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Gene Therapy – Learn Genetics

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Challenges In Gene Therapy

Researchers hoping to bring gene therapy to the clinic face unique challenges.

learn more

Approaches To Gene Therapy

Beyond adding a working copy of a broken gene, gene therapy can also repair or eliminate broken genes.

learn more

Gene Therapy Successes

The future of gene therapy is bright. Learn about some of its most encouraging success stories.

learn more

Gene Therapy Case Study: Cystic Fibrosis

Originally posted here:

Gene Therapy – Learn Genetics

What is Gene Therapy? – news-medical.net

Gene therapy is a form of therapy that involves inserting one or more corrective genes that have been designed in the laboratory, into the genetic material of a patient’s cells to cure a genetic disease.

The expression of the new gene or genes can then alter the DNA or RNA transcript used to synthesis proteins and therefore correct the disease. Gene therapy is still in the experimental stages and its use is therefore not yet widespread.

Gene therapy using an adenovirus vector: A new gene is injected into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.

One of the first hurdles of gene therapy is successful introduction of the corrected gene into the human cells. Once introduced, the gene needs to be incorporated into the genetic material of those cells.

Some of the main diseases currently being researched in gene therapy studies include:

One of the main vectors used to carry modified genes into cells is the virus. Viruses usually attach to a host cell and transfer their viral genetic material into it. They then take over the cell and use the cell components to make copies of the virus.

Scientists can modify these viruses so that they contain only therapeutic and not viral genetic material but still retain their infective ability so that the beneficial DNA can be transferred into host cells.

Commonly used vectors include adenoviruses, adeno-associated viruses, retroviruses and the herpes simplex virus.

There are two main types of gene therapy which include:

With this form of therapy, only somatic cells (body cells) are targeted and not the germline cells, otherwise known of as the gametes or sex cells. If the modification of DNA is confined to body cells only, then the altered genome only effects the individual treated and not any offspring produced when the gametes join to form a zygote.

This involves incorporation of a tailored gene into the gametes, permanently altering the genes inherited by future generations. This type of therapy is prohibited in many countries due to ethical and technical concerns.

Reviewed by Sally Robertson, BSc

See the original post:

What is Gene Therapy? – news-medical.net

Alliance for Cancer Gene Therapy (ACGT) Foundation

Spotlight News Spot

Video:ACGT fellow, Dr. Samuel Katz,Yale Medical Center, speak about cell and gene therapies for cancer treatment.

See all ACGT videos here

July 12, 2017

STAMFORD, Conn., July 12, 2017 Today the FDAs Oncologic Drugs Advisory Committee, an independent panel of experts, voted unanimously to recommend to the FDA Read More

June 26, 2017

On Saturday, June 24, 2017, more than 300 swimmers made waves in the fight to defeat cancer at the 11th annual Swim Across America Greenwich-Stamford Read More

May 25, 2017

Dear Friends: I have always considered myself to be a very lucky person, but I wasnt feeling very lucky the day I was told I Read More

June 22, 2017

I have always considered myself to be a very lucky person, but I wasnt feeling very lucky the day I was told I had chronic Read More

June 3, 2016

In 2012, Bob Levis, an Allentown, Pennsylvania resident, believed he had come to the end of his life. Diagnosed in 2002 with chronic lymphocytic leukemia, Read More

March 16, 2014

They took my cancer cells and irradiated them, and then injected them back into my body so my immune system would kick in, Morrow said. Read More

June 21, 2017

Alliance for Cancer Gene Therapy fellow, Dr. Samuel Katz at Yale Medical Center, speaks about cell and gene therapies for cancer treatment. There has been Read More

October 5, 2016

Dr. Stephen Eck, Member of Alliance for Cancer Gene Therapys Scientific Advisory Council, talks about the importance of personalized therapies for cancer. Historically, weve tried Read More

October 5, 2016

Dr. Michael Lotze, Member of Alliance for Cancer Gene Therapys Scientific Advisory Council speaks about progress in the fight against cancerhe has witnessed during his Read More

The mission of ACGT is to seek out and support revolutionary scientific research into the causes, treatment and prevention of all types of cancer using cells and genes as medicine.

Cell and gene therapies are destroying tumors every day in the laboratory and in human trials, targeting only the cancer with few side effects to healthy tissue. ACGTs rigorous grant review process, under the guidance of a preeminent Scientific Advisory Council, have resulted in some of the most successful breakthroughs in the history of cancer treatments and led to many patients in complete remission.

