gene therapy
Intellia says its CRISPR-based therapy can be redosed
Intellia reported early data yesterday showing that its CRISPR-based therapy that targets a harmful protein called TTR can be redosed. This is the first-ever clinical data showing redosing with an in vivo CRISPR-based therapy, the company said.
The data are from an ongoing Phase 1 study of the therapy, NTLA-2001. Three patients who received redosing experienced a 90% median reduction in serum TTR at 28 days, and it was well-tolerated, Intellia said.
The therapy is currently also in Phase 3 trials for the heart condition ATTR-CM and a rare nerve condition called ATTRv-PN. While redosing is not planned for these studies, the new data show the potential to redose in other disease areas where more than one dose may be needed to reach the desired benefit, the company said.
Investors are closely watching Intellia's program in ATTR-CM after Alnylam reported positive data on its RNAi treatment earlier this week. Stifel analyst Dae Gon Ha wrote that Alnylam's results bode well for Intellia, due to similarities in the approaches that aim to stop production of the TTR protein. "The HELIOS-B outcome has done the heavy lifting - by validating RNAi against hard endpoints in ATTR-CM, a CRISPR-based gene knockout should have little reason for a dramatically divergent outcome," Ha said.
artificial intelligence
How a Flagship startup is using AI to study cell biology
Cellarity, a startup launched by Flagship Pioneering, is trying to use AI to understand the full biology of cells and find new ways to treat disease. The company views traditional drug discovery as "reductionist," since it reduces diseases to one or two targets rather than embracing the complexity of cell biology.
With the startup in the middle of raising a Series D, the last round it hopes to raise before going public, STAT's Brittany Trang sat down with its CEO and one of its head scientists to discuss the company's approach.
Read more.
GLP-1s
Wegovy will soon be available in a major new market
China has approved Novo Nordisk's obesity drug Wegovy, opening the drug up to a massive new market. Recent survey data suggest that more than half of Chinese adults are overweight or have obesity, according to a Lancet series.
Novo shares were up yesterday on the news, but as Reuters points out, the company may not maintain a lead on the obesity market in China for long. Novo's patent on semaglutide, the active ingredient in Wegovy and in the sister diabetes drug Ozempic, is set to expire in less than two years in China, while it has a patent until 2031 in Europe and Japan and until 2032 in the U.S.
It's also not clear how Novo would handle supplying drugs for such a populous country. Novo (as well as its rival Lilly) have run into recurrent shortages with their GLP-1-based drugs due to the immense demand, even as the companies rapidly ramp up manufacturing capacity.
real estate
Home sweet home
If you've ever wanted to live in the offices of one of the world's largest pharmaceutical companies — now is your chance.
KENA BETANCUR/AFP via Getty Images
It turns out Pfizer's former New York headquarters on 42nd St are being converted into 1,500 apartments in the biggest conversion in the city's history, according to the Manhattan Borough president.
No comments