StemCell Therapy

Hurricane Harvey Flooding Claims 6 Members Of Same Family

Members of the Saldivar family ranging from 6 to 84 perished inside a van swept by currents as they sought higher ground. (Patch)

U.S. OK's First Gene Therapy Treatment For Childhood Leukemia

The Food and Drug Administration on Wednesday signed off on the first gene therapy treatment in the U.S. that doctors say uses a patient's own cells to seek out and destroy deadly childhood leukemia. (Patch)

Legendary Basketball Coach Rollie Massimino Dead At 82

Legendary college basketball coach Rollie Massimino, who led the Villanova Wildcats to a storied NCAA Championship in 1985 against Georgetown University, has died, according to the chancellor of Keiser University, who expressed "heartfelt sadness" on behalf of the school. Massimino served as head coach of the Keiser men's basketball team for the past 12 years. He was 82. (Patch)

World's Worst Babysitters Leave 9-Month-Old At Long Island Casino

Three people, including two teens, were arrested after they left a 9-month-old baby at a Long Island casino Tuesday, police said. The trio was supposed to be babysitting the child, but a dispute with the baby's mother quickly sent things haywire. (Patch)

Indiana Couple Completes Decades-long Goal To Visit Every Cracker Barrel

An elderly couple from Goshen finished a decades-long mission over the weekend: to visit every Cracker Barrel location, WGN-TV reports. Ray and Wilma Yoder flew out to Tualatin, Oregon, so they could visit and eat at their 645th Cracker Barrel on Monday morning. (Patch)

Quick Hits

Trump Claims To Have Witnessed Harvey's 'Horror And Devastation' Firsthand - But Didn't (Patch)

Here's How To Throw A Killer Labor Day Party, According To Pinterest (Patch)

'Having A Baby Means Losing A Paycheck' For Pregnant Police Officer Forced On Unpaid Leave: ACLU (Patch)

Microsoft, Amazon Collaborate So Personal Assistants Can Chat (Patch)

Deer In The Headlights Detained On San Francisco-Oakland Bay Bridge (Patch)

Girl, 7, Halfway Through Hug-A-Cop Tour Across USA

Rosalyn Baldwin is on a mission. Her goal? To hug a law enforcement officer in every state of the USA. Spanning 21 states so far, the seven-year-olds journey has been shared via her website, which is maintained by her two sisters, OceanRose and Scholar24, as well as her Facebook page. (Patch)

Harvey Slams Southeast Texas: Helicopter Rescues; Heartbreaking Scene As Toddler Clings To Mother

People are being plucked from homes by helicopter, boats are going door to door and the governor is sending the National Guard to the area. And in Beaumont, one of the more heartbreaking scenes of the storm unfolded when rescuers saved a toddler who was clinging to the body of her mother, who had died. (Patch)

All Houston ISD Students To Receive 3 Free Meals Per Day This School Year

All Houston ISD students will eat for free during the 2017-18 school year thanks to a waiver of the required application process for the National School Lunch/Breakfast Program from the United States Department of Agriculture and the Texas Department of Agriculture. (Patch)

Hurricane Harvey Scam Alert: Beware Of ICE, HSI Impersonators

Houston Police Chief Art Acevedo said there are reports that people impersonating Homeland Security Investigations special agents. Acevedo said they are knocking on doors in the Houston area telling residents to evacuate in the aftermath of Hurricane Harvey presumably so these impostors can rob the empty homes, he said. (Patch)

Gas Prices Rising Thanks To Harvey, Says AAA

While Hurricane Harvey was bearing down on Texas and the Gulf Coast, drivers looking to fill up their tanks were greeted at the gas station with one of the largest one-week surges in gas prices this summer, according to AAA. Prices increased 4 cents to a nationwide average of $2.37 per gallon. (Patch)

Parade For Purple Heart Recipient Planned In Illinois

A United States Air Force veteran who was seriously wounded in Afghanistan a dozen years ago and given a small chance to live will be given a parade in the town where he attended high school. (Patch)

Hurricane Harvey: More Than 300 Gators East Of Houston Could Soon Escape Enclosure

As if things weren't bad enough in the Houston area amid Tropical Storm Harvey, up to 350 alligators at a sanctuary east of town could soon escape if water levels continue to rise, according to reports. (Patch)

Photo of Overjoyed Mom Becomes A Social Media Sensation

Her daughters, Hayley and Millaina, knew the photo of their mom jumping for joy with a glass of wine in her hand was being taken as a joke, but mom told Patch, "Those facial expressions are for real!!!" (Patch)

This Day In History

1422 King Henry V of England dies of dysentery while in France. His son, Henry VI becomes King of England at the age of 9 months.

1864 During the American Civil War, Union forces led by General William T. Sherman launch an assault on Atlanta.

1897 Thomas Edison patents the Kinetoscope, the first movie projector.

1962 Trinidad and Tobago becomes independent.

1993 Russia completes removing its troops from Lithuania.

Famous Birthdays

AD 12 Caligula, Roman emperor

1928 James Coburn, American actor

1944 Roger Dean, English illustrator and publisher

1946 Tom Coughlin, American football player and coach

1970 Debbie Gibson, American singer-songwriter, producer, and actress

Photo: Former Villanova head coach Rollie Massimino looks on prior to the 2016 NCAA Men's Final Four National Championship game between the Villanova Wildcats and the North Carolina Tar Heels at NRG Stadium on April 4, 2016, in Houston. Credit: Streeter Lecka/ Staff/ Getty Images Sport/ Getty Images

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Originally published August 31, 2017.

Read more from the original source:
Harvey Haunts Family Leukemia Gene Therapy Massimino Dies: Patch Morning Briefing - Patch.com

FINDINGS

Scientists from UCLA and the Howard Hughes Medical Institute successfully used a gene that suppresses cholesterol levels as part of a treatment to reduce plaque in mice with a disorder called familial hypercholesterolemia. In a preclinical study, researchers found that the gene, LeXis, lowered cholesterol and blockages in the arteries, and the treatment appeared to reduce the build-up of fat in liver cells.

