BioBlast Pharma Enhances Leadership Team Appoints Industry Veteran, Robert W. Cook, as Chief Financial Officer New Haven, CT

BioBlast Pharma Enhances Leadership Team

Appoints Industry Veteran, Robert W.

Cook, as Chief Financial Officer

New Haven, CT - January 11, 2016

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

for patients with rare and ultra-rare genetic diseases, announced today that it has appointed Robert (Bob) Cook to the position

of Chief Financial Officer (CFO). Mr. Cook will be a member of BioBlast's Executive Committee, situated out of the Company's

U.S. headquarters in New Haven, CT.

As previously announced, Udi Gilboa, co-founder

of the Company, who until today was the CFO, will now transition to a special advisor role reporting to the Chief Executive Officer

(CEO), and will continue as a member of the Company's board of directors.

"I'm delighted to have Bob

join our executive team given his extensive experience in international banking and as a public company CFO within the pharmaceutical

and medical technology fields," said Colin Foster, President and CEO of BioBlast. "I look forward to working closely

with Bob as we ever more increase our U.S. leadership presence and connection with the U.S. and international investment communities.

I'm grateful for Udi's service since the start of the Company and I look forward to continuing to work with him in

his new role, and on the board of directors."

Mr. Cook brings 25 years of international

experience in senior financial leadership roles in the healthcare finance, pharmaceutical, and medical technology sectors, most

recently serving as Executive Vice President and Chief Financial Officer of Strata Skin Sciences, formerly Mela Sciences, a publicly-held

medical technology company focused on the therapeutic and diagnostic dermatology market. Mr. Cook's prior pharma experience

includes ten years as the CFO of Immune Pharmaceuticals and its predecessor EpiCept Corporation, including one year as Interim

President and CEO of EpiCept in which he completed the reverse merger of EpiCept into Immune. Prior to that he served six years

as CFO at publicly-held Pharmos Corporation. Mr. Cook began his career in financial services at Chase Manhattan, including several

years as a senior executive in its Hong Kong investment bank subsidiary, during which it lead managed $8 billion in private and

public debt financing for governments, public sector corporations and multinational enterprises. Additionally, he spent two years

as a Vice President in the Healthcare Group at General Electric Capital Commercial Finance. Mr. Cook holds a B.S. in Finance, magna

cum laude, from The American University, in Washington, DC.

"I am very much looking forward to

working with Colin and the executive team as we develop the inherent value of BioBlast," said Mr. Cook. "I see great

opportunity in the company's orphan disease platforms and am excited about the potential to significantly grow the company's

value to the benefit of patients and investors."

BioBlast Pharma is a clinical-stage biotechnology

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

is building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases.

The BioBlast platforms are based on deep understandings of the disease-causing biological processes, and potentially offer solutions

for several diseases that share the same biological pathology. For more information please visit the Company's website, www.bioblast-pharma.com,

the content of which is not incorporated herein by reference.

Forward-Looking Statements

This press release contains 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, the Company is using forward looking statements when it discusses building a diverse

portfolio of products candidates with the potential to address unmet medical needs for incurable diseases, and that the Company's

platforms potentially offer solutions for several diseases that share the same biological pathology. 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 those historic results referred to in this press release would not be interpreted differently

in light of additional research and clinical and preclinical trials results. Because such statements deal with future events and

are based on BioBlast Pharma Ltd.'s current expectations, they are subject to various risks and uncertainties and actual

results, performance or achievements of BioBlast 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 BioBlast Pharma's Annual

Report on Form 20-F filed with the Securities and Exchange Commission ("SEC") on March 31, 2015, and in any subsequent

filings with the SEC. Except as otherwise required by law, BioBlast 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.

U.S. Investor Contact

LifeSci Advisors, LLC