100% of our funding supports innovation and translational science that is proving again and again to offer better treatment options than the traditional therapies of chemotherapy, surgery and radiation. Every donation brings us closer to saving lives and changing the way we treat cancer forever.

Video:ACGT fellow, Dr. Samuel Katz,Yale Medical Center, speak about cell and gene therapies for cancer treatment.

See all ACGT videos here

At the ACGT anniversary gala in April, Robert Vonderheide, MD, DPhil, the Hanna Wise Professor in Cancer Research and newly appointed Director of the Abramson Read More

This fourth in our blog series on the progress of cell and gene therapy for cancer focuses on a treatment that has made huge strides Read More

In our last blog, we described the progress and promise of CAR-T, an immunotherapy that has been particularly successful with leukemia and lymphoma. As a Read More

2017 All rights reserved by ACGT. ACGT is a 501(c)(3) organization and all contributions are tax-deductible under the law of your state.

Read the original:

Alliance for Cancer Gene Therapy (ACGT) Foundation

Gene Therapy Net – News, Conferences, Vectors, Literature …

Posted on: 18 June 2017, source: mesothelioma.com Technological innovations are changing the world we live in every day, including the way society views diseases that have been around for hundreds of years. Cancer continues to be a leading cause of death around the world, but day by day we, as a society, are getting closer to finding a cure. In 2016, former President Barack Obama asked former Vice President Joe Biden to head the Cancer Moonshot Initiative (CMI), whose overarching goal would be to find a cure for cancer by the year 2020. In the time thats passed, Biden has formed a committee of governmental organizations and a Blue Ribbon Panel that provided recommendations of how to achieve the lofty goals, and set to bringing this moonshot into reality.

Read this article:

Gene Therapy Net – News, Conferences, Vectors, Literature …

Krystal files for IPO to move gene therapies into clinic | FierceBiotech – FierceBiotech

Krystal Biotech has filed to raise about $35 million in a Nasdaq IPO. The money will equip Krystals ex-Intrexon executive team to file an IND for its rare skin disease gene therapy in the first quarter of next year.

That gene therapy, KB103, is designed to treat dystrophic epidermolysis bullosa, a rare disease that makes the skin fragile and susceptible to blisters and tears. Krystal thinks it can improve outcomes in these patients, who currently only have palliative options, by using a HSV-1-based vector to fix defects in the COL7A1 gene.

Pittsburgh, Pennsylvania-based Krystal has yet to back up the idea with clinical data. But having run preclinical tests of topical and intradermal formulations of KB103 and met with the FDA late last year, it plans to embark on a whistle-stop development program. The plan is to file an IND in the first quarter, have results by the middle of the year and use that readout to guide the design of a pivotal trial.

Krystal will need more than the forecast IPO haul to execute that strategy, although it does expect to have some money left over from the Nasdaq listing to fund work on its follow-up program, KB104. That gene therapy treatment for Netherton syndrome is due to reach the IND stage by the second half of next year.

The early-stage nature of Krystals pipeline is one of several characteristics that mark it out as a biotech trying to make the leap to public markets early in its development as a company. Notably, given the financial reporting requirements for public companies, Krystal has yet to hire a full-time CFO or in-house principal accounting officer. The biotechs seven-person team is heavily skewed towardR&D.

Krish Krishnan, CEO, and Suma Krishnan, COO, lead this small team. The pair have worked together for most of the past 13 years, starting at New River Pharmaceuticals in the run-up to its takeover by Shire. Both Krishnans popped up next at Pinnacle Pharmaceuticals, where Krish was CEO, before reuniting with their former New River boss Randal Kirk at Intrexon.

Krystal sees the pair strike out on their ownand stand to win big if their gene therapies succeed.A family trust owned by Krish and Suma owns more than two-thirds of Krystals stock. The only other organization to own more than 5% of the company is Sun Pharma, which bought its 16.5% stake in a $7 million series A round earlier this month. That round bolstered Krystals cash position, which stood at $3.5 million at the end of June.

View post:

Krystal files for IPO to move gene therapies into clinic | FierceBiotech – FierceBiotech

Apic Bio Launches to Advance First-in-Class Gene Therapy for … – Business Wire (press release)

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Apic Bio, Inc., a pre-clinical stage gene therapy company leveraging its proprietary platform to advance therapies to treat rare diseases with complex mechanisms, in particular Alpha-1 Antitrypsin Deficiency (Alpha 1), launched today with an initial investment led by the venture philanthropy arm of the Alpha-1 Foundation and a private investor with the disease.