Familial hypercholesterolemia is an inherited condition characterized by extremely high levels of low-density lipoprotein cholesterol (commonly referred to as bad cholesterol) and an increased risk of early heart disease.

The LeXis gene belongs to a unique group of genes that until recently were considered junk DNA because scientists believed they served little purpose. However, evidence from the human genome project led to the discovery that genes like LeXis are actually active. The study of these genes,now referred to as long noncoding ribonucleic acids, or lncRNAs, is a rapidly evolving area in biology.

Researchers wanted to test whether a single injection of LeXis could slow the development of heart disease. To do so, they gave the mice either LeXis or a control gene, and fed them a 15-week diet consisting of food high in sodium and cholesterol the mouse equivalent of fast-food hamburgers and french fries. Researchers then measured the progression of heart disease.

In the next phase of the study, researchers intend to confirm the findings in larger animals and test the therapy in combination with currently available treatments.

Although previous research has shown that lncRNAs can be important, this is the first study to show that they could potentially be used to treat a human disease using gene therapy. Junk genes could one day offer a framework for treating people with familial hypercholesterolemia and other conditions that are otherwise very difficult to treat.

The papers authors are Xiaohui Wu, Zhengyi Zhang and Dr. Tamer Sallam of UCLA; and Dr. Peter Tontonoz, Marius Jones and David Salisbury of the Howard Hughes Medical Institute.

The study waspublished onlinein the journal Circulation.

The research was supported by grants from the National Heart, Lung, and Blood Institute; the American College of Cardiology; and the Lauren B. Leichtman and Arthur E. Levine UCLA Cardiovascular Discovery Fund.

Learn more about the cardiovascular research theme at UCLA.

More here:
Gene therapy using 'junk DNA' could lower risk for heart disease - UCLA Newsroom

Three patients who received SPK-8011 a gene therapy developed by Spark Therapeutics for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company.

The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with early data showing they have not yet experienced spontaneous bleeding episodes.

The encouraging start of our SPK-8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof-of-concept in a second liver-mediated disease a significant achievement in the gene therapy field and positions us well to potentially transform the current treatment approach for this life-altering disease with a one-time intervention, Katherine A. High, president and CEO of Spark Therapeutics, said inreleasingsecond-quarter 2017 financial results.

The study (NCT03003533) examines escalating doses of the treatment a one time infusion of a non-infectious virus that delivers a functional copy of the gene encoding clotting factor VIII.

The first two patients received the lowest dose and have now been followed for 23 weeks and 12 weeks. Measurements show their levels of the clotting factor steadily rising, reaching a stable level of 11 and 14 percent of normal values. Withthose results in hand, researchers decided to go for a higher dose in the third patient. So far, the increase of factor VIII in that third patient is higher than that observed in the other two, corresponding to the increased dose.

Researchers have not detected immune reactions to the therapy in any of the patients. This is important, as antibodies to SPK-8011 ould potentially render it ineffective.No serious adverse events have been reported and none of the patients required treatment with corticosteroids.

Spark, based in Philadelphia, has also reported on its second gene therapy, SPK-9001, now being developed to treat hemophilia B.

Researchers presented data from the Phase 1/2 study (NCT02484092) at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, showing that the 10 participants had lowered their annual bleeding rate by 96 percent. They also lowered their annualized infusion rate by 99 percent.

In addition, their levels of clotting factor IX, 12 weeks after the treatment, had stabilized at 33 percent. Four of the patients have been followed for more than a year after the treatment, and one for as long as 18 months.

None have developed immune reactions to the treatment and no bleeding episodes have been recorded. One patient with severe joint disease had precautionary factor infusions for persistent knee pain.

We are excited about the progress we are making to achieve our goals of our investigational hemophilia A and B programs: to safely achieve predictable, consistent and sustained activity levels that prevent spontaneous bleeding, said High.

Go here to read the rest:
Spark's Gene Therapy SPK-8011 to Treat Hemophilia A and B Advance in Clinical Trials - Hemophilia News Today

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GAINESVILLE, Florida Imagine only being able to see the things in front of you in soft focus, and just in black and white.

For people with the genetic eye condition achromatopsia, those are just some of the side effects.

Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun.

"I need to have very dark, very tinted sunglasses to feel comfortable outside, to see really well," said Tara.

Tara has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light.She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance.

"I cannot drive a car so I rely on public transportation and my bike to get around," she said.

"There are currently no approved or no effective treatments for achromatopsia," said Dr. Christine Kay, a surgical ophthalmologist at the University of Florida.

But she's working to change that.

She is one of a handful of experts testing a gene therapy.

"For achromatopsia the cells we have to target are cone cells responsible for decreased vision and color vision. and those are cells at the very bottom layer of the retina," explained Dr. Kay.

Using a tiny cannula, doctors deliver a normal copy of one of two mutated genes, the CNGA-3 or CNGB-3 gene, directly into the eye to restore vision.

Tara`s myopia is so severe that her risk of retinal detachment from any retinal surgery is high, which rules her out for the current trial.

In the meantime, Tara says she's learned to adapt to achromatopsia and live without limitations.

"And I hope, ya know, all young achromats, ya know, learn the same thing."

Dr Kay says if the gene could eventually be delivered to the surface of the retina, additional patients, like Tara, could be treated. AGTC, the biotech company that developed the therapies, and several U.S. universities have successfully tested this therapy in dogs and sheep.

NEW TECHNOLOGY: A new clinical trial is underway testing gene therapy for achromatopsia. The genes are responsible for releasing proteins essential for the function of all the cells. Researchers remove the virus from the host DNA so it does not have the capacity to make the patient sick, and then insert the gene of interest into the DNA. Surgically done by a vitro retinal surgeon, the gene is then directly delivered to the retinal tissue. Researchers try to avoid directly touching the retina, to avoid detaching it and keep the surgery less evasive. The CNGA3 or CNGB3 gene would help restore the patient`s vision. This can only be done on certain patients; those with extremely severe achromatopsia are not able to participate in this trial because of the risk of retinal rupturing.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.com or Marjorie Bekaert Thomas at mthomas@ivanhoe.com.

