Novo Nordisk Obtains Worldwide Rights to EpiDestiny's Sickle
Cell Disease Therapy - Sickle Cell Anemia News
Not exact matches
«The better we understand these
cell types and how they affect
disease, the better we will be able to develop new
therapies to treat and cure
disease.»
I admire SQZ Biotech's unique platform — they're on the forefront of something huge that has the potential to revolutionize
disease research and
cell therapy.
Venter reiterated this sentiment: «Using the combined power of our core areas of expertise — genomics, informatics, and stem
cell therapies, we are tackling one of the greatest medical / scientific and societal challenges — aging and aging related
diseases,» he said in a statement.
About Nohla Therapeutics Nohla Therapeutics is a leading developer of off - the shelf
cell therapies for the treatment of cancer and other critical
diseases.
His research has spanned hematopoiesis, gene
therapy, stem
cell biology, genomics and cancer, consistently focusing on bringing the very latest research advances to patients with heretofore incurable
diseases.
Instead of getting a better, updated
therapy for a
disease every decade or so, we might begin to see second - generation
cell therapies in a few years.
«Using the combined power of our core areas of expertise — genomics, informatics, and stem
cell therapies, we are tackling one of the greatest medical / scientific and societal challenges — aging and aging related
diseases,» said Dr. Venter.
Using advances in genomic sequencing, the human microbiome, proteomics, informatics, computing, and
cell therapy technologies, HLI is building the world's most comprehensive database of human genotypes and phenotypes as a basis for a variety of commercialization opportunities to help solve aging related
disease and human biological decline.
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.
Cambridge, MA — March 30, 2017 — Aura Biosciences, a biotechnology company developing a new class of
therapies to target and selectively destroy cancer
cells using viral nanoparticle conjugates, announced today that it has enrolled and dosed the first patient in its Phase 1b clinical trial of light - activated AU - 011, an investigational, first - in - class targeted
therapy in development for the treatment of ocular melanoma, a rare and life - threatening
disease.
What Stephen Hawking Missed: Small Biotechs Developing Promising
Cell Therapies for Devastating Disease Source: Streetwise Reports (5/2/18) In the second of a two - part series exploring the disruptive cell therapy space, Maxim Group analyst Jason McCarthy takes a look at small - cap companies targeting big - ticket indications and their potential to drive blockbuster value for both patients and invest
Cell Therapies for Devastating
Disease Source: Streetwise Reports (5/2/18) In the second of a two - part series exploring the disruptive
cell therapy space, Maxim Group analyst Jason McCarthy takes a look at small - cap companies targeting big - ticket indications and their potential to drive blockbuster value for both patients and invest
cell therapy space, Maxim Group analyst Jason McCarthy takes a look at small - cap companies targeting big - ticket indications and their potential to drive blockbuster value for both patients and investors.
For instance, specific anti-T-cell
therapies don't work at all for rheumatoid arthritis (RA), and Edwards doesn't think T -
cells explained how the
disease persisted.
«In effort to treat rare blinding
disease, researchers turn stem
cells into blood vessels: Patients around the world contribute skin samples to test potential new
therapy.»
They isolated blood
cells from HIV - positive patients on antiretroviral
therapy and at different stages of
disease progression, as well as
cells from non-infected individuals.
«Step toward gene
therapy for sickle
cell disease.»
Increasingly, though, better techniques are raising hopes for practical
therapies that can permanently cure genetic
diseases like sickle
cell.
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.
«Cultural revolution in the study of the gut microbiome: Human gut - on - a-chip technology used to co-culture gut microbiome, human intestinal
cells could lead to new
therapies for inflammatory bowel
diseases.»
A team of researchers at the Stanford University School of Medicine has used a gene - editing tool known as CRISPR to repair the gene that causes sickle
cell disease in human stem
cells, which they say is a key step toward developing a gene
therapy for the disorder.
«This research has broad impact, because by deepening our understanding of
cell reprogramming we have the potential to improve
disease modeling and the generation of better sources of patient - specific specialized
cells suitable for replacement
therapy,» said Plath.
However, there is still limited data on the short and long - term effects of administering stem
cell therapy in chronic respiratory
disease.
We believe that they will also lead to the development of a whole new range of
therapies for neurodegenerative
diseases of the central nervous system,» explains corresponding author of the study Jihwan Song, professor and director of Neural Regeneration and
Therapy Group at the CHA Stem
Cell Institute of CHA University.
A new study has found that stem
cell therapy can reduce lung inflammation in an animal model of chronic obstructive pulmonary
disease (COPD) and cystic fibrosis.