Its lead product, APB-101, targets the liver via an AAV delivered Dual Function Vector (df-AAV) whereby the Z-AAT protein is silenced and M-AAT protein is augmented. APB-101 has achieved a pre-clinical proof of concept with efficacy demonstrated in vitro and in vivo. It is currently undergoing pre-clinical GLP toxicology studies in non-human primates. Patients living with Alpha 1 lack sufficient levels of circulating AAT protein to protect lung tissue against damage from proteases, and experience the accumulation of mutant AAT polymers in the liver. Clinically, the deficiency is manifested by progressive emphysema and the accumulation presents a significant risk of liver cirrhosis.

John Reilly, Co-Founder & President said: We are grateful to TAP and A1AT Investors, LLC who have supported the successful start of Apic Bio by providing the first tranche of our seed financing round allowing us to secure key intellectual property rights and operational support. With such strong support from the advocacy and patient community, we are confident that we will identify the right corporate partners to help us achieve our business development goals and bring this exciting new therapy to patients.

The df-AAV platform allows treatment of other diseases with complex mechanisms where the mutant gene product must be reduced and the normal gene product must be augmented.

Dr. Chris Mueller, Co-founder and Chief Scientific Officer of Apic Bio said: We are encouraged by the feedback that we have received during our pre-IND meeting with the FDA that there is a clear path for us to conduct a first-in-human Phase 1/2 clinical study. Furthermore, we are very much looking forward to demonstrating the benefit of APB-101 to patients that have been living with alpha-1 and have had very little hope for a cure. Our data suggests this is a liver sparing approach for gene augmentation which may exceed the therapeutic and safety margins when compared to a strict gene augmentation without gene silencing that may exacerbate the underlying liver disease.

TAP is very pleased to provide this funding to Apic Bio. Their cutting-edge work on a therapy that addresses both the liver and lung disease brings us closer to finding a cure for Alpha-1 Antitrypsin Deficiency, thus fulfilling our mission, said Jean-Marc Quach, CEO for The Alpha-1 Project.

Todays launch of Apic Bio has been a long time coming for the hundreds of thousands of people who are challenged by Alpha 1, said Ed Krapels, who has been living with Alpha 1 and is the new companys first individual investor. Now that we are moving forward, we hope to work with patients, their advocates and researchers to make a cure readily available. Krapels added.

About Apic Bio: Apic Bio, Inc. is a spin-off from the University of Massachusetts Medical School (UMMS) and is based upon nearly 30 years of gene therapy research by its scientific founders Christian Mueller, PhD, Associate Professor of Pediatrics and a member of the Horae Gene Therapy Center at the University of Massachusetts Medical School, Terence R. Flotte, MD, the Celia and Isaac Haidak Professor in Medical Education, dean of the School of Medicine and provost and executive deputy chancellor of the University of Massachusetts Medical School; and colleagues at the Horae Gene Therapy Center. Their research is funded in part by an $11M grant from the National Heart, Lung, and Blood Institute (NHLBI).

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Apic Bio Launches to Advance First-in-Class Gene Therapy for … – Business Wire (press release)

Gene therapy darling Oxford BioMedica pares losses – Telegraph.co.uk

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.

View post:

Gene therapy darling Oxford BioMedica pares losses – Telegraph.co.uk

MHRA backs gene therapy in battle with ‘bubble baby’ disease – The Pharma Letter (registration)

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a Promising Innovative

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View original post here:

MHRA backs gene therapy in battle with ‘bubble baby’ disease – The Pharma Letter (registration)

Gene Therapy – Learn Genetics

learn more

Challenges In Gene Therapy

Researchers hoping to bring gene therapy to the clinic face unique challenges.

learn more

Approaches To Gene Therapy

Beyond adding a working copy of a broken gene, gene therapy can also repair or eliminate broken genes.

learn more

Gene Therapy Successes

The future of gene therapy is bright. Learn about some of its most encouraging success stories.

learn more

Gene Therapy Case Study: Cystic Fibrosis

See the original post:

Gene Therapy – Learn Genetics

What is Gene Therapy? – news-medical.net

Gene therapy is a form of therapy that involves inserting one or more corrective genes that have been designed in the laboratory, into the genetic material of a patient’s cells to cure a genetic disease.

The expression of the new gene or genes can then alter the DNA or RNA transcript used to synthesis proteins and therefore correct the disease. Gene therapy is still in the experimental stages and its use is therefore not yet widespread.

