He has authored ≥ 420 publications (h - index 75), is Editor - in Chief of The Journal of Gene Medicine, Editor of Pharmaceutical Research, board member of the German Society for Gene Therapy, committee member of the American Society
of Gene and Cell Therapy (ASGCT), invited guest professor at Utrecht University (1996), Fudan University (Shanghai 2012 - 13), Sichuan University (Chengdu 2014 - 17), and was awarded with the Attocube research award, Phoenix Pharma Science award, and the election into CRS College of Fellows.
American Society
of Gene and Cell Therapy Annual Meeting Speaker: Olivia Kelly, Ph.D., Senior Director, Cell Biology Presentation: Developing a Cell Therapy Combination Product for Diabetes Date / Time: May 16, 8:30 AM EDT Location: Hilton Chicago, Chicago
He is Member of the European Molecular Biology Organization (EMBO), has been President of the European Society
of Gene and Cell Therapy (ESGCT), and has been appointed as expert on the «Human Gene Editing Study» of the US National Academies of Sciences and of Medicine, and on the Italian National Committee for Biosafety, Biotechnology and Life Sciences.
Data from this trial was previously presented at World Advanced Therapies and Regenerative Medicine (May 17 - 19,2017; London, UK) and the 20th Annual Meeting American Society
of Gene and Cell Therapy (ASGCT, May 10 - 13, 2017; DC, USA).
He was awarded a European Research Council Advanced Investigator Grant (ERC) in 2009, the Outstanding Achievement Award from the American Society
of Gene and Cell Therapy (ASGCT) in 2014 and from ESGCT in 2015, an Honorary doctorate from the Vrije University, Brussel, in 2015, the Jimenez Diaz Prize in 2016, and the Beutler Prize from the American Society of Hematology (ASH) in 2017.
Not exact matches
I won't reveal yet who my favorites are, but I will say that these young scientist - founders came up with very creative solutions for preventing infections in some common surgeries, tackling resistance in targeted antibody drugs, improving
gene vectors for
cell therapies, helping the vision - impaired «see» faces
and better read their environments, imaging hard - to - see spots in the lungs
and other organs, improving genetic risk analysis,
and expediting the logistical operations
of hospitals.
US - based pharmaceutical company Gilead Sciences entered the chimeric antigen receptor (CAR) T -
cell therapy business through its acquisition
of Kite Pharma,
and Australian biopharma company CSL Behring acquired US - based Calimmune, a company that develops clinical - stage
gene therapy solutions.
The Chiesi fund will consider a wide range
of opportunities, from traditional small molecules
and biologics, to
gene therapies and cell therapies, to diagnostic
and disease management technologies.
With major clinical successes in areas such as CAR - T,
gene therapy, immune - oncology,
cell therapy and gene editing, many see 2017 as the year that biotech really came
of age.
Risk Versus Reward: The Value
of Cell Therapy for Patients and Investors Source: Streetwise Reports (4/25/18) The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developm
Cell Therapy for Patients and Investors Source: Streetwise Reports (4/25/18) The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage devel
Therapy for Patients
and Investors Source: Streetwise Reports (4/25/18) The
cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developm
cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage devel
therapy space, encompassing disruptive new treatment including stem
cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developm
cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage devel
therapy, immunotherapy
and gene editing, has begun to mature, with a handful
of product approvals
and others in late - stage development.
The
cell therapy space, encompassing disruptive new treatment including stem
cell therapy, immunotherapy
and gene editing, has begun to mature, with a handful
of product approvals
and others in late - stage development.
«The event, the fourth
of its kind, seeks to raise global awareness
and create a forum for collaboration around the wide array
of powerful
and promising
cell therapies,
gene therapies,
and immunotherapies emerging from medical institutions around the world, as well as the impact new technology will have on humanity
and society,» a press release by the Cure Foundation explains (h / t Christian Post).
They include going after the damage to
cells done by free radicals, making use
of hormone
therapy, or caloric restrictions, or vitamin supplements, or, most dramatically, healthy
gene selection through pre-implantation genetic diagnosis
and even repairing the entire human genome.
