Bio Blast Pharma Announces Initiation of its Canadian OPMD Clinical Study Center at McGill University, Montreal

Bio Blast Pharma Announces Initiation

of its Canadian OPMD Clinical Study Center at McGill University, Montreal

TEL AVIV, Israel, September 22, 2014 - Bio

Blast Pharma Ltd (NasdaqGM: ORPN), a clinical-stage biotechnology company committed to developing clinically meaningful therapies

for patients with rare and ultra-rare genetic diseases, announced today it has received Health Canada approval to begin enrolling

patients at the Montreal Neurological Institute at McGill University for its HOPEMD Phase 2/3 study investigating Cabaletta in

patients with Occulopharyngeal Muscular Dystrophy (OPMD). Professor Bernard Brais, co-director of the neuromuscular group of the

Montreal Neurological Institute and Hospital, is the principal investigator of the study in Canada.

Study initiation and investigator conferences were conducted

at the Montreal Neurological Institute and Hospital on September 19th. Investigators and key opinion leaders from Europe, Canada,

the United States and Israel participated.

The HOPEMD study is a Phase 2/3 multicenter study testing the

safety, tolerability and efficacy of Cabaletta in patients suffering from OPMD. The study will be conducted across three centers

in Israel, Canada and the US where the main clusters of patients reside. The center in Israel is already enrolling patients and

published initial positive safety results last week.

"We are honored to be working with the world's leading

researchers of OPMD to find a treatment for an incurable disease that affects the lives of thousands of patients worldwide,"

stated Dr. Dalia Megiddo, Chief Executive Officer of Bio Blast Pharma. "Initiation of the Canadian center of the HOPEMD study

is an important milestone in our clinical development program for OPMD. The Montreal Neurological institute, as well as our Canadian

collaborators, Professor Guy Rouleau, Director of the Montreal Neurological Institute, Professor Bernard Brais, co-director of

the neuromuscular group of the Montreal Neurological Institute, and Professor Emeritus Jean Pierre Bouchard from Universit

Laval, Quebec, have been at the forefront of OPMD research for many years. We are thrilled and appreciative of their active interest

and participation in our program."

About Montreal Neurological Institute and Hospital

The Montreal Neurological Institute and Hospital is an academic

medical Centre dedicated to neuroscience research, training and clinical care. The institute is part of McGill University and the

hospital is one of the six teaching hospitals of the McGill University Health Centre.

Cabaletta is chemical chaperone that protects against pathological

processes in cells. It has been shown to reduce pathological aggregation of proteins within cells in several diseases associated

with abnormal cellular-protein aggregation as well as acting as an autophagy enhancer. Cabaletta has been documented as demonstrating

significant efficacy in preclinical animal models of OPMD and other PolyA/PolyQ diseases.

OPMD is an inherited myopathy characterized by dysphagia (difficulty

in swallowing) and the loss of muscular strength and weakness in multiple parts of the body. As the dysphagia becomes more severe,

patients become malnourished, lose significant weight, become dehydrated and suffer from repeated incidents of aspiration pneumonia.

These last two are often the cause of death. The disease is caused by genetic mutation responsible for the creation of a mutant

unstable protein (PABPN1) that aggregates within patient's cell.

About Bio Blast Pharma

Bio Blast Pharma is a publicly traded, clinical-stage biotechnology

company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases.

Founded in 2012, the company is rapidly building a diverse portfolio

of product candidates with the potential to address unmet medical needs for incurable diseases.

The Bio Blast platforms are based on deep understanding of the

disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology.

Forward-Looking Statements

This press release may contain forward-looking statements

within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other

Federal securities laws. For example, we are using forward-looking statements when we discuss our HOPMED study, when we discuss

our efforts to develop a treatment for OPMD, or that our platforms potentially offer solutions for several genetic diseases. Because

such statements deal with future events and are based on Bio Blast Pharma Ltd.'s current expectations, they are subject to various

risks and uncertainties and actual results, performance or achievements of Bio Blast Pharma could differ materially from those

described in or implied by the statements in this press release, including those discussed under the heading "Risk Factors"

in Bio Blast Pharma's registration statement on Form F-1 filed with the Securities and Exchange Commission ("SEC") and

in any subsequent filings with the SEC. In addition, historic results of scientific research and clinical and preclinical trials

do not guarantee that the conclusions of future research or trials would not suggest different conclusions or that historic results

referred to in this press release would be interpreted differently in light of additional research and clinical and preclinical

trials results. Except as otherwise required by law, Bio Blast Pharma disclaims any intention or obligation to update or revise

any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or

circumstances or otherwise.