Gene therapy using an adenovirus vector: A new gene is injected into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.

One of the first hurdles of gene therapy is successful introduction of the corrected gene into the human cells. Once introduced, the gene needs to be incorporated into the genetic material of those cells.

Some of the main diseases currently being researched in gene therapy studies include:

One of the main vectors used to carry modified genes into cells is the virus. Viruses usually attach to a host cell and transfer their viral genetic material into it. They then take over the cell and use the cell components to make copies of the virus.

Scientists can modify these viruses so that they contain only therapeutic and not viral genetic material but still retain their infective ability so that the beneficial DNA can be transferred into host cells.

Commonly used vectors include adenoviruses, adeno-associated viruses, retroviruses and the herpes simplex virus.

There are two main types of gene therapy which include:

With this form of therapy, only somatic cells (body cells) are targeted and not the germline cells, otherwise known of as the gametes or sex cells. If the modification of DNA is confined to body cells only, then the altered genome only effects the individual treated and not any offspring produced when the gametes join to form a zygote.

This involves incorporation of a tailored gene into the gametes, permanently altering the genes inherited by future generations. This type of therapy is prohibited in many countries due to ethical and technical concerns.

Reviewed by Sally Robertson, BSc

Link:

What is Gene Therapy? – news-medical.net

Alliance for Cancer Gene Therapy (ACGT) Foundation

Spotlight News Spot

Video:ACGT fellow, Dr. Samuel Katz,Yale Medical Center, speak about cell and gene therapies for cancer treatment.

See all ACGT videos here

July 12, 2017

STAMFORD, Conn., July 12, 2017 Today the FDAs Oncologic Drugs Advisory Committee, an independent panel of experts, voted unanimously to recommend to the FDA Read More

June 26, 2017

On Saturday, June 24, 2017, more than 300 swimmers made waves in the fight to defeat cancer at the 11th annual Swim Across America Greenwich-Stamford Read More

May 25, 2017

Dear Friends: I have always considered myself to be a very lucky person, but I wasnt feeling very lucky the day I was told I Read More

June 22, 2017

I have always considered myself to be a very lucky person, but I wasnt feeling very lucky the day I was told I had chronic Read More

June 3, 2016

In 2012, Bob Levis, an Allentown, Pennsylvania resident, believed he had come to the end of his life. Diagnosed in 2002 with chronic lymphocytic leukemia, Read More

March 16, 2014

They took my cancer cells and irradiated them, and then injected them back into my body so my immune system would kick in, Morrow said. Read More

June 21, 2017

Alliance for Cancer Gene Therapy fellow, Dr. Samuel Katz at Yale Medical Center, speaks about cell and gene therapies for cancer treatment. There has been Read More

October 5, 2016

Dr. Stephen Eck, Member of Alliance for Cancer Gene Therapys Scientific Advisory Council, talks about the importance of personalized therapies for cancer. Historically, weve tried Read More

October 5, 2016

Dr. Michael Lotze, Member of Alliance for Cancer Gene Therapys Scientific Advisory Council speaks about progress in the fight against cancerhe has witnessed during his Read More

The mission of ACGT is to seek out and support revolutionary scientific research into the causes, treatment and prevention of all types of cancer using cells and genes as medicine.

Cell and gene therapies are destroying tumors every day in the laboratory and in human trials, targeting only the cancer with few side effects to healthy tissue. ACGTs rigorous grant review process, under the guidance of a preeminent Scientific Advisory Council, have resulted in some of the most successful breakthroughs in the history of cancer treatments and led to many patients in complete remission.

100% of our funding supports innovation and translational science that is proving again and again to offer better treatment options than the traditional therapies of chemotherapy, surgery and radiation. Every donation brings us closer to saving lives and changing the way we treat cancer forever.

Video:ACGT fellow, Dr. Samuel Katz,Yale Medical Center, speak about cell and gene therapies for cancer treatment.

See all ACGT videos here

At the ACGT anniversary gala in April, Robert Vonderheide, MD, DPhil, the Hanna Wise Professor in Cancer Research and newly appointed Director of the Abramson Read More

This fourth in our blog series on the progress of cell and gene therapy for cancer focuses on a treatment that has made huge strides Read More

In our last blog, we described the progress and promise of CAR-T, an immunotherapy that has been particularly successful with leukemia and lymphoma. As a Read More

2017 All rights reserved by ACGT. ACGT is a 501(c)(3) organization and all contributions are tax-deductible under the law of your state.

