Sangamo Biosciences, Inc. ($6.42 BUY)
Assuming with Buy, Risk/Reward Skewed to Upside
Gena Wang, PhD, CFA
Key Takeaway
With stock trading at ~$300M EV, we believe the ZFN gene-editing technology platform
with initial clinical proof-of-concept established, should support the current valuation for SGMO, while any further clinical validation would provide substantial upside.
Price target $12 to $14
Edward Rebar, Sangamo Biosciences presented "Genome Editing with Engineered Zinc Finger Nucleases" at the International Society for Experimental Hematology annual meeting on September 18th 2015. For the record Phil Gregory was the original invited speaker. The beat goes on.
http://en.hdbuzz.net/204
Today brings news that the first Huntington’s Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene. These brave volunteers are the first HD patients to ever be treated with drugs designed to attack HD at its root cause, a treatment approach with huge potential. What about this news has us so excited?
Gene Silencing
Many HD researchers, including the editors of HDBuzz, believe that a treatment approach called gene silencing is the most exciting thing happening in HD research right now. To understand why, we need to cover a little bit of HD basics. Gene silencing drugs interfere with the way that genes are used to make damaging proteins. ASOs work by targeting specific messenger molecules for destruction, which has the effect of reducing levels of the damaging protein. Every HD patient has inherited a mutated version of a gene that we sometimes call the HD gene. In scientific circles, the gene is actually called Huntingtin, and abbreviated HTT, but that can be a bit confusing.
Everyone on earth has two copies of the HD gene, one they inherited from mom and one from dad. In every HD patient, one of these copies has a sort of genetic stutter, a repetitive bit of code near one end of the gene that tweaks the way this gene does its job. This turns out to be bad news - Huntington’s Disease is the consequence of this stutter lengthening beyond a critical threshold.
We still don’t understand a lot about HD. What the HD gene normally does, why does it have this repetitive stretch of DNA, and what makes it stretch out are questions that we still don’t have scientific consensus on.
But what’s clear beyond a shadow of a doubt is that every single HD patient has the same type of mutation - a lengthening of the repetitive stretch of DNA in the HD gene. In the code scientists use to describe genetic sequences, the stuttering letters are abbreviated “C-A-G”, which is why you might have heard of people talking about “C-A-G repeats”.
This genetic certainty is terrifying for family members - if your mom or dad has HD you have an exactly 50% chance of inheriting this awful mutation. But there’s a flip side to this bad news, which is that this certainty gives us a really good target for trying to attack HD. Since every single HD patient has a mutant HD gene, why don’t we just try to get rid of the mutant gene itself?
In previous generations, this would have been like asking someone to reach up into the sky and pluck out your favorite star, but we live in amazing times. It turns out that this kind of thing might actually be possible now because over the last 20-30 years, techniques have been developed by scientists that let us pretty much switch off a given gene at will.
At root, scientists are a curious bunch. As they’ve poked and prodded how cells achieve particular tasks, they’ve uncovered a wide range of ways to shut off certain genes. You might hear about “antisense oligonucleotides (ASOs)”, or “zinc finger nucleases (ZFNs)” or even “transcription activator-like effector nucleases (TALENs)”. The idea behind this zoo of approaches is the same: trick cells into shutting off the HD gene and only the HD gene.
“This genetic certainty is terrifying for family members - if your mom or dad has HD you have an exactly 50% chance of inheriting this awful mutation. But there’s a flip side to this bad news, which is that this certainty gives us a really good target for trying to attack HD. ”
Isis and ASOs
A wide range of scientists are applying all of the above approaches (and more!) to the problem of shutting off the HD gene. The most advanced program uses a type of drugs called “antisense oligonucleotides”, or “ASOs”. Basically, ASO drugs are short heavily modified bits of DNA that instruct a cell to destroy a particular gene.
Compared to some of the other technologies that turn on or off genes, ASOs have been around for a long time. The company with the most advanced HD gene silencing program is called Isis Pharmaceuticals, which was founded in 1989. For those of us that remember the fall of the Berlin Wall, 1989 might not seem like so long ago, but in the biotechnology world that’s a long run.
The benefit of all this experience is that Isis has a long history in applying ASOs to the problem of human diseases. Versions of their various ASO drugs have been tested in thousands of humans with a wide range of health problems. They’ve also successfully had drugs approved by regulatory agencies like the FDA, so they’ve got a realistic idea of what it takes to get a drug to people.
Luckily for us, Isis has developed an ASO drug they call “ISIS-HTTRx” which targets the HD gene for destruction. Animals treated with the mouse version of this drug show remarkable and important improvements in HD-like symptoms, which has a lot of scientists very excited.
The success of HD silencing drugs in mice is neat science, but actually running a trial for HD patients is a big and expensive problem. Isis understands that they need partners with deep pockets and even more clinical trial experience to get drugs to patients as quickly as possible. For this reason, they’ve partnered with pharmaceutical giant Roche to test ISIS-HTTRx as quickly and competently as possible.
