From MIT Technology Review.
Read the entire article here:
http://www.technologyreview.com/news/542371/a-tale-of-do-it-yourself-gene-therapy/
One Seattle-area woman says she has tried exactly that. Her claim has entangled some high-profile American academics in a strange tale of do-it-yourself medicine that involves plane flights to Latin America, an L.A. film crew, and what’s purported to be the first attempt to use gene therapy to forestall normal aging.
Elizabeth Parrish, the 44-year-old CEO of a biotechnology startup called BioViva, says she underwent a gene therapy at an undisclosed location overseas last month, a first step in what she says is a plan to develop treatments for ravages of old age like Alzheimer’s and muscle loss. “I am patient zero,” she declared during a Q&A on the website Reddit on Sunday. “I have aging as a disease.”
...
Another prominent science advisor listed by BioViva is Harvard Medical School genomics expert George Church, who also includes BioViva on his own website of around 100 companies he collaborates with. Church said last week he was also trying to learn what exactly had occurred in Latin America. “I think it is real,” he said in an interview. “There were some indications it might happen. Companies in stealth mode can do anything they want.”
Church says he didn’t agree with dodging regulators and added that BioViva appears to be “a one-person show.” But he says he found Parrish’s claims plausible. A student in his lab, he says, could prepare a genetic treatment suitable for experiments in animals in a matter of days.
Wednesday, October 14, 2015
Sunday, October 11, 2015
Philly Gene Therapy Renaissance
Sangamo collaborators mentioned in article:
"
Philadelphia's gene therapy community last week marked another milestone in its resurgence when locally based Spark Therapeutics said its most advanced product helped restore some vision in patients suffering from a rare eye condition during a clinical trial. The company plans to apply for FDA approval next year.
Philadelphia was in some minds the gene therapy hub in the late 1990s, until an 18-year-old man died during a clinical trial by University of Pennsylvania researcher James M. Wilson. The death set back the field for years.
But now, Philadelphia is among the hubs, with research centers, companies, and dozens of researchers trying to cure diseases by injecting genetic material in people's cells to correct mutations.
"The little-known secret in this narrow space called gene therapy - now a little less narrow - is that Philadelphia is the best place because of the concentration of talent," said Spark
Read more at http://www.philly.com/philly/business/20151011_Gene_therapy_headline_here_in_1_line_of_36-point_type.html#kZmaOhmV5mPIKBsm.99
"
Philadelphia's gene therapy community last week marked another milestone in its resurgence when locally based Spark Therapeutics said its most advanced product helped restore some vision in patients suffering from a rare eye condition during a clinical trial. The company plans to apply for FDA approval next year.
Philadelphia was in some minds the gene therapy hub in the late 1990s, until an 18-year-old man died during a clinical trial by University of Pennsylvania researcher James M. Wilson. The death set back the field for years.
But now, Philadelphia is among the hubs, with research centers, companies, and dozens of researchers trying to cure diseases by injecting genetic material in people's cells to correct mutations.
"The little-known secret in this narrow space called gene therapy - now a little less narrow - is that Philadelphia is the best place because of the concentration of talent," said Spark
Read more at http://www.philly.com/philly/business/20151011_Gene_therapy_headline_here_in_1_line_of_36-point_type.html#kZmaOhmV5mPIKBsm.99
Friday, October 9, 2015
MPN Forum Reviews Sangamo's Gene Editing Work, CCR5 Trial subject 71 week Treatment Interruption
Gene edited SCT is already here
mpnforum.com/gene-editing-stem-cells-for-autologous-transplant/
Gene editing for blood disease?
No problem — it’s already happening!
When gene editing comes to MPN therapy, it will not be its first clinical application. And CRISPR/Cas9 won’t be the first editing tool. Clinical trials for gene-edited therapies for blood disorders are already wrapping up. And more are in the works.
Today, people are walking around clear or awful diseaes like HIV because of successful insertions or deletions into DNA. In fact, the history of gene editing pre-dates the discovery of CRISPR/Cas9. Because of the intense need for treatment of diseases like HIV, hemophilia, and sickle cell anemia — and because blood is such a readily available tissue — gene editing of human hematopoietic stem cells has advanced earlier and further than other human in vivo application.
At the Cold Spring Harbor Laboratory’s genome engineering meeting, Dr. Fyodor Urnov, of Sangamo Biosciences, reported on the disruption of HIV infection through editing of the CCR5 gene.
CCR5 is a chemokine receptor on the surface of white blood cells. While its function as part of the immune system is to attract T cells to cellular targets, its function as a gatekeeper makes it an easy entry point for the HIV, AIDS-causing virus.
