ISRAEL 
HIGH-TECH & INVESTMENT REPORT

from the May 2014 issue


Regenerative Medicine

Scientific regulation in Israel is enforced by Judaism's emphasis on the saving of life. The country's relatively liberal approach to stem cell research for therapeutic purposes derives from this tradition, which has positioned Israeli scientists among stem-cell research's pioneers and kept them at the heart of the regenerative medicine map, helped by a government-sponsored research consortium spanning academia and industry. While no stem-cell medication yet exists anywhere, several are on the way from Israeli companies.

The four-year-old Jerusalem start-up Cellcure Neurosciences is starting clinical trials in patients with age-related macular degeneration (AMD), the leading cause of blindness in over-50s in the Western world, which is estimated to affect some 30 million people.

The disease is caused by the dysfunction, degeneration and death of pigment-including retina cells, which lie between the retina's photoreceptors and the nourishing blood vessels at the back of the eye. Cellcure creates healthy retinal pigment cells from human embryonic stem cells, and injects them into the eye to replace the dying cells.

Jerusalem-based Gamida Cell has developed stem cells from umbilical cord-blood to treat blood cancers, autoimmune diseases, metabolic disorders and the hematological disease neutropenia. Its lead product is StemEx, which was given FDA Fast Track Designation in mid-2010. It is now being tested in international Phase III clinical trials as an alternative to bone marrow transplant in patients with advanced blood cancers, who are unable to find a matched donor.

BrainStorm Cell Therapeutics in Petah Tikva is a leading developer of adult stem cell technology and therapy. It has created a stem cell treatment for patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) and Parkinson's disease based on autologous bone-marrow-derived adult stem-cells. In a first clinical trial, conducted at the Hadassah Medical Center, ALS patients will be re-implanted with stem cells taken from their own pelvis.

Drugs derived from living cells
Uplyso, a medicine for Gaucher's disease based on the enzyme taliglucerase alfa, is an example of the ongoing push into biologics - drugs derived from living cells rather than from chemicals. It was developed by Protalix Biotherapeutics, a company that began life in 1994 within Israel's Meytav Technological Incubator, graduating to become an independent publicly held company with a market capitalization of more than $700 million, trading on both the NYSE Amex Exchange and the Tel Aviv Stock Exchange.

It recently sold the rights to this experimental Gaucher's medication for $60 million to the New York-based Pfizer, the world's largest-selling pharmaceutical firm.



Reprinted from the Israel High-Tech & Investment Report May 2014

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