See the original post here:
YOUR HEALTH Gene therapy for better eyesight - WQAD.com

A darling British biotech that is working on the development of the worlds leading gene therapy for cancer has posted reduced losses and a leap in sales.

Oxford BioMedica signed a deal worth up to $100m (77m) with Swiss drugs giant Novartis last month to supply cell material for its potential blockbuster treatment, known as CTL019, for a type of leukaemia.

The living drug was recommended for approval by US regulators in June, with a final determination expected this autumn.

It would be the first gene therapy for cancer and if approved is expected to generate a spike in revenues for Oxford BioMedica, with the potential for further tie-ups on other cancer treatments.

Oxford BioMedica is also trialling its lentiviral vectors, which help manipulate genes, in potential treatments for Parkinsons Disease and an eye condition.

In half-year results today, Oxford BioMedica pared back losses to 2.2m, compared to 6.9m in the red the previous year. Revenues jumped 26pc to 15.7m.

See original here:
Gene therapy darling Oxford BioMedica pares losses - Telegraph.co.uk

Investment Thesis

With an undervalued stock, a critically-acclaimed drug leading in its space and a barrage of financial and expertise-based backing, Sangamo Therapeutics (NASDAQ:SGMO) is the ideal vehicle for exposure to a market with high growth potential and a hedge against the antiquated methods (CRISPR/Cas9) of treating hemophilia. The same method has also been found to work to a high degree of accuracy on other genetic disorders.

Sangamo Therapeutics' accomplishments to date have helped to draw integrity to its operations and raise its profile against common competitors in the space (QURE, GSK, ONCE, BMRN, BLUE, BIVV (a pure-play hemophilia company). Principally, Sangamo has secured an exclusivity agreement (and subsequent vote of confidence) from Pfizer (NYSE:PFE) (also once a competitor) regarding the development and commercialization of gene therapy programs for hemophilia A (SB-525), which affects 85% of hemophilia sufferers (Nelson Pediatrics), bagging $70 million upfront and a potential $475 million in milestones and royalties (Reuters). (The Zinc Finger Protein Nuclease technology method adopted is targeted at diseases which are caused by genetic defects, as their removal is known to respond best to gene editing targeting, in particular, tauopathies, thalassemias, hemophilia, and HIV/AIDS).

The company's strong balance sheet was further bolstered by a raise of circa $78.1 million through public offering at a price of $7.25 per share, with a revised analyst share price target estimates from Jefferies LLC of $17 per share (jefferies.com), as a "financial investment hedge against emerging gene-editing technology of CRISPR," the CRISPR/Cas9 system, has seen less successful testing, coupled with scientific criticism for its reported high incidence of nonspecific DNA cleavage, mosaicism (failure to replicate mutant alleles in only some of the cells) and inversely, and overproduction of mutations in a given organism.

SGMO's latest 10-Q (SEC.gov):

In the context of gene therapy, Sangamo ($707.16 million market cap) (capitaliq.com) was the only firm with innovative gene therapy drugs to advance in two clinical trials (namely the SB-728-T program in the Sangamo ZFP Therapeutic Pipeline targeting HIV/AIDS), and the first to conduct genome editing studies in clinical trials, as covered in the Financial Times. The stock's performance has been volatile, and investors were disinclined to back the drug lottery in gene therapy as biotech became heavily saturated in players, regulations and setbacks, stunting the prospect of any tangible advancement.

Sangamo has jumped on the "radar" to become a buy. The FDA has authorized Fast Track designation, the scientific backing is legitimate (and has received industry-wide appraisal), the longer-term targets look plausible and Sangamo's management and consultants are capable of executing given their credentials and two decades of development.

By all measures of valuation, Sangamo is undervalued, and price-action enthusiasts will recognize the surge in trading volumes and the breaking of the 50-day moving average. Early riders would have capitalized on the short-term price depression triggered by the publicizing of the mucopolysaccharidosis Type I (MPS I) and MPS II delay, an overreaction from the market that posed a great value opportunity at the beginning of this month, whose benefits were felt just last week when the stock surged on the impressive FIH results.

Gene therapy (and emicizumab) renders recombinant factor VIII/IX proteins redundant. This a slice of the industry that is represented by $8-10 billion annually (with 90%+ profit margins) shared by Shire (NASDAQ:SHPG), Bayer (OTCPK:BAYRY), Novo Nordisk (NYSE:NVO), Pfizer and Bioverativ (NASDAQ:BIVV), an overvalued stock whose current operations consist entirely of the moribund method of treating hemophilia - a potential sell. It recently acquired True North and thus entering into cold agglutinin disease market, a result of severe hemolytic anemia.

Sangamo now has the backing and leeway to make its drugs commercially viable, magnetizing further interest - 48 institutions have increased their respective positions in SGMO, with Wasatch and BlackRock leading the pack. The smart money and medical experts are backing Sangamo.

Bold tickers indicate potential buys should this thesis play out as predicted.

This is not investment advice, you are advised to carry out your own due diligence.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Read the original here:
Sangamo Therapeutics: The Breakthrough In Gene Therapy - Seeking Alpha

MOSCOW, August 17. /TASS/. A Russian gene therapy drug for individuals infected with HIV called Dinavir is undergoing pre-clinical trials, and the drug has already proved its efficiency on cells. The pre-clinical tests on animal models, clinical trials and the registration procedure may take up to 10 years, senior research fellow at the Epidemiology Central Research Institute of Rospotrebnadzor (the Federal Service on Surveillance for Customers Rights Protection and Human Well-Being) Dina Glazkova told TASS.

"This is not about the next year, but rather in five years, at the earliest. It takes up to 10 years on the average," she said.

