Promising news for hemophilia B patients

June 30, 2020
It’s the last day of June. In a summer full of bad news, we have a promising development for hemophilia B patients. We also have a fascinating look at the animal origins of the world’s first vaccine and the latest from Capitol Hill. Here are around 850 words, just over…

It’s the last day of June. In a summer full of bad news, we have a promising development for hemophilia B patients. We also have a fascinating look at the animal origins of the world’s first vaccine and the latest from Capitol Hill. Here are around 850 words, just over 4 minutes.

Promising news for hemophilia B patients

We’re taking a quick break from COVID-19 news today—we know, we all need it—to look at some promising news for hemophilia B patients.

The news: CSL Behring (BIO member!) announced it will acquire the exclusive license to commercialize a novel late-stage gene therapy candidate for hemophilia B patients from uniQure, a gene therapy company. 

The therapy: Currently in Phase 3 clinical trials, AMT-061 (etranacogene dezaparvovec) has shown to increase the necessary blood clotting protein in hemophilia B patients to such a degree that it reduces or eliminates the tendency for bleeding for many years—possibly eliminating the need for frequent and ongoing replacement therapies with just one dose.

Why it matters: If successful, this could be one of the first gene therapies for hemophilia B to reach the market.

What they’re saying: “Our vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,” said CSL’s CEO and Managing Director Paul Perreault.

What’s next: uniQure will complete the clinical trial and scale up manufacturing for early commercial supply, and the companies need to obtain a few customary regulatory clearances—but this is an exciting step for patients.


More Health Care News: 

AP: Summer may decide fate of leading shots in vaccine race
“People on six continents already are getting jabs in the arm as the race for a COVID-19 vaccine enters a defining summer, with even bigger studies poised to prove if any shot really works—and maybe offer a reality check.”

Axios: Coronavirus vaccine may not be enough to achieve herd immunity in U.S.
“[Dr.] Fauci said he remained ‘cautiously optimistic’ that a vaccine for COVID-19 could be available by the end of the year or the start of 2021. But he noted ‘we have a lot of work to do’ in educating people on the safety of vaccines amid disinformation from the anti-vaccination movement.”

How cows and milkmaids gave us vaccines

Because our long, solitary nature walks aren’t ending anytime soon, BIO’s Cornelia Poku points us to a fascinating podcast on the history of vaccines—and the important role of cows, then and now.

The concept of “immunity” has been around since as early as 430 B.C.E. Greece, where historian Thucydides discovered that people who had recovered from the plague were able to attend to people sick with it, begins the NPR Planet Money podcast episode.

Then, in the Middle Ages, experimenters in China had the idea to “manufacture immunity” by scraping off pieces of smallpox scabs, grinding them to a powder, and blowing the powder up people’s noses. (Ew.)

And it worked—kind of. Severe infections dropped, and the method spread around the world.

Fast forward to late-1700s England, where milkmaids noticed they (and the cows) were developing spots that looked similar to smallpox but didn’t hurt or kill either the cows or the humans. Coincidentally, the milkmaids did not get smallpox.

An English physician decided to formalize the exposure process, which he called “variola vaccina,” for the Latin word for cow, vacca—and the rest is history.

Now, in in the midst of another pandemic, cows are yet again playing a role in the race to find a vaccine. As we’ve reported, BIO member SAB Biotherapeutics is also using cows to develop a vaccine for COVID-19 by using the animals to produce human antibodies.

The vaccine origin story reminds us of the importance of One Health policies—researching the links between human, animal, and environmental health to solve our most pressing challenges, including our worst pandemics.

Learn more about One Health.

BIO Beltway Report

President Trump’s Tuesday: Another day without much on the public agenda. 

What’s Happening on Capitol Hill: Before heading home to their districts for the July 4th weekend, the House and Senate are cramming in quite a few COVID-related hearings. The House is expected to consider the Democrats’ $1.5 trillion infrastructure bill, though Trump says he’ll veto. Meanwhile, Sen. Finance Chairman Chuck Grassley (R-IA) wrote in a WSJ op-ed that he plans to push the Prescription Drug Pricing Reduction Act after the holiday.

On Deck This Week: 

Tuesday, June 30 at 10 AM ET | Senate Foreign Relations Committee: COVID-19 and U.S. International Pandemic Preparedness, Prevention, and Response

Tuesday, June 30 at 10 AM ET | Senate HELP Committee: COVID-19: Update on Progress Toward Safely Getting Back to Work and Back to School, feat. Drs. Fauci (NIAD/NIH), Redfield (CDC), Giroir (HHS), and Hahn (FDA) 

Tuesday, June 30 at 11 AM ET | House Energy & Commerce Committee: High Anxiety and Stress: Legislation to Improve Mental Health During Crisis 

Thursday, July 2 at 9 AM ET | House Small Business Subcommittee on Economic Growth, Tax, and Capital Access: Supply Chain Resiliency

Thursday, July 2 at 10 AM ET | Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies: Review of Operation Warp Speed: Researching, Manufacturing, & Distributing a Safe & Effective Coronavirus Vaccine

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