HIV and leukemia? Call me lucky.
One patient,Timothy Brown, had been on anti-retroviral drugs to treat his HIV for 10 years before he was diagnosied with Acute Myeloid Leukemia. In his ensuing bone marrow transplant he was treated with stem cells that contained the CCR5 mutation on both strands of DNA. At the end, he left the hospital cured of both diseases…and remains clear of HIV six years later. This experience spurred research to knock out the CCR5 gene.
Discovery that some populations, due to mutation in CCR5, were naturally immune to HIV, led a number of drug companies to field CCR5-inhibitors with little or no success. It took the scissors of gene editing in the form of zinc finger nucleases to mutate the gene. Ex vivo, these mutated cells were expanded before reintroduction into a patient following full ablation.
Working with the NIH and the California Institute for Regenerative Medicine (CIRM), California’s stem cell agency, Sangamo Biosciences has applied gene editing to other blood disorders like hemophilia and beta-thelassemia , some of which are in or pending clinical trial.
The HIV work, extending back to the first FDA approved IND in 2009, (ClinicalTrials.gov NCT00979238) introduced interruption of the CCR5-inhibition via zinc finger nuclease edited HSCs. Since that time over 70 patients have been successfully treated.
“We have subjects, “said Urnov, “who have stopped taking their antiretroviral drug and are controlling their viral load without any medication. The longest a subject on the trial has gone without having to take retroviral drugs is 71 weeks. Think about it. This person was 24 years old and either faced the prospect of taking expensive and side effect-laden drugs the rest of his life or simply experience this clinical benefit.”
Hemophilia B
Sangamo’s work in Hemophilia B is “an alternative to the $500,000 annual cost of protein replacement delivery,” currently the alternative. The villain: Loss of factor IX (FIX) synthesis. In 2011, publication of a study in Nature, In vivo established proof of concept. Next step was to obtain NIH approval for human trial aimed at restoring FIX synthesis through genome editing.
The Clinical trial Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B (NCT00979238) description lays out the case for gene editing. Hemophilia B is caused by an absence or abnormality in the gene that produces the factor IX protein.
Affected individuals cannot make a blood clot effectively and suffer from severe bleeding episodes. Repeated bleeding episodes, specifically into joints, can cause chronic joint disease and lead to disability.
According to the Phase I protocol, “This research study will test the safety of giving an affected individual a normal factor IX gene which can produce factor IX protein in his body. We will give the normal gene for factor IX by using an inactivated (not able to function) virus called “the vector.” The vector used in this study was developed from an adeno-associated virus that has been changed so that it is unable to cause a viral infection in humans. This inactivated virus was further altered to carry the factor IX gene and to locate within liver cells where factor IX protein is normally made.”
Gene editing in myeloproliferative neoplasms– The CREATE Seminar
CRISPR-Cas9 could not have been used to edit the CCR5 in these gene editing efforts. The Sangamo program started nine years ago and CRISPR/Cas9 for all its widespread use is only three years old!
No problem — it’s already happening!
When gene editing comes to MPN therapy, it will not be its first clinical application. And CRISPR/Cas9 won’t be the first editing tool. Clinical trials for gene-edited therapies for blood disorders are already wrapping up. And more are in the works.
Today, people are walking around clear or awful diseaes like HIV because of successful insertions or deletions into DNA. In fact, the history of gene editing pre-dates the discovery of CRISPR/Cas9. Because of the intense need for treatment of diseases like HIV, hemophilia, and sickle cell anemia — and because blood is such a readily available tissue — gene editing of human hematopoietic stem cells has advanced earlier and further than other human in vivo application.
At the Cold Spring Harbor Laboratory’s genome engineering meeting, Dr. Fyodor Urnov, of Sangamo Biosciences, reported on the disruption of HIV infection through editing of the CCR5 gene.
Fyodor Urnov (L.) in discussion at CHSL (photo courtesy of Cold Spring Harbor Laboratories)
HIV and leukemia? Call me lucky.
One patient,Timothy Brown, had been on anti-retroviral drugs to treat his HIV for 10 years before he was diagnosied with Acute Myeloid Leukemia. In his ensuing bone marrow transplant he was treated with stem cells that contained the CCR5 mutation on both strands of DNA. At the end, he left the hospital cured of both diseases…and remains clear of HIV six years later. This experience spurred research to knock out the CCR5 gene.
Discovery that some populations, due to mutation in CCR5, were naturally immune to HIV, led a number of drug companies to field CCR5-inhibitors with little or no success. It took the scissors of gene editing in the form of zinc finger nucleases to mutate the gene. Ex vivo, these mutated cells were expanded before reintroduction into a patient following full ablation.