Glazkova reiterated that the registration is made after the clinical trials. "Again, the clinical trials are costly, and the drug production is costly as well," the scientist added.

Dinavir proved to be safe while tested on cells, in vitro. A Phase II pre-clinical trial will utilize animal models to test the efficiency and safety of treatment. A Phase I clinical trial will be carried out on humans to test safety of the therapy and will take up not less than a year.

"Phase II takes up two to three years, and it is unclear how much will be required from us. Phase I is about safety, and it takes a few patients: five, maybe ten. Phase II is when we have to prove that the drug works in these five to ten [patients] and that it had a positive effect on them. Phase III is when we enroll a lot of patients [in the trial] to show that the five were cured not by accident and that it [the gene therapy] really works," Glazkova explained.

The gene therapy for HIV treatment is being developed by a group of researches at the Epidemiology Central Research Institute of Rospotrebnadzor.

Original post:
Scientists foresee Russian gene therapy for HIV cure may be registered in 5-10 years - TASS

DUBLIN, August 16, 2017 /PRNewswire/ --

The "Global Cancer Gene Therapy Market 2017-2021" report has been added to Research and Markets' offering.

The global cancer gene therapy market to grow at a CAGR of 20.63% during the period 2017-2021.

The report, Global Cancer Gene Therapy Market 2017-2021, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.

One trend in the market is favorable government regulations for gene therapy programs. Gene therapy has been a big hope for the individuals suffering from rare diseases and various forms of cancers. Cancer is one of the biggest life-threatening diseases across the globe and has been a huge point of concern for the healthcare providers. This has led to rising support to R&D activities in cancer gene therapy from various governments.

According to the report, one driver in the market is rising geriatric population. The global geriatric population is continues to grow at a faster pace due to several factors such as rapidly falling fertility rates and growing life expectancy due to better medical facilities. The US Census Bureau reported that the total population aging 65 years and above was estimated at 617 million in 2015 and is expected to rise to 1,566 million by 2050. Asia has the largest and fastest growing aging population due to several factors such as the huge population of the region, government policies such as one child policy of China has reduced the addition of young population, and higher investment in the healthcare sector has led to better medical facilities and longer life expectancy.

Further, the report states that one challenge in the market is high cost of gene therapy treatment. The sky-high cost of gene therapy has made it a point of debate across the globe. One of the major causes for the exorbitant cost of gene therapy is the requirement of intensive clinical trials. The gene therapy needs to be tailored to suit the genetic acceptance for each individual, unlike small and large molecule drug clinical trials where the trials are done in large and random samples. In the clinical trials of gene therapy, the stem cell is extracted from each patient's bone marrow, then the correct version of the gene is mingled with the cell, and the corrected stem cells are introduced to the individual patient.

Key vendors

Other prominent vendors

Key Topics Covered:

Part 01: Executive Summary

Part 02: Scope Of The Report

Part 03: Research Methodology

Part 04: Market Landscape

Part 05: Pipeline Analysis

Part 06: Market Segmentation By Therapy

Part 07: Geographical Segmentation

Part 08: Decision Framework

Part 09: Drivers And Challenges

Part 10: Market Trends

Part 11: Vendor Landscape

Part 12: Key Vendor Analysis

Part 13: Appendix

For more information about this report visit https://www.researchandmarkets.com/research/drvk7v/global_cancer

Media Contact:

Research and Markets Laura Wood, Senior Manager rel="nofollow">press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

Link:
Global Cancer Gene Therapy Market to Grow at a CAGR of 20.6% by 2021: Favorable Government Regulations for ... - Markets Insider

LONDON--(BUSINESS WIRE)--According to the latest market study released by Technavio, the global cancer gene therapy market is expected to grow at a CAGR of almost 21% during the forecast period.

This research report titled Global Cancer Gene Therapy Market 2017-2021 provides an in-depth analysis of the market in terms of revenue and emerging market trends. This market research report also includes up to date analysis and forecasts for various market segments and all geographical regions.

The rising prevalence rate of cancer has been a huge challenge for the global economies as the disease leads to high rate of mortality and economic losses. The current treatment options available come with many drawbacks such as severe side effects and relapse of cancer. These factors have led to high investment in the R&D for development of various novel therapies with cancer gene therapy being one of the major ones of them. The therapy mainly uses three types of treatment options namely oncolytic virotherapy, gene transfer therapy, and gene-induced immunotherapy.

This report is available at a USD 1,000 discount for a limited time only: View market snapshot before purchasing

Buy 1 Technavio report and get the second for 50% off. Buy 2 Technavio reports and get the third for free.

Technavios healthcare and life sciences research analysts categorize the global cancer gene therapy market into the following segments by therapy. They are:

Looking for more information on this market? Request a free sample report

Technavios sample reports are free of charge and contain multiple sections of the report including the market size and forecast, drivers, challenges, trends, and more.

Oncolytic virotherapy

Oncolytic virotherapy is one of the fastest growing treatment modality. In this therapy, the anti-cancer cells specifically destroy the cancer cells without causing harm to the normal cells. Each virus has a specific cellular tropism that determines which tissue will be preferentially infected by the virus and thus will further lead to the disease.

According to Sapna Jha, a lead oncology research analyst from Technavio, The oncolytic virotherapy has shown encouraging results in the pre-clinical studies. The novel treatment option holds great opportunity to make a significant effect on quality and length of the life of the individual. Adenovirus is the most commonly used virus in oncolytic virotherapy.

Gene transfer

Gene transfer or gene insertion is one of the most exciting and emerging cancer treatment methods. The therapy is expected to be the fastest growing type of therapy in the cancer gene therapy market. This is a radical new treatment method that involves the introduction of a new gene into the cancer cell or the surrounding tissues.

Genes with different functions have been proposed for this therapy; some of them include antiangiogenesis genes, cellular stasis genes, and suicide genes. Many different viral vectors are used to deliver these genes, Adenovirus being most common of them. Other than viral vectors, certain non-viral methods are also studied in the various clinical trial, which includes oligodendromer DNA coatings and naked DNA transfer, adds Sapna.

