STAT

STAT+: Gene therapy eyedrops restored a boy’s sight. Similar treatments could help millions

MIAMI — Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye.

MIAMI — Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye.

Antonio, who’s been legally blind for much of his 14 years, can see again.

The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio’s eyes?

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1 year 8 months ago

Biotech, biotechnology

STAT

AstraZeneca is third member to leave PhRMA in five months

WASHINGTON — AstraZeneca has decided to leave the brand drug lobbying powerhouse PhRMA halfway through the year, the organization said.

The exit is the group’s third in five months, as AbbVie exited PhRMA in December and Teva Pharmaceuticals left in February.

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1 year 11 months ago

Pharma, Politics, Advocacy, biotechnology, Congress, drug pricing, life sciences, Pharmaceuticals, policy

STAT

STAT+: Focused exclusively on life sciences, three top investors set out on their own

Almost a year ago, three top investors at Lux Capital and Obvious Ventures announced they were leaving the firms, teasing on Twitter that they were starting “something new.”

That something is a new venture capital firm, which launched Wednesday with $350 million for its first fund.

Almost a year ago, three top investors at Lux Capital and Obvious Ventures announced they were leaving the firms, teasing on Twitter that they were starting “something new.”

That something is a new venture capital firm, which launched Wednesday with $350 million for its first fund.

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2 years 2 months ago

Biotech, biotechnology, finance, STAT+, venture capital

STAT

STAT+: Study points to new ‘king on the block’ for treatment of chronic lymphocytic leukemia

NEW ORLEANS — One of the best therapies for some types of lymphoma and leukemia has been a drug called ibrutinib, made by AbbVie. When it hit the market in 2013, the drug revolutionized the treatment of these cancers and represented a major step forward from chemotherapy and some other drugs at the time.

But research presented at the American Society of Hematology meeting in New Orleans suggested that a second-generation drug called zanubrutinib from BeiGene is about to unseat ibrutinib as “the king on the block” for the treatment of chronic lymphocytic leukemia, said Catherine Diefenbach, medical director of the lymphoma program at the NYU Langone’s Perlmutter Cancer Center.

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2 years 4 months ago

Biotech, Health, ASH22, biotechnology, Cancer, STAT+

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