged-in reporters only

Newswise ANN ARBOR, Mich. University of Michigan Health System doctors have started testing a unique new approach to fighting brain tumors -- one that delivers a one-two punch designed to knock out the most dangerous brain cancer.

The experimental approach, based on U-M research, delivers two different genes directly into the brains of patients following the operation to remove the bulk of their tumors.

The idea: trigger immune activity within the brain itself to kill remaining tumor cells -- the ones neurosurgeons cant take out, which make this type of tumor so dangerous.

Its the first time this gene therapy approach is being tried in humans, after more than a decade of research in experimental models.

One of the genes is designed to kill tumor cells directly, and is turned on when the patient takes a certain drug. The other gene spurs the bodys own immune system to attack remaining cancer cells. Both are delivered into brain cells via a harmless virus.

The Phase I clinical trial has already enrolled two patients who have tolerated the gene delivery without complications. All patients in the study must have a presumptive diagnosis of WHO grade 3 or 4 malignant primary glioma, such as glioblastoma multiforme; patients must not have been treated yet by any therapy. They must also meet other criteria for inclusion in the trial.

More patients will be able to enroll at a pace of about one every three weeks, through a careful selection process. In addition to surgery and gene therapy at U-M, each will receive standard chemotherapy and radiation therapy as well as follow-up assessments for up to two years.

Were very pleased to see our years of research lead to a clinical trial, because based on our prior work we believe this combination of cell-killing and immune-stimulating approaches holds important promise, says principal investigator Pedro Lowenstein, M.D., Ph.D., the U-M Medical School Department of Neurosurgery professor who has co-led the basic research effort to develop and test the strategy.

Continue reading here:

A One-Two Punch for Brain Tumors? New Clinical Trial Opens at U-M

First published in News Last updated by Robert Merrick, Parliamentary Correspondent

PROTESTS by a group of MPs have failed to derail plans for a controversial gene therapy, to stop incurable diseases passing to the next generations.

Ministers vowed to plough ahead with preparations for the DNA-altering procedure, which is being pioneered by a team at Newcastle University.

However, the department of health declined to say when the issue would be put to a vote in Parliament, despite suggestions that it could be before the end of the year.

The treatment involves replacing faulty mitochondria responsible for inherited diseases, including muscle wasting, heart problems, vision loss, organ failure and epilepsy.

Embryos are given healthy DNA from donor eggs, meaning a baby has the DNA of three people from two parents, plus less than one per cent from the donor.

Professor Doug Turnbull, who leads the Newcastle team, has urged the Government to draw up legislation as soon as possible, because of the number of patients waiting for treatment.

But, in the Commons, MPs brought forward a motion demanding further research and for new regulations to be delayed in light of public safety concerns.

Fiona Bruce, a Conservative backbencher, claimed the Human Fertilisation and Embryology Authority (HFEA) wanted further research, saying: This is a case of genetic engineering.

It is the alteration of a potential human being - the removal of certain genes and their replacement with others, to create children.

See the rest here:

MPs' protests fail to derail gene therapy plans

ABC Erin Griffin was an advocate for childhood cancer awareness.

A 14-year-old girl who campaigned to raise awareness about childhood cancer has died after making history through her participation in a gene therapy trial.

Erin Griffin died at the Children's Hospital in Adelaide with her parents, brother and grandmother by her side.

In 2013, Erin received a Children's Week Award for her advocacy work in raising childhood cancer awareness.

Erin was born in Scotland and moved to Australia when she was six.

She was diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG), an incurable brain cancer, on February 18, 2012.

"We're all very sad to lose this special girl," Erin's specialist oncologist Dr Geoff McCowage told the ABC.

Dr McCowage, who runs a gene therapy trial at the Children's Hospital at Westmead in Sydney, said Erin made history.

"She was only the second child in the world to take part [in the trial]," he said.

"Throughout her ordeal she stayed courageous, she took two trips to the United States, one to Scotland and she was active on the internet telling the story of what she was going through and offering support to others going through the same thing.

Here is the original post:

14-year-old childhood cancer campaigner Erin Griffin dies in Adelaide

Gene Therapy EDTECH

By: Krittamet Tatsanapornsatith

Go here to read the rest:

Gene Therapy EDTECH - Video

The current method to treat acute toxin poisoning is to inject antibodies, commonly produced in animals, to neutralize the toxin. But this method has challenges ranging from safety to difficulties in developing, producing and maintaining the anti-serums in large quantities.

New research led by Charles Shoemaker, Ph.D., professor in the Department of Infectious Disease and Global Health at the Cummings School of Veterinary Medicine at Tufts University, shows that gene therapy may offer significant advantages in prevention and treatment of botulism exposure over current methods. The findings of the National Institutes of Health funded study appear in the August 29 issue of PLOS ONE.