Gene-induced immunotherapy

Immunotherapy works on the concept of boosting the immune system of the individual to target and destroy cancer cells. However, traditional immunotherapy has shown limited success rate in the field. Various gene therapy techniques are being used to overcome this limitation.

The next-generation gene-induced immunotherapy vaccines are already in clinical trial. Gene-induced immunotherapy is a type of gene therapy where genetically engineered genes are used to generate an immune response against cancer. Growing knowledge and understanding of mechanisms regulating the initiation and maintenance of cytotoxic immune response has led to the designing of several genetic immunization strategies.

The top vendors highlighted by Technavios research analysts in this report are:

Browse Related Reports:

About Technavio

Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions.

With over 500 specialized analysts, Technavios report library consists of more than 10,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

If you are interested in more information, please contact our media team at media@technavio.com.

View post:
Cancer Gene Therapy Market - Forecasts and Opportunity Assessment by Technavio - Business Wire (press release)

Imagine only being able to see the things in front of you in soft focus, and just in black and white. For people with the genetic eye condition achromatopsia those are just some of the side effects. But, researchers are testing a new treatment designed to cure the condition by fixing the gene responsible.

Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun.

I need to have very dark, very tinted sunglasses to feel comfortable outside and to see really well. Cataldo said.

Cataldo has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light. She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance.

I cannot drive a car so I rely on public transportation and my bike to get around. Cataldo explained.

"There are currently no approved and no effective treatments for achromatopsia, said University of Florida surgical ophthalmologist Christine Kay.

Kay is working to change that. She is one of a handful of experts testing a gene therapy.

For achromatopsia the cells we have to target are cone cells responsible for decreased vision and color vision and those are cells at the very bottom layer of the retina, Kay explained.

Using a tiny cannula, surgeons deliver a normal copy of one of two mutated genes; the CNGA3 or CNGB3 gene, directly into the eye - restoring vision.

Cataldos myopia is so severe that her risk of retinal detachment from any retinal surgery is high, which rules her out for the current trial. In the meantime, Cataldo says shes learned to adapt to achromatopsia and live without limitations.

And I hope all young achromats learn the same thing, Cataldo said.

Kay says if the gene could eventually be delivered to the surface of the retina; additional patients, like Cataldo, could be treated. Applied Genetic Technologies Corporation, the biotech company that developed the therapies, and several U.S. universities have successfully tested this therapy in dogs and sheep.

Published at 2:10 PM CDT on Aug 16, 2017

Read the original here:
Achromatopsia: Gene Therapy Cures Eye Disease? - NBC 5 Dallas-Fort Worth

The global cancer gene therapy market to grow at a CAGR of 20.63% during the period 2017-2021.

The report, Global Cancer Gene Therapy Market 2017-2021, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.

One trend in the market is favorable government regulations for gene therapy programs. Gene therapy has been a big hope for the individuals suffering from rare diseases and various forms of cancers. Cancer is one of the biggest life-threatening diseases across the globe and has been a huge point of concern for the healthcare providers. This has led to rising support to R&D activities in cancer gene therapy from various governments.

According to the report, one driver in the market is rising geriatric population. The global geriatric population is continues to grow at a faster pace due to several factors such as rapidly falling fertility rates and growing life expectancy due to better medical facilities. The US Census Bureau reported that the total population aging 65 years and above was estimated at 617 million in 2015 and is expected to rise to 1,566 million by 2050. Asia has the largest and fastest growing aging population due to several factors such as the huge population of the region, government policies such as one child policy of China has reduced the addition of young population, and higher investment in the healthcare sector has led to better medical facilities and longer life expectancy.

Further, the report states that one challenge in the market is high cost of gene therapy treatment. The sky-high cost of gene therapy has made it a point of debate across the globe. One of the major causes for the exorbitant cost of gene therapy is the requirement of intensive clinical trials. The gene therapy needs to be tailored to suit the genetic acceptance for each individual, unlike small and large molecule drug clinical trials where the trials are done in large and random samples. In the clinical trials of gene therapy, the stem cell is extracted from each patient's bone marrow, then the correct version of the gene is mingled with the cell, and the corrected stem cells are introduced to the individual patient.

Key vendors

Other prominent vendors

Key Topics Covered:

Part 01: Executive Summary

Part 02: Scope Of The Report

Part 03: Research Methodology

Part 04: Market Landscape

Part 05: Pipeline Analysis

Part 06: Market Segmentation By Therapy

Part 07: Geographical Segmentation

Part 08: Decision Framework

Part 09: Drivers And Challenges

Part 10: Market Trends

Part 11: Vendor Landscape

Part 12: Key Vendor Analysis

Part 13: Appendix

For more information about this report visit https://www.researchandmarkets.com/research/drvk7v/global_cancer

Media Contact:

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

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View original content:http://www.prnewswire.com/news-releases/global-cancer-gene-therapy-market-to-grow-at-a-cagr-of-206-by-2021-favorable-government-regulations-for-gene-therapy-programs-300504680.html

SOURCE Research and Markets

http://www.researchandmarkets.com

Read the original:
Global Cancer Gene Therapy Market to Grow at a CAGR of 20.6% by 2021: Favorable Government Regulations for ... - PR Newswire (press release)

(Logo: http://mma.prnewswire.com/media/523989/Visiongain_Logo.jpg )

How this report will benefit you:

Read on to discover how you can exploit the future business opportunities emerging in this sector.

In this brand-new127 page reportyou will receive70 charts- all unavailable elsewhere.

The127-page report provides clear detailed insight into the gene therapy market. Discover the key drivers and challenges affecting the market.

By ordering and reading our brand-new report today you stay better informed and ready to act.