Tissue engineering provides a more practical means for researchers to study
cell behavior, such as cancer
cell resistance to
therapy, and test new drugs or combinations of drugs to treat many
diseases.
Rashid's ultimate hope is that one day scientsts will be able to correct the
diseased cells in the lab and transplant them back into the patient, but he cautions that
cell - based
therapies won't be available anytime soon.
Researchers at the University of Louisville have discovered a mechanism involved in skeletal muscle repair that may enable clinicians to boost the effectiveness of adult stem
cell therapies for
diseases such as muscular dystrophy.
Realistic stem
cell therapies to replace
diseased or damaged tissue may still be years away, but researchers have uncovered a promising new use for these undifferentiated
cells: they can be programmed to become patient - specific laboratory models of inherited liver
disease.
But scientists are making progress in refining these
therapies, and the first ever trial of fetal stem
cells injected directly into the brain is currently under way in children with Batten
disease, a rare and fatal illness of the nervous system.
«Our study reveals a new mechanism that could be harnessed for biological
therapies for lupus and other autoimmune
diseases, where the immune system mistakenly targets the body's own
cells,» says senior study author Boris Reizis, PhD, professor of Pathology and Medicine at NYU Langone.
«Due to the inhibitory function of Treg
cells, people have been trying to use these
cells for
therapy in human autoimmune
diseases or transplantation,» explains professor Yun Cai Liu, Ph.D., who led the current study.
New products and developments, such as new drugs for cancer, fresh
therapies for rare
diseases, progress in medications for HIV / AIDS, and advances in stem
cell research had the greatest positive impact.
Eye
diseases — such as age - related macular degeneration, as well as a genetic condition called Stargardt's macular dystrophy that afflicts young people — are considered excellent candidates for stem
cell therapy because the eye is an immune - privileged site, meaning transplanted
cells are not as likely to be rejected as foreign compared with transplants elsewhere.
«Stem
cell gene
therapy could be key to treating Duchenne muscular dystrophy: Approach developed at UCLA holds promise for 60 percent of patients with the deadly
disease.»
This accomplishment opens the door for
disease modeling and drug screening and brings personalized
cell therapy a step closer for patients with diabetes.
Diabetes researchers are considering various replacements for insulin injections: Transplanting new pancreatic islet
cells that make insulin, coaxing the patient's own islets to regenerate, or treating diabetics early in the
disease with immune - suppressing
therapies to prevent their body from destroying the rest of their pancreatic islets.
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.
Additionally, work in a mouse model revealed similar
cells, indicating that the progenitors are conserved from mouse to human, and therefore, they must be «important
cells with promising potential for
cell therapy in treating liver
disease,» explained Dr. Gouon - Evans.
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.
Researchers can create iPSCs from a patient's blood or skin
cells, and use these patient - specific
cells to study
diseases or even create new tissues that could be transplanted back into the patient as
therapy.
«It's fantastic news that they are going into the clinic with a
cell therapy for eye
disease,» says Pete Coffey of University College London, and head of a team developing tiny «patches» of RPEs for treating age - related macular degeneration.
«If you want to develop a
therapy for autoimmune
diseases, the idea is, How do we get Xist to the inactive X chromosome and keep it there so we maintain dosage compensation in these B
cells.»
A specific genetic marker influences response to
therapy in the early stages of RA; a link thought to be due to the gene activating a
cell - signalling protein involved in the inflammatory
disease process.
The technology and its resulting research also may become of great value in medical laboratories and hospitals in improving the detection and diagnosis of
diseased cells and in evaluating a patient's response to
therapy.
In addition to providing insights regarding selection of treatment, this blood test may also prove helpful in assessing the presence of residual
disease, recurrence, or relapse and detection of
therapy - resistant cancer
cells more quickly than conventional tissue - based methods.
«Our findings could have a significant impact on the treatment of autoimmune
diseases, as well as on stem
cell and immuno - oncology
therapies,» said Gladstone Senior Investigator Sheng Ding, PhD, who is also a professor of pharmaceutical chemistry at the University of California, San Francisco.
«By reprogramming human
cells, scientists have created new opportunities to study
diseases and develop methods for diagnosis and
therapy,» the Nobel judges wrote.
Combining radiation
therapy with chemotherapy for patients with limited metastatic non-small
cell lung cancer (NSCLC) may curb
disease progression dramatically when compared to NSCLC patients who only receive chemotherapy, according to a new randomized phase II clinical trial reported today at the 59th Annual Meeting of the American Society for Radiation Oncology (ASTRO).
«Investigators create complex kidney structures from human stem
cells derived from adults: New technique offers model for studying
disease, progress toward
cell therapy.»