Shoemaker has been studying gene therapy as a novel way to treat diseases such as botulism, a rare but serious paralytic illness caused by a nerve toxin that is produced by the bacterium Clostridium botulinum. Despite the relatively small number of botulism poisoning cases nationally, there are global concerns that the toxin can be produced easily and inexpensively for bioterrorism use. Botulism, like E. coli food poisoning and C. difficile infection, is a toxin-mediated disease, meaning it occurs from a toxin that is produced by a microbial infection.

Shoemaker's previously reported antitoxin treatments use proteins produced from the genetic material extracted from alpacas that were immunized against a toxin. Alpacas, which are members of the camelid family, produce an unusual type of antibody that is particularly useful in developing effective, inexpensive antitoxin agents. A small piece of the camelid antibody -- called a VHH -- can bind to and neutralize the botulism toxin. The research team has found that linking two or more different toxin-neutralizing VHHs results in VHH-based neutralizing agents (VNAs) that have extraordinary antitoxin potency and can be produced as a single molecule in bacteria at low cost. Additionally, VNAs have a longer shelf life than traditional antibodies so they can be better stored until needed.

The newly published PLOS ONE study assessed the long-term efficacy of the therapy and demonstrated that a single gene therapy treatment led to prolonged production of VNA in blood and protected the mice from subsequent exposures to C. botulinum toxin for up to several months. Virtually all mice pretreated with VNA gene therapy survived when exposed to a normally lethal dose of botulinum toxin administered up to nine weeks later. Approximately 40 percent survived when exposed to this toxin as late as 13 or 17 weeks post-treatment. With gene therapy the VNA genetic material is delivered to animals by a vector that induces the animals to produce their own antitoxin VNA proteins over a prolonged period of time, thus preventing illness from toxin exposures.

The second part of the study showed that mice were rapidly protected from C. botulinum toxin exposure by the same VNA gene therapy, surviving even when treated 90 minutes after the toxin exposure.

"We envision this treatment approach having a broad range of applications such as protecting military personnel from biothreat agents or protecting the public from other toxin-mediated diseases such as C. difficile and Shiga toxin-producing E. coli infections," said Shoemaker, the paper's senior author. "More research is being conducted with VNA gene therapy and it's hard to deny the potential of this rapid-acting and long-lasting therapy in treating these and several other important illnesses."

Story Source:

The above story is based on materials provided by Tufts University. Note: Materials may be edited for content and length.

Continue reading here:

Efficacy of new gene therapy approach for toxin exposures shown in mouse study

PUBLIC RELEASE DATE:

29-Aug-2014

Contact: Rushmie A Nofsinger rushmie.nofsinger@tufts.edu 508-839-7910 Tufts University, Health Sciences Campus

NORTH GRAFTON, Mass. (August 29, 2014, 2 PM US Eastern Time)The current method to treat acute toxin poisoning is to inject antibodies, commonly produced in animals, to neutralize the toxin. But this method has challenges ranging from safety to difficulties in developing, producing and maintaining the anti-serums in large quantities.

New research led by Charles Shoemaker, Ph.D., professor in the Department of Infectious Disease and Global Health at the Cummings School of Veterinary Medicine at Tufts University, shows that gene therapy may offer significant advantages in prevention and treatment of botulism exposure over current methods. The findings of the National Institutes of Health funded study appear in the August 29 issue of PLOS ONE.

Shoemaker has been studying gene therapy as a novel way to treat diseases such as botulism, a rare but serious paralytic illness caused by a nerve toxin that is produced by the bacterium Clostridium botulinum. Despite the relatively small number of botulism poisoning cases nationally, there are global concerns that the toxin can be produced easily and inexpensively for bioterrorism use. Botulism, like E. coli food poisoning and C. difficile infection, is a toxin-mediated disease, meaning it occurs from a toxin that is produced by a microbial infection.

Shoemaker's previously reported antitoxin treatments use proteins produced from the genetic material extracted from alpacas that were immunized against a toxin. Alpacas, which are members of the camelid family, produce an unusual type of antibody that is particularly useful in developing effective, inexpensive antitoxin agents. A small piece of the camelid antibody called a VHH can bind to and neutralize the botulism toxin. The research team has found that linking two or more different toxin-neutralizing VHHs results in VHH-based neutralizing agents (VNAs) that have extraordinary antitoxin potency and can be produced as a single molecule in bacteria at low cost. Additionally, VNAs have a longer shelf life than traditional antibodies so they can be better stored until needed.