The first HTTRx study
Two companies - Isis Pharmaceuticals and Roche Pharma - are working hard to bring gene silencing drugs to HD patients. As we recently described at HDBuzz (http://en.hdbuzz.net/203), getting drugs approved is a long and complicated process. The first step along the way is what’s called a Phase 1 trial. Any phase 1 study has a single, essential, goal: to make sure that an experimental drug is safe in people. Not mice, not monkeys, not rats, but people.
Often phase 1 studies are conducted in healthy volunteers, but in this case the phase 1 study of ISIS-HTTRx is being carried out in 36 HD patients in Canada, the United Kingdom and Germany. This may seem like a small number of patients, but remember the goal of this study is to establish safety, so what we want is to treat a small number of volunteers to look for any problems before we test the drug in a larger group.
This is especially true for a drug like ISIS-HTTRx which must be delivered directly into the brain. After a lot of experimenting, Isis developed a plan to deliver ASOs directly into the fluid that bathes the brain, the cerebrospinal fluid (or CSF). Because this fluid circulates throughout the brain, delivering a small amount of drug at the base of the spine results in the drug getting carried throughout the entire brain.
A lot of homework has been done in both animals and humans to figure out this delivery trick, but of course anytime we deliver an experimental drug to someone’s brain we have to exercise extreme caution. That is why a relatively small number of patients are enrolled in this first phase 1 study of ISIS-HTTRx.
This question of the spread of the drug within the brain raises an important concern. The data collected by Isis so far suggests that the drug gets most everywhere in the brain, but not very much into part of brain called the striatum.
This is too bad, because the striatum is the most damaged part of the brain in HD. The hope is that fixing other parts of the brain with an ASO may prove a big benefit for the striatum -figuring out if this is true is one of the goals of these studies.
What’s happened?
“Luckily for us, Isis has developed a particular ASO drug they call “ISIS-HTTRx” which targets the HD gene for destruction. ”
So what’s the big news? Simple - Isis has just announced that the first few patients have been successfully dosed with ISIS-HTTRx. Professor Sarah Tabrizi, global chief clinical investigator of the study at University College London, has said: “I’m thrilled that this antisense drug has now been safely administered to the first patients. Families ravaged by Huntington’s disease have been waiting for this milestone for decades. I look forward to ensuring the smooth running of this first trial and hopefully seeing ISIS-HTTRx through to efficacy testing and licensing”.
This means that the trial is underway, and that the first volunteers have been treated without any immediate complications. The next year or so will be a period of intense study of these trial volunteers to make sure that they don’t have unexpected complications from the treatment. They’ll also be examined for a range of measures of whether or not the drug is working, which will provide critical information for planning future HD gene silencing studies.
Where’s this going?
This announcement represents a huge milestone, but it’s only one step on the path towards developing a gene silencing approach to HD. If this phase 1 study is successful and the drug is proven safe, another trial will be required to prove that the drug has an impact on HD symptoms. And also remember that these trials are experiments that we don’t know the outcome of - it’s possible the drug will be safe, but not have enough impact in the brain to significantly impact HD symptoms.
This sounds daunting, but patients and families can be comforted by the knowledge that planning for this next trial is already underway. Everyone involved in this project wants safe and effective drugs for HD in the clinic as soon as possible.
Today’s announcement represents a remarkable step the long fight against HD. We feel great excitement, as well as a deep sense of gratitude to the researchers and participants in this pivotal trial. Stay tuned to HDBuzz for more updates as these trials progress.
Have you been pining for a "teacup" pig but worried that the supposedly petite porcine pet might grow as big as your bathtub?
A Chinese biotech firm says it now has the answer: a genetically modified swine that tops out around 33 pounds.
BGI, a company based in the southern city of Shenzhen that is known for its work sequencing human, plant and animal DNA, recently announced that it intends to start selling $1,600 miniature pigs that it initially created as laboratory models for studying human ailments.
The pigs created a splash late last month when BGI showed them at the Shenzhen International Biotech Leaders Summit. The pint-size porkers were created through a process known as gene editing. Rather than introduce another organism's DNA into the pigs, scientists "edit" the swine's own genetic material, disabling a copy of the growth hormone receptor gene so that cells don't get a signal to grow.
Read the entire article here:
http://www.latimes.com/world/asia/la-fg-china-micropigs-20151005-story.html
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 19, 2015-- bluebird bio, Inc. (Nasdaq: BLUE) announced that Marina Cavazzana, M.D., Ph.D., professor of hematology and director of the Department of Biotherapy at Hospital Necker, University Paris Descartes and lead investigator for the HGB-205 clinical study, presented a review of the clinical study experience with lentiviral-based gene therapies for beta-thalassemia at the 10th Annual Cooley’s Anemia Foundation Symposium in Chicago today.
