Gene therapy found safe in trial of Pompe disease treatment
April 26, 2018 – 12:00 am
The first human trial of gene therapy to treat respiratory problems in early-onset Pompe disease of children was found safe, according to health researchers in Florida.
University of Florida Health scientists conducted a successful trial of nine participants who had the incurable disease, which damages muscle and nerve cells throughout the body. Results of the treatment, which utilizes an adeno-associated virus, were published in the journal Human Gene Therapy Clinical Development.
DNA Vaccine Protects Immune-privileged Tissues
January 12, 2018 – 2:10 pm
When Zika virus made headlines in 2015, it left stark images in public memory of children born with birth defects such as microcephaly. As of 2016, Zika virus is estimated to have infected 1 million people worldwide.
In addition to its dramatic impact on brain development, Gary Kobinger of the University of Quebec and his colleagues discovered that the virus could enter and damage testes in mice, reducing sperm count and motility. Whether this also happens in humans is still unknown, but it could have serious consequences. “People with subclinical infections may not seek treatment, but that doesn’t mean there’s no damage,” Kobinger said. “In a worst case scenario, we might see fertility rates plummeting in 5–10 years.”
Gene Therapy Via Skin Could Treat Many Diseases, Even Obesity
August 9, 2017 – 10:58 am
A research team based at the University of Chicago has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases.
In the August 3, 2017 issue of the journal Cell Stem Cell, the researchers provide "proof-of-concept." They describe a new form of gene-therapy -- administered through skin transplants -- to treat two related and extremely common human ailments: type-2 diabetes and obesity.
"We resolved some technical hurdles and designed a mouse-to-mouse skin transplantation model in animals with intact immune systems," said study author Xiaoyang Wu, PhD, assistant professor in the Ben May Department for Cancer Research at the University of Chicago. "We think this platform has the potential to lead to safe and durable gene therapy, in mice and we hope, someday, in humans, using selected and modified cells from skin."
DNA-Based Compound Shuts Down Seasonal Flu in Preclinical Study
August 4, 2017 – 10:53 am
Inovio Pharmaceuticals Inc. (INO:NASDAQ) announced its "DNA-based monoclonal antibody [dMAb] product for flu produced broadly cross-reactive antibodies that provided complete protection from a lethal challenge with multiple viruses from both influenza A and B types in a preclinical study," in a July 6 press release.
The results of the preclinical study were published in the journal npj Vaccines.
Inovio's dMAb products "provide cross-strain protection and may offer prevention against unexpected changes of seasonal influenza strains," H. C. Wainwright analyst Raghuram (Ram) Selvaraju stated in a July 7 research report. "We note that these data corroborate previous positive findings on dMAb products designed for HIV, dengue, and Chikungunya, and further validate the dMAb platform that aims to deliver DNA sequences that encode and rapidly generate therapeutic monoclonal antibodies directly in the recipients."
One Step Closer to a DNA Vaccine Against Dengue Virus
July 31, 2017 – 2:33 pm
In a new study, researchers inoculated mice with a new DNA vaccine candidate (pVAX1-D1ME) in order to evaluate its efficiency. They found that the vaccine candidate was able to induce persistent humoral and cellular immune responses and provided efficient protection against lethal challenge from one of the four serotypes of dengue virus (DV1). They also evaluated the immunoprotective potential of a combined (bivalent) DNA vaccine, which was found to generate a balanced immunogenic response to two serotypes of dengue virus (DV1 & DV2). These results are encouraging for the future development of a tetravalent vaccine that could provide efficient protection against all four serotypes of the virus.
Dogs with Duchenne Treated with Gene Therapy
July 26, 2017 – 12:09 pm
Like humans, some golden retrievers develop Duchenne muscular dystrophy (DMD), a hereditary muscle wasting condition that begins early in life. Using gene therapy, scientists were able to restore muscle function in dogs with the disease, according to a study published today (July 25) in Nature Communications.
Researchers injected microdystrophin, a shortened version of the dystrophin gene that individuals with DMD lack, into 12 dogs with the disease. The treatment led to improved muscle function in those animals for more than two years.
‘Major Advance’: Leukemia Treatment Could be First U.S. Gene Therapy
July 21, 2017 – 9:51 am
A treatment for a common childhood blood cancer could become the first gene therapy available in the U.S.
A Food and Drug Administration advisory panel voted 10-0 on Wednesday in favor of the leukemia treatment developed by the University of Pennsylvania and Novartis Corp. The FDA usually follows recommendations from its expert panels but isn’t obligated to do so.
The therapy could be the first of a wave of treatments custom-made to target a patient’s cancer. Called CAR-T, this type of therapy involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells that can zero in on and destroy cancer cells and injecting them back into the patient.
“This is a major advance,” said panel member Dr. Malcolm A. Smith of the National Cancer Institute. He said the treatment is “ushering in a new era.”
New Partnership to Advance Novel Airway-Delivered Gene Therapy for Treating Pulmonary Hypertension
July 13, 2017 – 11:04 am
Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure. If the therapy succeeds in human clinical trials, it will provide patients for the first time with a way to reverse the damage caused by PH.
This gene therapy technique comes from the research of Roger J. Hajjar, MD, Professor of Medicine and Director of the Cardiovascular Research Center at the Icahn School of Medicine at Mount Sinai, and has been proven effective in rodent and pig animal models. PH is a deadly disease that disproportionately affects young adults and women; 58 percent of cases are found in young adults and 72 percent are women. There is currently no effective cure for PH, and about 50 percent of people who are diagnosed will die from the disease within five years.
First Gene Therapy — ‘a True Living Drug’ — on the Cusp of FDA Approval
July 11, 2017 – 11:14 am
PHILADELPHIA — When doctors saw the report on Bill Ludwig’s bone-marrow biopsy, they thought it was a mistake and ordered the test repeated. But the results came back the same: His lethal leukemia had been wiped out by an experimental treatment never used in humans.
“We were hoping for a little improvement,” remembers the 72-year-old retired New Jersey corrections officer, who had battled the disease for a decade. He and his oncologist both broke down when she delivered the good news in 2010. “Nobody was hoping for zero cancer.”
The pioneering therapy with Ludwig and a few other adults at the University of Pennsylvania hospital paved the way for clinical trials with children. Six-year-old Emily Whitehead, who was near death, became the first pediatric recipient in 2012. Like Ludwig, she remains cancer-free.
Such results are why the treatment is on track to become the first gene therapy approved by the Food and Drug Administration. An FDA advisory committee will decide Wednesday whether to recommend approval of the approach, which uses patients’ own genetically altered immune cells to fight blood cancers.
Gene Therapy for Juvenile Batten Disease Gets Orphan Drug Designation
July 5, 2017 – 9:23 am
The FDA has granted Abeona Therapeutics Inc. Orphan Drug Designation (ODD) for ABO-201 (AAV-CLN3), its gene therapy program for juvenile Batten disease, (juvenile neuronal ceroid lipofuscinosis, JNCL). The AAV-based gene therapy is being designed as a single intravenous infusion to treat children with the currently fatal rare disease. Simply put, the gene therapy is being developed not to treat these children, but to cure them. This is the 4th gene therapy the company has in development that has obtained an Orphan Drug Designation. The other 3 are being developed to treat recessive dystrophic epidermolysis bullosa and both sanfilippo syndrome type A and type B. The company also has a variety of other gene therapies in development for other rare diseases.