The newly published PLOS ONE study assessed the long-term efficacy of the therapy and demonstrated that a single gene therapy treatment led to prolonged production of VNA in blood and protected the mice from subsequent exposures to C. botulinum toxin for up to several months. Virtually all mice pretreated with VNA gene therapy survived when exposed to a normally lethal dose of botulinum toxin administered up to nine weeks later. Approximately 40 percent survived when exposed to this toxin as late as 13 or 17 weeks post-treatment. With gene therapy the VNA genetic material is delivered to animals by a vector that induces the animals to produce their own antitoxin VNA proteins over a prolonged period of time, thus preventing illness from toxin exposures.

The second part of the study showed that mice were rapidly protected from C. botulinum toxin exposure by the same VNA gene therapy, surviving even when treated 90 minutes after the toxin exposure.

"We envision this treatment approach having a broad range of applications such as protecting military personnel from biothreat agents or protecting the public from other toxin-mediated diseases such as C. difficile and Shiga toxin-producing E. coli infections," said Shoemaker, the paper's senior author. "More research is being conducted with VNA gene therapy and it's hard to deny the potential of this rapid-acting and long-lasting therapy in treating these and several other important illnesses."

Read more from the original source:

Mice study shows efficacy of new gene therapy approach for toxin exposures

"How to Predict and Improve Your Future Health with Gene Therapy" by Devin Stone at MetroMD
"How to Predict and Improve Your Future Health with Gene Therapy" by Devin Stone at MetroMD.

By: weight-loss

Continue reading here:

"How to Predict and Improve Your Future Health with Gene Therapy" by Devin Stone at MetroMD - Video

Combine some microbiologists with some mechanical engineers and you never know what might happen.

A recent collaboration of those two fields of scientists at Brigham Young University produced a machine so small it takes a microscope to see it.

It's not just for fun, however. It could pave the way for research into diseases including Alzheimer's, cancer and diabetes.

For decades scientists have conducted gene research by transferring genetic material into a new cell by a process called microinjection. It was just considered an unfortunate by-product of the process to have 40 percent of the cells die.

Enter BYU and a different process called nanoinjection, which occurs by transferring material including DNA into cells.

"Because DNA is naturally negatively charged, it is attracted to the outside of the lance using positive voltage," said Brian Jensen, a BYU professor of mechanical engineering, in a release. "Once we insert the lance into a cell, we simply reverse the polarity of the electrical force and the lance releases the DNA."

The lance being used is 10 times smaller than what was used previously. In the past, researchers used a hollow needle to pump a DNA-filled liquid into an egg cell nucleus, but the extra fluid caused many of the cells to swell and die.

With the new method, the team found that nearly 80 percent of the cells proceeded to the next stage of development, a significant increase from the previous 60 percent success rate.

No extra fluid is used and the cells undergo less stress and have a higher survival rate.

"It is really great engineering stuff," said Sandra Burnett, associate professor of microbiology at BYU.

The rest is here:

Smaller is better: BYU creates new gene therapy technology

Gene Therapy Cure Cancer
This is my final project for science subject at my school. I learning about biotechnology. My topic is about " How gene therapy can cure cancer?" and i have to fin the solution. I am very...

By: della lathifah

More here:

Gene Therapy Cure Cancer - Video

Albany, New York (PRWEB) May 22, 2014

Gene therapy involves use of DNA as a pharmaceutical agent to treat diseases. It is one of the most important developments in the field of medicine that has potential to treat various lethal diseases such as HIV, cancer and cystic fibrosis. In the long run, biotechnology and clinical trial industries will benefit from developments in gene therapy and provide potential treatment solutions for various incurable diseases.

Browse the full report - http://www.transparencymarketresearch.com/gene-therapy-market.html.

In the present scenario, various pharmaceutical companies are using clinical data to validate the concept of gene therapy. Moreover, many venture capital investors are also showing their interest in gene therapy, and are investing heavily in its development. However, gene therapy is highly dependent on the regulatory approvals and most of the products are currently in clinical trial phase. Most of these gene therapy products are for cancer and cardiovascular diseases, and are in Phase III/ Phase II of clinical trials.

In addition, growing popularity of DNA vaccines boost advances in gene therapy and is likely to be practiced in clinics in the near future, with a number of therapy programs now in phase II/III trials, showing promising results.

Get report sample - http://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=1838.

Some of the major players operating in the market are AnGes MG, BioSante Pharmaceuticals, GenVec, Genzyme Corporation, Oxford BioMedica, Transgene, Urigen Pharmaceuticals and Vical.

This research report analyzes this market depending on its market segments, major geographies, and current market trends. Geographies analyzed under this research report include:

This report provides comprehensive analysis of:

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porters five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

More:

Global Gene Therapy Market: Analysis, Size, Share, Growth, Trends and Forecast 2013 - 2019

In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients blood cells into soldiers that seek and destroy cancer.