Professor Cavazzana summarized results from HGB-205, an ongoing clinical study using LentiGlobin® BB305 for the treatment of beta-thalassemia major and severe sickle cell disease, which were presented earlier this year at the Annual Congress of the European Hematology Association. She also provided an update on subject 1003, a patient with beta-thalassemia major treated by Professor Cavazzana in 2007 in the LG001 study using the first-generation HPV569 lentiviral vector. Following approximately seven years of transfusion independence, subject 1003 has recently required two blood transfusions after experiencing clinical symptoms of anemia. Importantly, both the expression of HbAT87Q and the vector copy number in peripheral blood leukocytes, a measure of the persistence of the gene therapy, have remained largely unchanged. The safety profile for both vectors is consistent with autologous transplantation, with no gene-therapy related serious adverse events.
Development of bluebird bio’s approach to treating hemoglobinopathies was guided by the data collected from the LG001 study, a pioneering clinical study that served to highlight both the potential for gene therapy as an intervention in beta-thalassemia as well as the need for an improved lentiviral vector. To this end, Professor Cavazzana also discussed the advances in the design of lentiviral vectors for hemoglobinopathy gene therapies -- specifically the evolution from the first-generation HPV569 lentiviral vector to bluebird bio’s current lentiviral vector, BB305, which results in substantially improved vector copy number and HbAT87Q expression.
“The early clinical experience with HPV569 represented a crucial proof of concept for the potential of gene therapy to bring life-changing treatment to patients in need,” said David Davidson, M.D., chief medical officer, bluebird bio. “The data from the LG001 study were invaluable to our efforts over the last five years to optimize our gene therapy approach with improvements to the potency, robustness and manufacturing for the next-generation lentiviral vector, BB305. LentiGlobin BB305 drug product is being evaluated in three ongoing clinical trials for the treatment of patients with beta-thalassemia major and severe sickle cell disease, and three abstracts related to these ongoing clinical trials have been accepted for presentation at this year’s American Society of Hematology’s Annual Meeting in December.”
We are excited to launch another high energy, action filled, synthetic biology conference this year in San Francisco. At SynBioBeta SF 2015, you’ll meet attendees from around the globe, learn best practices, and hear from industry thought leaders, investors, and entrepreneurs about the ever changing landscape of synthetic biology.
Join us for conversations on sector-specific progress ranging from research-enabling tools to low-cost DNA synthesis, scale-up of industrial biotech platforms and the growing importance of the organism industry. As an added bonus, we have extended the conference to two full days this year, which means more networking opportunities and additional chances to connect with the growing community. This year in addition to having 11 thought enticing sessions and over 80+ industry leading speakers, we will be hosting two amazing new events and workshops this year: Blue Sky Bio Competition – Where startups, industry, and academics can compete to win over $500k in prizes. During the event, special guest and Angel Investor, Esther Dyson will introduce the top three contenders on stage.
1st Annual Engineering Biology Awards – This awards ceremony has been developed to give special recognition to individuals that are making great strides to shape the synthetic biology industry. This year we are honored to have, NASA Astronaut, Catherine Coleman, hosting the awards. Workshops – This year we are delighted to announce that workshops will be available to attend during the event. These featured workshops will be taking place during the session breaks and are free, but you must register ahead of time to attend them as seating is limited.
From his days as a troubled teenager on the gritty South Side of Chicago, Patrick Girondi has never shirked a fight.
Even on his way to making a fortune as a commodities trader, he said, he was fired from one job for “socking someone” on the trading floor.
Now Mr. Girondi, a high school dropout, is in a fight of a different kind — against the august Memorial Sloan Kettering Cancer Center over the rarefied field of gene therapy. It is a fight, he said, to save his son.
Mr. Girondi accuses the cancer center of dawdling on developing a gene therapy that could potentially cure his son of an inherited blood disease called beta thalassemia, or Cooley’s anemia. The disease often kills people by their late 20s — an age his son will reach in a few years.
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.
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
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.
Fyodor Urnov (L.) in discussion at CHSL (photo courtesy of Cold Spring Harbor Laboratories)
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!
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.
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.
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)
Lecture Room National Academy of Sciences Building 2101 Constitution Avenue, NW Washington, D.C. 20418
October 5, 2015 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
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
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
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
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
4:15 Discussion with the Summit Planning Committee and the Advisory Group for NAS/NAM Initiative on Human Gene Editing
ROCKVILLE, Md., Oct. 1, 2015 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (REGENXBIO) (Nasdaq:RGNX), a leading biotechnology company in gene therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to REGENXBIO's investigational gene therapy product candidate RGX-111 for the treatment of mucopolysaccharidosis Type I (MPS I).
"REGENXBIO is pleased to have received Orphan Drug Designation from the FDA for RGX-111," said Kenneth T. Mills, President and CEO of REGENXBIO. "MPS I is a severely debilitating disease and patients and their caregivers do not currently have adequate therapeutic options. We remain committed to our vision of developing gene therapies for patients with high unmet medical needs, including MPS I."
FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.
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.
REGENXBIO intends to file an Investigational New Drug Application (IND) with the FDA in the first half of 2016 to support the initiation of a dose-escalation Phase I/II clinical trial of RGX-111 beginning in the first half of 2016. RGX-111 is not approved for sale in the United States or elsewhere
mpnforum.com/gene-editing-stem-cells-for-autologous-transplant/
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.
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.
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.