But as new methods
of genetic modification emerge, both the scientific
and the sports communities are becoming increasingly aware that
gene therapy — the insertion
of genes into an individual's
cells and tissues — will make its way onto the playing field.
ORDINARY
cells from people with a genetic disease can be «fixed» by
gene therapy and then reprogrammed to be stem
cells that will produce a limitless supply
of defect - free
cells.
Researchers such as geneticist Richard King
of the University
of Minnesota
and cell biologist Vitali Alexeev
of Thomas Jefferson University are working on
gene therapies or drugs that would fix albinism - causing mutations.
Then immunotherapy firm Juno Therapeutics shook hands with
gene - editing start - up Editas to create anticancer immune
cell therapies; Vertex Pharmaceuticals
and Crispr Therapeutics, another start - up, inked an agreement that could be valued at $ 2.6 billion; while Regeneron Pharmaceuticals formed a patent licence agreement with ERS Genomics, which holds the rights to the foundational Crispr intellectual property from Emmanuelle Charpentier, one
of the Crispr pioneers.
Because
of this, families
and doctors often shrink from transplant options, particularly when it comes to treating children,
and it will limit the extent to which the breakthroughs in
gene therapy and gene editing will be applied, explained Scadden, who is a practicing hematologist at MGH
and chairman
of Harvard's Department
of Stem
Cell and Regenerative Biology.
The stem
cell gene therapy could be applicable for 60 percent
of people with Duchenne, which affects approximately 1 in 5,000 boys in the U.S.
and is the most common fatal childhood genetic disease.
These include the ability to bring new, innovative products to the market; progress in oncology, such as the approval
of Genentech's drug Avastin for breast cancer
and advances in the use
of gene therapy, despite some setbacks; continuing progress in research on stem
cells; the emergence
of treatments for previously untreated diseases;
and solutions for food
and fuel shortages, such as biocrops
and biofuels.
The breakthrough has resulted from the merger
of interlocking fields —
gene therapy and cell therapy — which are now spawning near - miraculous treatments
and cures.
And because mouse embryo
cells with inactivated copies
of BRCA2 are more sensitive to ionizing radiation than normal
cells are, «it's a reasonable extrapolation» that breast cancers with mutated copies
of the
gene may be especially good candidates for radiation
therapy.
«It was kind
of fun being at a medical school
and known as the weird guy who worked with dogs,» says Modiano, who is now a professor
of comparative oncology at the University
of Minnesota College
of Veterinary Medicine
and the Masonic Cancer Center, where his research focuses on immunology, cancer
cell biology, cancer genetics,
and applications
of gene therapy.
Two
of 10 children treated with
gene therapy for SCID in a French trial develop leukemia, researchers announced in 2002,
and it is discovered that the virus had inserted
genes in several unexpected places around the genome, leading the
cells to become cancerous.
For his part, Collins, who has led NIH since 2009
and been kept on by the Trump administration, pointed to an array
of promising NIH activities, including the development
of new technologies to provide insights into human brain circuitry
and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative)
and the use
of the
gene - editing tool CRISPR - Cas9 to correct mutations
and clear the way to develop
and test a «curative
therapy» for the first molecular disease: sickle
cell disease.
Epigenetic
therapies are thought to work in two ways to fix these errors in cancer
cells — by correcting the «position»
of the
gene switches
and by making the
cell appear as though it's infected by a virus, triggering the immune system.
Priscilla N. Kelly Associate Editor Education: B.Sc., University
of Western Australia; Ph.D., University
of Melbourne Areas
of responsibility: Preclinical development, translational medicine, cancer immunotherapy, drug discovery, clinical trials,
gene and cell therapy E-Mail:
[email protected]
2011: Another success makes headlines: David Porter
and Carl June report that immune
cells modified with
gene therapy had cured two terminal leukemia patients
of their cancer.
In a last - ditch effort to save a dying 7 - year - old boy, scientists have used stem
cells and gene therapy to replace about 80 percent
of his skin.
Muscle biologists Qi Long Lu
and Terence Partridge at the Medical Research Council Clinical Sciences Centre in London, U.K.,
and their colleagues decided to combined the antisense strategy with a chemical often used in
gene therapy because it is known to improve delivery
of DNA into
cells.