A few patients with one type of leukemia were given this one-time, experimental therapy several years ago, and some remain cancer-free today. Now, at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, with stunning results.

Its really exciting, said Dr. Janis Abkowitz, blood diseases chief at the University of Washington in Seattle and president of the American Society of Hematology. You can take a cell that belongs to a patient and engineer it to be an attack cell.

In one study, all five adults and 19 of 22 children with acute lymphocytic leukemia, commonly known as ALL, had a complete remission, meaning no cancer could be found after treatment, although a few have relapsed since then.

These were gravely ill patients out of options. Some had tried multiple bone marrow transplants and up to 10 types of chemotherapy or other treatments.

Cancer was so advanced in Emily Whitehead, now 8, of Philipsburg, Pa., that doctors said her major organs would fail within days. She was the first child given the gene therapy; now almost two years later, she shows no sign of cancer.

The regimen also can be used to treat myeloma, lymphoma and chronic lymphocytic leukemia, commonly known as CLL.

This has the potential to become the first gene therapy approved in the United States and the first for cancer worldwide, doctors said. Only one gene therapy is approved in Europe, for a rare metabolic disease.

The treatment involves filtering patients blood to remove millions of white blood cells called T-cells, altering them in the lab to contain a gene that targets cancer, and returning them to the patient in infusions over three days.

What we are giving essentially is a living drug permanently altered cells that multiply in the body into an army to fight the cancer, said Dr. David Porter, a University of Pennsylvania scientist who led one study.

View original post here:

Gene therapy hits blood cancers hard

PUBLIC RELEASE DATE:

21-May-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, May 21, 2014A targeted gene silencing strategy blocks production of the dysfunctional huntingtin (Htt) protein, the cause of Huntington's disease, a fatal, inherited neurodegenerative disorder. The effectiveness of this RNA interference (RNAi) approach in reducing levels of mutant Htt protein and disease symptoms in a mouse model of the disease is described in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website.

Lisa Stanek and coauthors from Genzyme (Framingham, MA) used an adeno-associated viral (AAV) vector to deliver a targeted nucleic acid sequence called a small interfering RNA (siRNA) into the cells of affected mice. The siRNA selectively binds to the mutated gene, blocking disease-causing Htt production. The authors present data demonstrating the ability to deliver the therapeutic RNAi into the cells, reduce mutant Htt levels, and impact behavioral deficits in the mice without causing any noticeable neurotoxicity, in their article "Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease."

"The Genzyme group uses state-of-the-art delivery technology and a gene silencing approach to generate very promising preclinical data for Huntington's disease," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

###

About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

See the original post:

Novel RNAi therapy silences mutated Huntington's disease gene and reduces symptoms

Nanoinjector Device: New Gene Therapy Advance from BYU Microbiologists and Engineers
BYU researchers create tiny nano-device in newest gene therapy advance: Nanoinjector is used to transfer genes and DNA to new cells The ability to transfer a gene or DNA sequence from one animal...

By: BYU

The rest is here:

Nanoinjector Device: New Gene Therapy Advance from BYU Microbiologists and Engineers - Video

Gene Therapy Biology Project for Swavely
A project for my biology class.

By: TeddyBearPower7

View post:

Gene Therapy Biology Project for Swavely - Video

Gene Therapy (Using Biolistic gene gun for gene delivery)

By: Kang Weiling

See original here:

Gene Therapy (Using Biolistic gene gun for gene delivery) - Video

Gene therapy for RTT syndrome benefits and drawbacks

By: osama ashmawy

Read the original here:

Gene therapy for RTT syndrome benefits and drawbacks - Video

Germline Gene Therapy

By: Farhana Zahari

Here is the original post:

Germline Gene Therapy - Video

Spark Therapeutics, a gene therapy medical company, this week signed an agreement for a headquarters in West Philadelphia. Spark Therapeutics, spun out of Children's Hospital of Philadelphia in October with $50 million in capital, will build out a 28,000-square-foot facility at 3737 Market St. to house its business operations, clinical research and development, and manufacturing.

Jeffrey D. Marrazzo, cofounder and chief executive, said the new facility "will support the continued expansion of our team and expand our manufacturing capacity to support our clinical development and commercial plans."

Spark anticipates moving into its new headquarters and expanding to 50 full-time employees by the end of 2014. Spark is preparing to complete clinical development of its lead, Phase 3 clinical program to address inherited retinal dystrophies caused by mutations in the RPE65 gene.

- Erin Arvedlund

Read the original here:

Spark Therapeutics to open headquarters in West Philadelphia