Working with the NIH and the California Institute for Regenerative Medicine (CIRM), California’s stem cell agency, Sangamo Biosciences has applied gene editing to other blood disorders like hemophilia and beta-thelassemia , some of which are in or pending clinical trial.
The HIV work, extending back to the first FDA approved IND in 2009, (ClinicalTrials.gov NCT00979238) introduced interruption of the CCR5-inhibition via zinc finger nuclease edited HSCs. Since that time over 70 patients have been successfully treated.
“We have subjects, “said Urnov, “who have stopped taking their antiretroviral drug and are controlling their viral load without any medication. The longest a subject on the trial has gone without having to take retroviral drugs is 71 weeks. Think about it. This person was 24 years old and either faced the prospect of taking expensive and side effect-laden drugs the rest of his life or simply experience this clinical benefit.”
Hemophilia B
Sangamo’s work in Hemophilia B is “an alternative to the $500,000 annual cost of protein replacement delivery,” currently the alternative. The villain: Loss of factor IX (FIX) synthesis. In 2011, publication of a study in Nature, In vivo established proof of concept. Next step was to obtain NIH approval for human trial aimed at restoring FIX synthesis through genome editing.
The Clinical trial Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B (NCT00979238) description lays out the case for gene editing. Hemophilia B is caused by an absence or abnormality in the gene that produces the factor IX protein.
Affected individuals cannot make a blood clot effectively and suffer from severe bleeding episodes. Repeated bleeding episodes, specifically into joints, can cause chronic joint disease and lead to disability.According to the Phase I protocol, “This research study will test the safety of giving an affected individual a normal factor IX gene which can produce factor IX protein in his body. We will give the normal gene for factor IX by using an inactivated (not able to function) virus called “the vector.” The vector used in this study was developed from an adeno-associated virus that has been changed so that it is unable to cause a viral infection in humans. This inactivated virus was further altered to carry the factor IX gene and to locate within liver cells where factor IX protein is normally made.”
Gene editing in myeloproliferative neoplasms– The CREATE Seminar
CRISPR-Cas9 could not have been used to edit the CCR5 in these gene editing efforts. The Sangamo program started nine years ago and CRISPR/Cas9 for all its widespread use is only three years old!
Today, work across multiple blood genetic diseases is being done by several well funded corporations like Editas, CRISPR Therapeutics, Intellia and giant Johnson and Johnson’s Transposagen. When this Sangamo work began, Zinc Finger nucleases were the scissors of choice. Today increasingly accurate and powerful CRISPR enzymes are available to follow the same path: Apheresis of hematopoietic stem cells, correction of mutation through ex vivo gene editing and reintroduction into the patient in an autologous trnasplant procedure.
The CREATE seminar — sponsored by the MPN Research Foundation and MPN Genetics Network — will be held two months from now in association with this year’s ASH meeting. The seminar brings together MPN and transplantation specialists with synthetic biologists and genetic engineers to explore steps necessary to get MPN gene editing into clinical trial.Wednesday, October 7, 2015
Sunday, October 4, 2015
Interesting Article on CRISPR Patent War
Excerpt:
There are no prizes for coming second, at least no Nobel prizes which is why everyone’s eyes will be on Stockholm next week when the greatest accolades in science will be announced.
Hot favourites for the chemistry prize are two scientists widely credited with discovering a revolutionary gene-editing technique that is changing the scientific landscape of everything from genetic medicine to the development of new crops and bio-products.
...
But a looming patent dispute threatens to overshadow next week’s announcement and may well scare off the Nobel committee from going anywhere near Crispr-Cas9 – the committee is notorious for two things; its obsessive secrecy and an institutional aversion to controversy. And the patent row is now making CRISPR exceedingly controversial.
Read the article here:
http://www.independent.co.uk/news/science/crispr-scientists-hopes-to-win-nobel-prize-for-gene-editing-technique-at-risk-over-patent-dispute-a6677436.html
There are no prizes for coming second, at least no Nobel prizes which is why everyone’s eyes will be on Stockholm next week when the greatest accolades in science will be announced.
Hot favourites for the chemistry prize are two scientists widely credited with discovering a revolutionary gene-editing technique that is changing the scientific landscape of everything from genetic medicine to the development of new crops and bio-products.
...
But a looming patent dispute threatens to overshadow next week’s announcement and may well scare off the Nobel committee from going anywhere near Crispr-Cas9 – the committee is notorious for two things; its obsessive secrecy and an institutional aversion to controversy. And the patent row is now making CRISPR exceedingly controversial.
