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05 Nov 2019

Nonprofit Emily’s Entourage is Encouraged to See CF Gene Therapy Advance Through Spirovant Acquisition

Acquisition will advance two mutation-agnostic gene therapy programs for Cystic Fibrosis, a rare disease that affects 70,000 people worldwide. 

Lower Merion, PA — Emily’s Entourage, an innovative 501(c)3 foundation that has raised millions of dollars since 2011 to speed groundbreaking treatments and a cure for rare nonsense mutations of Cystic Fibrosis, welcomes with great enthusiasm the news that Sumitomo Dainippon Pharma, in partnership with Roivant Sciences, has acquired Spirovant Sciences (formerly Talee Bio). The acquisition validates Emily’s Entourage’s venture philanthropy business model of funding scientific research that supports the development of therapies for Cystic Fibrosis.  

“We are thrilled about the acquisition of Spirovant (formerly Talee Bio) and Roivant’s commitment to expeditiously develop the two gene therapy programs,” said EE founder Emily Kramer-Golinkoff. “This reflects EE’s focus on accelerating the development of lifesaving therapies for Cystic Fibrosis and bringing them to patients quickly. We believed in the science and in the team’s deep expertise in CF gene therapy since the start and we are encouraged to see our early investment move forward towards clinical development.” 

In 2016, Emily’s Entourage provided seed funding to launch Talee Bio, a novel gene therapy company focused on developing revolutionary treatments and a cure for CF. A unique partnership between the University of Iowa, Children’s Hospital of Philadelphia and Philadelphia venture firm Militia Hill Ventures, Talee Bio was made possible through the organization’s scientific connections and commitment to expedite advances by harnessing the power of venture capital.

Over the past three years, this work has developed two promising gene therapy programs originating from the company’s academic founders at the University of Iowa. Among the most exciting elements of the program is that, unlike many of the targeted therapies in market and development for CF treatment, these gene therapy approaches could go far beyond merely controlling the disease–they could cure the disease. In addition, the gene therapies are “mutation-agnostic,” meaning they would treat everyone with CF no matter the individual’s genetic mutation. Earlier this year, Talee Bio (Spirovant) received a $4.5M award from the Cystic Fibrosis Foundation to advance development of 2 gene therapy compounds (TL-101 and T-102).

The Sumitomo-Roivant partnership confirms that the work that has already begun will continue at all due speed with a focused goal of finding effective therapies and a cure for CF. 

“It is impossible to overstate the urgency of this work,” said Kramer-Golinkoff. “There have been recent advances in the treatment of CF that benefit 90% of the CF community, but there has been little progress for the remaining 10%, including those, like me, with nonsense mutations of CF. We refuse to accept being left behind.”

About Emily’s Entourage
Emily’s Entourage (EE) is an innovative 501(c)3 foundation that has raised millions of dollars since 2011 to speed groundbreaking treatments and a cure for rare nonsense mutations of Cystic Fibrosis, a fatal genetic lung disease. The organization was founded by Emily Kramer-Golinkoff, a young woman with advanced stage Cystic Fibrosis, and her devoted friends and family.

The urgency of Emily’s condition is spurring revolutionary research and drug development that could help not only those with Cystic Fibrosis (CF), but people who suffer from a wide variety of genetic diseases caused by nonsense mutations, including forms of muscular dystrophy and cancer. Nonsense mutations, while rare among those with CF, are the cause of approximately 12% of all genetic diseases and affect 30 million people around the world.

For media inquiries, please reach out to Kira Rentas, Senior Marketing and Program Coordinator, at kira@emilysentourage.org or 631-902-1993.

 

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The opinions stated by our guest contributors reflect the experiences and thoughts of the author alone. Our publication of these pieces does not reflect Emily’s Entourage’s endorsement or suggest a position on those opinions. The content on this site is for informational or educational purposes only and does not substitute professional medical advice or consultations with healthcare professionals.
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Emily’s Entourage
PO Box 71
Merion Station, PA 19066

Tax ID #45-3768161

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