Report Scope:

Gene Therapy market forecastsfrom2017-2027

This reportassesses the approved gene therapy productsin the market and givesrevenue to 2027 for Neovasculgen

Providesqualitative analysis and forecastof thesubmarket by indicationfor the period 2017-2027: Cancer Cardiovascular disorders Rare diseases Ophthalmological diseases Other therapeutic uses

Profilesleading companiesthat will be important in the development of the gene therapy market. For each company, developments and outlooks are discussed and companies covered in this chapter include: UniQure Biogen Bluebird Bio Spark Therapeutics Applied Genetics Technologies Corporation Oxford Biomedica GenSight Biologics

Assesses the outlook for theleading gene treatment R&D pipelinefor 2016 and discusses technological progress and potential. Profiles appear for gene therapy drug candidates, withrevenue forecasts for six leading agents: SPK-RPE65 (Spark Therapeutics) Collategene (AMG0001, AnGes MG/Vical) Invossa (TissueGene-C, TissueGene Inc/Kolon Life Science) BC-819 (BioCancell) Lenti-D (Bluebird Bio) GSK2696273 (GlaxoSmithKline)

Provides qualitative analysis of trends that will affect the gene therapies market, from the perspective of pharmaceutical companies, during the period 2017 to 2027.SWOT analysisis provided andan overview of regulation of the gene therapy market by leading regiongiven.

Our study discussesfactors that influence the marketincluding these: Translation of research into marketable products modifying human DNA - gene transfer for therapeutic use, altering the nuclear genome Genomic editing technology and other supporting components Collaborations to develop and launch gene-based products - acquisitions and licensing deals Supporting technologies for human genetic modification, gene replacement and targeted drug delivery Gene therapies for ophthalmologic diseases - next-generation medicines Regulations in the United States, the European Union and Japan - overcoming technological and medical challenges to pass clinical trials.

Visiongain's study is intended for anyone requiring commercial analyses for the gene therapy market. You find data, trends and predictions.

Buy our report todayGene Therapy R&D and Revenue Forecasts 2017-2027: Cancer, Cardiovascular, Rare Diseases, Ophthalmologic, Other Diseases.

To request a report overview of this report please email Sara Peerun at sara.peerun@visiongain.com or call Tel: +44-(0)-20-7336-6100

Or click on https://www.visiongain.com/Report/1954/Gene-Therapy-R-D-and-Revenue-Forecasts-2017-2027

List of Companies and Organisations Mentioned in the Report:

Active Medical, Inc.

AngioDynamics, Inc.

Aspen Laboratories

AtriCure, Inc.

Barcapel Foundation

Biosense Webster, Inc.

Boston Scientific Corporation

British Association of Aesthetic Plastic Surgeons (BAAPS)

BSD Medical Corporation

C.R. Bard

Cosman medical, Inc.

Covidien

DFINE, Inc.

Endosense SA

Ethicon

Food and Drug Administration (FDA)

Galil medical, Inc.

Johnson & Johnson

Linvatec Canada ULC

Macmillan Cancer Support

Medtronic

Microsulis Medical Ltd.

Monteris Medical

National Institute of Health Research (NIHR)

nContact, Inc.

NeuroTherm, Inc.

NeuWave Medical, Inc.

NxThera, Inc.

Olympus Corporation

Perseon Corporation

Profound Medical Corp.

Royal Brompton & Harefield NHS Foundation Trust

Royal Philips

Shandong Provincial Hospital

Smith & Nephew

SonaCare Medical

St Jude Medical

Terumo Europe

The American Heart Association

Trod Medical N.V.

University College London

To see a report overview please email Sara Peerun on sara.peerun@visiongain.com

SOURCE Visiongain Ltd

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Gene Therapy R&D and Revenue Forecasts 2017-2027 - PR Newswire (press release)

DUBLIN--(BUSINESS WIRE)--The "Global Cancer Gene Therapy Market 2017-2021" report has been added to Research and Markets' offering.

The global cancer gene therapy market to grow at a CAGR of 20.63% during the period 2017-2021.

The report, Global Cancer Gene Therapy Market 2017-2021, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.

According to the report, one driver in the market is rising geriatric population. The global geriatric population is continues to grow at a faster pace due to several factors such as rapidly falling fertility rates and growing life expectancy due to better medical facilities. The US Census Bureau reported that the total population aging 65 years and above was estimated at 617 million in 2015 and is expected to rise to 1,566 million by 2050.

Asia has the largest and fastest growing aging population due to several factors such as the huge population of the region, government policies such as one child policy of China has reduced the addition of young population, and higher investment in the healthcare sector has led to better medical facilities and longer life expectancy.

Key vendors

Other prominent vendors

Key Topics Covered:

Part 01: Executive Summary

Part 02: Scope Of The Report

Part 03: Research Methodology

Part 04: Market Landscape

Part 05: Pipeline Analysis

Part 06: Market Segmentation By Therapy

Part 07: Geographical Segmentation

Part 08: Decision Framework

Part 09: Drivers And Challenges

Part 10: Market Trends

Part 11: Vendor Landscape

Part 12: Key Vendor Analysis

Part 13: Appendix

For more information about this report visit https://www.researchandmarkets.com/research/hrzr8h/global_cancer

Read more:
Global Cancer Gene Therapy Market to Grow at a CAGR of 20.6 ... - Business Wire (press release)

Human protein-coding genes number from 20,000 up to 25,000.

If just one of these genes gets altered or a code gets missing, it can be fatal to an individual.

In fact, approximately 30 per cent of infant mortality at birth in developed countries are caused by genetic disease. Almost 50 per cent of all miscarriages worldwide are due to chromosomally defective fetus.

Furthermore, according to the World Health Organization, over 10,000 human diseases are linked to single gene mutation alone. Among these monogenic diseases are thalassaemia, sickle cell anemia, haemophilia, Fragile-X syndrome, cystic fibrosis, and Huntingtons disease.

The other two major types of genetic disorders are chromosomal and complex disorder, where theres mutation in two or more genes.