However, in the wake
of fatalities from
gene therapy and other technologies, as well as the potential for cancers associated with stem
cell transplants, governments are understandably nervous about safety issues — not to mention the ethical maze
of tinkering with fledgling life.
However, ChABC
gene therapy decreased the presence
of these
cells and increased the presence
of other immune
cells called M2 macrophages that help to reduce inflammation
and enhance tissue repair.
Researchers at the Center for
Cell and Gene Therapy at Baylor College
of Medicine, Texas Children's Hospital
and Houston Methodist have developed an alternative treatment in which virus - specific
cells protect patients against severe, drug - resistant viral infections.
In the recessive deafness model,
gene therapy with TMC1 restored the ability
of sensory hair
cells to respond to sound — producing a measurable electrical current —
and also restored activity in the auditory portion
of the brainstem.
Delivering a single injection
of a scar - busting
gene therapy to the spinal cord
of rats following injury promotes the survival
of nerve
cells and improves hind limb function within weeks, according to a study published April 2 in The Journal
of Neuroscience.
It showed effectiveness against liver cancer in a phase II clinical trial
and will move into a phase III trial later this year, David Kirn, an oncologist
and the company's president
and chief executive officer, said at a recent meeting
of the American Society for
Gene &
Cell Therapy in Washington, D.C..
«Single
gene encourages growth
of intestinal stem
cells, supporting «niche»
cells,
and cancer: Finding in mice could lead to new
therapies for damaged organs, cancer.»
At the Center for
Cell and Gene Therapy at Baylor College
of Medicine in Houston, director Malcolm Brenner, a geneticist, says he has turned «AdV from poacher into gamekeeper.»
The technical advances emerging from
gene therapy have fueled the larger fields
of cell and immune
therapy, where DNA, immune molecules,
and viruses are all elements to be manipulated, in concert or one at a time.
In an effort to expand the number
of cancer
gene mutations that can be specifically targeted with personalized
therapies, researchers at University
of California San Diego School
of Medicine
and Moores Cancer Center looked for combinations
of mutated
genes and drugs that together kill cancer
cells.
Over several years, Dr. Stewart
and colleagues unraveled
genes and signaling pathways that drive multiplication (proliferation)
of beta
cells,
and then confirmed proposed mechanisms with
gene therapy.
The proposed clinical trial, in which researchers would use CRISPR to engineer immune
cells to fight cancer, won approval from the Recombinant DNA Advisory Committee (RAC) at the U.S. National Institutes
of Health, a panel that has traditionally vetted the safety
and ethics
of gene therapy trials funded by the U.S. government
and others.
«Research into basic workings
of immune system points to way
of improving
therapies for cancer: Differences in wiring
of «exhausted»
and effective T
cells indicate possible
gene - editing targets.»
In a step toward accelerating the production
of new
gene therapies, scientists report in ACS Nano that they have developed remote - controlled, needle - like nanospears capable
of piercing membrane walls
and delivering DNA into selected
cells.
Most
gene -
therapy trials use viruses to deliver
genes to a patient's
cells,
and most
of those viruses are retroviruses, which have the ability to neatly splice their
genes —
and the human
gene they're carrying — into a
cell's chromosomes.
Now stem
cells are being combined with
gene and immune
therapies, compounding the pace
of progress.
«Identifying targets essential to
cell survival in tumor suppressor
genes has long been an investigational goal with the aim
of offering cancer - specific vulnerabilities for targeted
therapy,» said Ronald DePinho, M.D., professor
of Cancer Biology, MD Anderson president,
and senior author for the Nature paper.
Scientists used a combination
of stem
cells and gene therapy to repair the damage.
As in the former case, after being treated with telomerase
gene therapy «the telomeres in the peripheral blood in these mice also lengthened
and the number
of blood
cells increased considerably,» write the authors.
A new
gene therapy treatment has restored some sight in a handful
of blind patients suffering from Leber's congenital amaurosis, a syndrome in which, because
of a broken or missing
gene called RPE65, retinal photoreceptor
cells malfunction
and eventually die.