Read the article here:
http://www.independent.co.uk/news/science/crispr-scientists-hopes-to-win-nobel-prize-for-gene-editing-technique-at-risk-over-patent-dispute-a6677436.html
Saturday, October 3, 2015
Sangamo Biosciences Represented on the Planning Committee Organizing the International Summit on Human Gene Editing
Dr. Fyodor Urnov of Sangamo Biosciences and UC-Berkeley will take part in the planing process for the International Summit on Human Gene Editing. This committee is composed of the luminaries in this field. This event will be webcast.
Information-Gathering Meeting for the Planning Committee
Organizing the International Summit on Human Gene Editing
Convened by:
The Chinese Academy of Sciences (CAS), The Royal Society (RS),
The U.S. National Academy of Sciences (NAS), and The U.S. National Academy of Medicine (NAM)
The Chinese Academy of Sciences (CAS), The Royal Society (RS),
The U.S. National Academy of Sciences (NAS), and The U.S. National Academy of Medicine (NAM)
Lecture Room
National Academy of Sciences Building
2101 Constitution Avenue, NW
Washington, D.C. 20418
National Academy of Sciences Building
2101 Constitution Avenue, NW
Washington, D.C. 20418
October 5, 2015
9 a.m. - 5:30 p.m. EDT
9 a.m. - 5:30 p.m. EDT
Agenda for Open Session
9:00 Welcome, Introductions, and Purpose of Today’s Meeting
David Baltimore, Summit Planning Committee Chair, California Institute of Technology
9:15 Session 1: Background and Historical Perspective on Embryo and Gene Manipulation
Speakers:
Robin Lovell-Badge, The Francis Crick Institute
Jane Maienschein, Arizona State University
Robin Lovell-Badge, The Francis Crick Institute
Jane Maienschein, Arizona State University
9:50 Discussion with the Summit Planning Committee and the Advisory Group for NAS/NAM Initiative on Human Gene Editing
10:30 Break
10:45 Session 2: Gene Editing Technologies
Speakers:
Jennifer Doudna, University of California, Berkeley
George Church, Harvard Medical School
Feng Zhang, Broad Institute of Harvard and Massachusetts Institute of Technology
Jennifer Doudna, University of California, Berkeley
George Church, Harvard Medical School
Feng Zhang, Broad Institute of Harvard and Massachusetts Institute of Technology
11:30 Discussion with the Summit Planning Committee and the Advisory Group for NAS/NAM Initiative on Human Gene Editing
12:00 Lunch
1:00 Session 3: Measuring Off-Target Events, Efficiency, and Utility
Speakers:
Richard Frock, Harvard University
Chad Cowan, Harvard University
Fyodor Urnov, Sangamo Biosciences
Richard Frock, Harvard University
Chad Cowan, Harvard University
Fyodor Urnov, Sangamo Biosciences
1:45 Discussion with the Summit Planning Committee and the Advisory Group for NAS/NAM Initiative on Human Gene Editing
2:15 Break
2:30 Session 4: Overview of Chinese Gene Editing Research and Policy
Speakers:
Duanqing Pei, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences
Qi Zhou, Institute of Zoology, Chinese Academy of Sciences
Duanqing Pei, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences
Qi Zhou, Institute of Zoology, Chinese Academy of Sciences
3:00 Discussion with the Summit Planning Committee and the Advisory Group for NAS/NAM Initiative on Human Gene Editing
3:30 Break
3:45 Session 5: Approaches to Treat, Avoid, and Prevent Genetic Disease
Speakers:
Nancy Wexler, Columbia University
George Daley, Boston Children’s Hospital and Dana-Farber Cancer Institute
Nancy Wexler, Columbia University
George Daley, Boston Children’s Hospital and Dana-Farber Cancer Institute
4:15 Discussion with the Summit Planning Committee and the Advisory Group for NAS/NAM Initiative on Human Gene Editing
4:45 Public Comments
5:30 ADJOURN OPEN SESSION
Webcast: http://nationalacademies.org/gene-editing/webcast-10-5-2015/index.htm
Friday, October 2, 2015
FDA Grants Orphan Drug Designation to REGENXBIO's Gene Therapy for the Treatment of Hurler's Disease
"
FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in
MPS I is a rare neurodegenerative disease caused by deficiency of the a-l-iduronidase (IDUA) gene. Over 1,000 individuals with MPS I are estimated to be born each year worldwide. Symptoms include excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment. RGX-111 uses an AAV9 vector to deliver the IDUA gene to the central nervous system.
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