Genetic disease is not also simply inherited, our environment is another factor that can trigger mutation. Cancer, diabetes, and heart disease are classified as multifactorial inheritance genetic disorders.

Considering all these, one would expect that the world will be welcoming the revolutionary gene therapy with wide-open arms.

Yet, UniQures Glybera has been recently withdrawn from the European market in spite of its promising one-time cure for lipoprotein lipase deficiency (LPLD).

LPLD is a rare genetic disorder characterized by the bodys lack of lipase, which is an enzyme that breaks down triglycerides from the blood. The deficiency results to recurrent abdominal pain, fat deposits in the skin (xanthomata), and repeated attacks of acute pancreatitis. LPLD is known to affect one person in a million. However, UniQures Glybera costs as much as $1 million per patient. Since the drugs introduction in 2012, only one patient has been subscribed to the treatment.

Another genetic drug that offers one-time cure for Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) is GlaxoSmithKlines Strimvelis. ADA-SCID is an inherited genetic condition characterized by a damaged immune system. People with SCID are prone to persistent and recurring infections since they absolutely have no immune protection from microbes. Symptoms begin to appear in a babys first 6 months of life, and afflicted infants hardly reach two years of age without treatment.

GlaxoSmithKlines Strimvelis can cure the genetic disease and save precious lifes. But the $700,000 drug had only a couple of sales in 2016 and another two expected this year. With this disappointing development, GSK might simply sell its rare diseases unit.

Data shows that the prices of the current gene therapy in the market are too hard if not impossible for most families to reach, especially since it has to be a one-time payment. And health care systems which only pay on monthly basis are not of much help to pharmaceuticals, which have made such enormous investments to formulate genetic cures.

Is there real hope?

Many drug companies still think so.

Pfizer, Sanofi, and Shire are now also making the revolutionary pursuits. And GSK has not completely given up as it strives to use its gene therapy platform in the development of cure for more common genetic illnesses.

Yes, at the moment, the whole picture may appear dim. But, by creating new business models, the leading companies in the biopharmaceutical industry if they are really serious about doing something in relation to rampant increases in drug prices, can start by creating a business model which is first based on humanism and then their respective bottom lines.

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Gene Therapy is Finally Here, But Who Will Foot the Bill - Wall Street Pit

LONDON, Aug 8 (Reuters) - Gene therapy, which aims to patch faulty genes with working DNA, has been a long time in development. The following are major milestones:

1972 - Researchers first suggest gene therapy as a treatment for genetic diseases but oppose its use in humans "for the foreseeable future", pending greater understanding of the technology.

1990 - A four-year-old girl with severe immunodeficiency became the first patient to undergo gene therapy in the United States.

1999 - American patient Jesse Gelsinger dies following a gene therapy experiment, setting the field back several years as U.S. regulators put some experiments on hold.

2002-03 - Cases of leukaemia are diagnosed in French children undergoing gene therapy in a further blow to the field.

2003 - The world's first gene therapy is approved in China for the treatment of head and neck cancer.

2007 - Doctors carry out the world's first operation using gene therapy to treat a serious sight disorder caused by a genetic defect.

2012 - Europe approves Glybera, the first gene therapy in a Western market, for an ultra-rare blood disorder.

2016 - Europe approves Strimvelis for a very rare type of immunodeficiency.

2017 or 2018 - The first gene therapy could be approved in United States. (Reporting by Ben Hirschler; editing by David Stamp)

Read this article:
TIMELINE-Gene therapy's long road to market - Reuters - Reuters

Pharmaceutical giant Pfizer is expanding its facilities in Sanford to accommodate the companys push into gene therapy.

The state Department of Commerce announced earlier this week that Pfizer would invest $100 million in the site and create 40 jobs there within three years. The average annual salary for the new positions will be $97,500, much higher than Lee Countys average annual wage of $38,250.

If it meets those goals, Wyeth Holdings, a wholly owned subsidiary of Pfizers, will receive a $250,000 grant from the One North Carolina Fund and a local incentive of up to $1,412,715 over five years.

The announcement comes a week after Gov. Roy Cooper visited Pfizers facilities in Sanford and a year after Pfizer bought Bamboo Therapeutics, a Chapel Hill startup. Pfizer also bought Bamboos gene therapy manufacturing facility, which Bamboo had acquired from UNC-Chapel Hill in January, 2016.

Gene therapy is an emerging technology that attacks the disorder by repairing mutated genes. Pfizer will use technology to introduce genetic material into a patients body so as to compensate for defective or missing genes.

More:
Pfizer's push into gene therapy adds more jobs in Sanford - News & Observer

Researchers from the University of Chicago have successfully completed a proof-of-concept studywhere they managed to treat obesity in mice using a new type of gene therapy that utilized skin transplants. Human skin transplantation is a well-established clinical approach that has been used for the treatment of burns. However, using it as a vehicle to deliver genetic treatments for non-skin diseases could be revolutionary.

There are several reasons why skin stem cell therapy can be applicable to a broad type of diseases. The skin is the largest human organ, providing an easy access to cells needed for genetic treatments. The skin enables easy monitoring for potential off-target mutations resulting from the CRISPR intervention, as well as easy removal of such mutations, should they occur. Most importantly, proteins that are secreted by epidermal cells can reach the blood circulation and achieve desired therapeutic effects for the entire body.

In the study published this month, titledEngineered Epidermal Progenitor Cells Can Correct Diet-Induced Obesity and Diabetes,the scientists genetically engineered skin cells to be able to deliver GLP1 (glucagon-like peptide 1) - a hormone which regulates blood glucose. Then they developed a surgical procedure which allowed them to successfully engraft the new skin onto a mouse host. Finally, the genetically modified cells had a mechanism for releasing GLP1, which was regulated by a small amount of antibiotic that was fed to the mice. The treated group of obese mice significantly reduced their body weight and insulin resistance, compared to the control group.

