Connect with us

Hi, what are you looking for?

Jewish Business News

Life science

Israeli Company SpliSense gets Fda approval for cystic fibrosis treatment

American FDA and the European Medicines Agency (EMA) have designated the Company’s Cystic Fibrosis treatment as an “orphan drug” which intended to treat rare diseases

Israeli biopharmaceutical company, SpliSense, announced that the US Food and Medicine Administration (FDA) and the European Medicines Agency have designated the cystic fibrosis treatment technology as an “orphan drug” (EMA).

The FDA and EMA designate medicines and biologic technologies as orphan drugs when they are intended to treat rare diseases.

“The orphan designations we received for our lead drug candidate both from the FDA and EMA are important milestones toward addressing the urgent, unmet needs of patients living with cystic fibrosis,” said Gili Hart, Ph.D., CEO, SpliSense. “We are looking forward to initiating the clinical trial with SPL84-23, which is expected to commence in mid-2022 in multiple centers in the US, Europe, and Israel.”

Please help us out :
Will you offer us a hand? Every gift, regardless of size, fuels our future.
Your critical contribution enables us to maintain our independence from shareholders or wealthy owners, allowing us to keep up reporting without bias. It means we can continue to make Jewish Business News available to everyone.
You can support us for as little as $1 via PayPal at
Thank you.

Cystic fibrosis is a hereditary condition that causes respiratory infections and disability in over 90,000 persons worldwide. It is caused by CFTR gene mutations, which result in defective proteins. While there are currently available medicines for somewhat more than 80% of cystic fibrosis cases, a tiny but significant fraction of people have no therapeutic options.

The product in question is SPL84-23, an ASO (or antisense oligonucleotide), a short and precisely targeted RNA strand that corrects a specific mutation in the CFTR gene that causes cystic fibrosis. A dose of these ASOs is inhaled into the lungs, where they are absorbed into the cells and begin producing corrected mRNA strands and normal proteins, potentially improving lung function significantly.

“In this scenario, we are concealing the [mutated] RNA sequences. We omit sequences that were incorrectly transferred to the RNA,” Prof. Batsheva Kerem, PhD, whose study serves as the cornerstone for SpliSense’s technology told Jpost.

Numerous untreated people are represented by the Cystic Fibrosis Foundation, a nonprofit organization founded in the United States to advance the development of a cystic fibrosis cure and to enable patients to live longer, better lives. The foundation committed $8.4 million in SpliSense’s therapy technology in early 2021 as part of a $28.5 million funding round.



You May Also Like

World News

In the 15th Nov 2015 edition of Israel’s good news, the highlights include:   ·         A new Israeli treatment brings hope to relapsed leukemia...


The Movie The Professional is what made Natalie Portman a Lolita.


After two decades without a rating system in Israel, at the end of 2012 an international tender for hotel rating was published.  Invited to place bids...

VC, Investments

You may not become a millionaire, but there is a lot to learn from George Soros.