Go here to see the original:

Alliance for Cancer Gene Therapy (ACGT) Foundation

Gene Therapy Net – News, Conferences, Vectors, Literature …

Posted on: 18 June 2017, source: mesothelioma.com Technological innovations are changing the world we live in every day, including the way society views diseases that have been around for hundreds of years. Cancer continues to be a leading cause of death around the world, but day by day we, as a society, are getting closer to finding a cure. In 2016, former President Barack Obama asked former Vice President Joe Biden to head the Cancer Moonshot Initiative (CMI), whose overarching goal would be to find a cure for cancer by the year 2020. In the time thats passed, Biden has formed a committee of governmental organizations and a Blue Ribbon Panel that provided recommendations of how to achieve the lofty goals, and set to bringing this moonshot into reality.

See original here:

Gene Therapy Net – News, Conferences, Vectors, Literature …

Spark’s Gene Therapy SPK-8011 to Treat Hemophilia A and B Advance in Clinical Trials – Hemophilia News Today

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.

Read the rest here:

Spark’s Gene Therapy SPK-8011 to Treat Hemophilia A and B Advance in Clinical Trials – Hemophilia News Today

Gene therapy darling Oxford BioMedica pares losses – Telegraph.co.uk

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.

Read the rest here:

Gene therapy darling Oxford BioMedica pares losses – Telegraph.co.uk

Sangamo Therapeutics: The Breakthrough In Gene Therapy – Seeking Alpha

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 more here:

Sangamo Therapeutics: The Breakthrough In Gene Therapy – Seeking Alpha

YOUR HEALTH Gene therapy for better eyesight – WQAD.com

Please enable Javascript to watch this video

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.

More here:

YOUR HEALTH Gene therapy for better eyesight – WQAD.com

Gene Therapy for Huntington’s Disease in Development – Rare Disease Report

Uniqure, the company best known for having the first approved gene therapy put on the market (for familial chylomicronemia syndrome), is expanding its pipeline to include a gene therapy for Huntingtons disease. While still in early development, the company stated they plan to file an Investigational New Drug (IND) application next year with the US Food and Drug Administration (FDA) in order to begin clinical studies in humans. Huntington’s disease is a genetic neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline when a person enters their 3rd or 4th decade. The disease is an autosomal dominant mutation, meaning that if a person has the condition, there is a 50% chance their offspring will have it as well, and is due to a mutation on the huntingin gene. Despite good understanding of the condition, current treatments can only alleviate the symptoms of it, but they do not delay the onset or slow the progression of the disease. It is hopeful that gene therapy can do that. In Huntingtons disease animal models, the gene therapy called AMT-130, an AAV5 vector carrying a DNA cassette encoding artificial micro-RNA, is effective. In April, data was presented at the 12th Annual CHDI HD Therapeutics Conference in Malta showing the drug was able to silence the human mutant huntingtin gene in pig model. In the animal study, AMT-130 was administered into the striatum and thalamus of minipigs that had the mutant Huntingtin gene. Three months after treatment, the vector was observed throughout the minipig brain and expression of mutant HTT mRNA was significantly reduced in the striatum and thalamus by 50% to 80% and reductions were also observed in the cortex (reduced up to 40% compared to controls). Lead author of that study, Prof. Jan Motlik, Director of the Institute of Animal Physiology and Genetics in the Czech Republic said, “This study demonstrated that a single administration of AAV5-miHTT resulted in significant reductions in HTT mRNA in all regions of the brain transduced by AMT-130, as well as in the cortex. Consistent with the reduction in HTT mRNA, we also observed a clear dose-dependent reduction in mutant huntingtin protein levels in the brain, with similar trends in the cerebral spinal fluid. Taking into account the similarities of CHDI’s proprietary transgenic pig model to the human brain, these results provide additional data to support moving forward with clinical trials of uniQure’s promising gene therapy for Huntington’s disease.” For more news and information about orphan drugs in development, followRare Disease ReportonFacebookandTwitter.

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Gene Therapy for Huntington’s Disease in Development – Rare Disease Report

YOUR HEALTH Gene therapy for better eyesight – WQAD.com

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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.

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YOUR HEALTH Gene therapy for better eyesight – WQAD.com

Gene therapy darling Oxford BioMedica pares losses – Telegraph.co.uk

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.

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Gene therapy darling Oxford BioMedica pares losses – Telegraph.co.uk

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

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.

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


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