Xiaoyang Wu, one of the authors of the study, says in an interview for ResearchGate:

We established a novel mouse to mouse skin transplantation system to test skin gene therapy. [] Our proof-of-concept work demonstrated its possible to use engineered skin grafts to treat many non-skin diseases. Clinical translation of our findings will be relatively easy, as skin transplantation in human patients has been well established and clinically used for treatment of burn wounds for many years.

Skin grafts are an exciting new avenue to explore for genetic treatments of diseases. They are relatively inexpensive compared to other types of gene therapy, the procedure is minimally invasive, and it has already been tested and proven safe.

See the rest here:
A Novel Form of Gene Therapy Can Treat Diabetes With Genetically Modified Skin Transplants - Big Think

Pfizer is moving forward with plans to invest in a new clinical and commercial gene therapy manufacturing facility in Sanford, NC, but the work is still in the preliminary stages, said the company. A $100-million investment in the Sanford facilities is expected to create 40 jobs, according to a press release from the North Carolina governors office.

The facility will build upon a technology first developed at the University of North Carolina at Chapel Hill. Gene therapy focuses on highly specialized, one-time treatments that address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patients cells to compensate for a defective or missing gene.

Gene therapy is an important area of focus for Pfizer. In 2016, the company acquired Bamboo Therapeutics, a privately held biotechnology company based in Chapel Hill focused on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system. Pfizer also committed $4 million to support postdoctoral fellowships in North Carolina universities for training in gene therapy research, according to the press release.

A performance-based grant of $250,000 from the One North Carolina (NC) Fund will help facilitate Pfizers expansion. The One NC grant will formally be awarded to Wyeth Holdings, a wholly owned subsidiary of Pfizer. The One NC Fund provides financial assistance to local governments to help attract economic investment and to create jobs. Companies receive no money upfront and must meet job creation and capital investment targets to qualify for payment. All One NC grants require a matching grant from local governments and any award is contingent upon that condition being met.

Source: Pfizer, NC Governors Office

Continue reading here:
Pfizer Plans Gene Therapy Manufacturing Investment in North ... - Pharmaceutical Technology Magazine

Regenxbio has joined forces with investment firm OrbiMed and a new nonprofit foundation to create Prevail Therapeutics, a startup focused on new biologics and gene therapiesfor Parkinson's disease (PD).

Prevail will draw on the expertise of the Silverstein Foundation for Parkinson's with GBA, which concentrates on a particular form of the disease caused by mutations in the glucocerebrosidase gene.

The foundation was set up this year by OrbiMed's co-head of private equity Jonathan Silverstein, who was diagnosed with GBA-linked PD in February and is mobilizing efforts to discover a cure for the disease. Silverstein backed the foundation with $10 million of his own money, and is intent on accelerating research into PD with GBA as well as other forms of the disease.

Prevail says it will focus initially on research coming out of the lab of its co-founder and CEO Asa Abeliovich, M.D., Ph.D., who is on the faculty of Columbia University as well as being a scientific adviser to the Silverstein Foundation and co-founder of neurodegenerative disease biotech Alector.

By joining forces with Regenxbio, Prevail launches with an exclusive license to the gene therapy specialist's adeno-associated virus (AAV) based vector technology NAV AAV9 for PD and other neurodegenerative disorders.

Silverstein said that the NAV platform and Dr. Abeliovich's "deep expertise in the molecular mechanisms of neurodegeneration provides us with a promising opportunity to develop potential life-changing therapies for patients suffering from Parkinson's disease and other neurodegenerative diseases."

He told CNBC today that Prevail's board will also have some big names, including Leonard Bell, co-founder and former CEO of Alexion, OrbiMed venture partner and Alexion co-founder Steve Squinto and serial entrepreneur Peter Thompson of Silverback Therapeutics and Corvus Pharmaceuticals.

The new company will initially focus on GBA1, the most common of the PD mutations, which is estimated to be present in up to 10% of U.S. PD patients and perhaps 100,000 people worldwide. The disease mechanism linked to the mutationan accumulation of alpha-synuclein in the brainmay have implications for the broader PD population and other neurodegenerative diseases.

"Many of the drugs we are trying for Parkinson's with GBA may work in the broader Parkinson's population," said Silverstein. The aim will be to get drugs approved for use in GBA patients first, and then expand their use into other patient groups.

The work of the foundation is attracting investment from companies who are not even active in PD, with cancer specialist Celgene today pledging a grant of $5 million.

See original here:
Silverstein-backed startup will test gene therapy for Parkinson's - FierceBiotech

A new consortium, led by Oxford BioMedica, will embark on a two-year, 2 million project focused on gene and cell therapy manufacturing.

Other partners include the Cell and Gene Therapy Catapult, Stratophase and Synthace, and the collaboration is co-funded by Innovate UK.

The aim of the consortium is to explore and apply novel advanced technologies to further evolve OXBs proprietary suspension LentiVector platform to deliver higher quality vectors for both clinical and commercial use. The project aims to deliver tangible benefits to patients by shortening the time-to-clinic and time-to-market as well as to improve the cost and access of bringing novel gene and cell therapies to patients.

Each partner in the collaboration holds proprietary technology and know-how that can be used to develop an innovative approach to viral vector manufacturing. The aims of this pioneering project are closely aligned with the current government national priorities to make the UK a global hub for manufacturing advanced therapies, which will benefit economic growth and create and retain more highly skilled employment.

John Dawson, CEO of Oxford BioMedica, commented: Cell and gene therapies offer unprecedented promise for the cure, treatment or long term management of disease and we are delighted that this consortium has been awarded funding from Innovate UK that will help to keep Oxford BioMedica, our partners and the UK at the forefront of innovation in industrial viral vector manufacturing."

Keith Thompson, CEO of Cell and Gene Therapy Catapult, added: Collaborating on developing improved process analytic technologies with our partners will help drive productivity in viral vector manufacturing, accelerating the development of these transformative advanced therapies. We have the opportunity to both transform patients lives and grow an industry in the UK that we can be proud of.

More here:
2m UK consortium to tackle gene therapy - PharmaTimes