BioMarin, a leading American biotechnology company (BMRN-NASDAQ), has chosen Sheba Medical Center in Ramat Gan, Israel, to conduct research and a clinical trial of a ground-breaking gene therapy product that has shown promise as the “magic bullet” cure for hemophilia A.









BioMarin, a leading American biotechnology company (BMRN-NASDAQ), has chosen Sheba Medical Center in Ramat Gan, Israel, to conduct research and a clinical trial of a ground-breaking gene therapy product that has shown promise as the “magic bullet” cure for hemophilia A, the dreaded blood disease. Ground-breaking gene single therapy treatments use viruses to implant new genes into patients’ cells so that a single dose might be enough to arrest or cure the disease.

As a result of the initial trial conducted by BioMarin on nine patients, they all stopped using clotting factor drugs to treat their hemophilia, and their annualized bleeding rate fell about 16 episodes a year to just one! “Their bleeding rates collapsed to zero or nearly zero and we’ve improved their quality of life beyond recognition,” said one of the doctors associated with the initial trial. 







Professor Gili Kenet

Professor Gili Kenet

“Sheba Medical Center was chosen to take part in the next phase of international trials and will start enrolling patients (pending Ministry of Health approval, of course) in 2018 due to our excellent record conducting multicenter international therapeutic trials and basic research in patients with hemophilia and severe coagulation disorders,” Professor Gili Kenet, Director of the National Hemophilia Center at Sheba Medical Center, revealed. “So far, nine patients were cured and followed for over one year, after one infusion of gene therapy product (normal “engineered” gene of coagulation factor, delivered via AAV-(Adeno associated virus) vector to patients’ blood and to their liver, inducing production of the normal factor in hepatocytes). After the initial research results, people in the medical community are beginning to speak about curing hemophilia after a one-time treatment with this new drug.”

The National Hemophilia Center at Sheba Medical Center is also a leading internationally-known Hemophilia Training Center (HTC). The center currently treats over 700 hemophiliacs a year with over 50 patients currently participating in clinical studies. These studies are led by Professor Kenet and her team of physicians and experienced study coordinators, who recently underwent 2 FDA inspections and were graded as excellent.

Professor Kenet is a world-renowned expert on hemophilia, thrombosis and hemostasis and is the author of over 190 medical manuscripts, many of which have been published in noted journals such as the New England Journal of Medicine and Lancet (UK). She is also an active member of the American Society of Hematology, World Federation of Hemophilia and Israeli Society of Pediatric Hematology and Oncology and Israel Thrombosis Society. She has chaired the scientific subcommittee (SSC) of ISTH (Int’l Society of Thrombosis and Hemostasis for pediatric and perinatal hemostasis) and currently co-chairs the SSC of FVIII, FIX (genes encoded for blood coagulation), and rare bleeding disorders.

She added, “Indeed these are exciting times, as there are options to cure hemophiliac patients. Thus, we are hoping that results of this trial at Sheba will be good enough that this is will be the therapy of choice that will able to stop this dreaded disease once and for all.”

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