Jared Whitlock

Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans Chinese firm Belief BioMed launches $350K hemophilia B gene therapy, challenging $3.5M Hemgenix by CSL Behring/uniQure, as China leads global race for cheaper gene therapies.

Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans

Latest feature + data analysis

endpoints.news/china-is-tak...

Exclusive: EveryOne Medicines shuts down, ending custom cure venture that banked on new FDA pathway EveryOne Medicines, a biotech developing individualized genetic therapies, to close after FDA's new guidance falls short of expectations for streamlined drug approvals.

EveryOne Medicines, a startup making individualized therapies for genetically unique diseases, is shutting down a week after the FDA announced a streamlined pathway for those types of bespoke medicines. @jaredwhitlock.bsky.social and I have the scoop for @endpts.com: endpoints.news/everyone-med...

Exclusive: EveryOne Medicines shuts down, ending custom cure venture that banked on new FDA pathway EveryOne Medicines, a biotech developing individualized genetic therapies, to close after FDA's new guidance falls short of expectations for streamlined drug approvals.

Exclusive: EveryOne Medicines shuts down, ending custom cure venture that banked on new FDA pathway

From @scienceboss.bsky.social and me:

endpoints.news/everyone-med...

China's MGI sells US subsidiary Complete Genomics after Biosecure Act stalled ambitions Swiss-based life sciences company Swiss Rockets will buy MGI subsidiary Complete Genomics, the companies said Monday. Terms of the deal were not disclosed.

China's MGI sells US subsidiary after Biosecure Act stalled ambitions

endpoints.news/swiss-rocket...

After rallying, Grail's cancer test faces new uncertainty due to UK study After mounting a comeback with fresh capital and a flashy partnership, cancer testing firm Grail once again faces major questions over the future of its blood test that screens for dozens of cancers.

After rallying, Grail's cancer test faces new uncertainty due to UK study

My latest via @endpts.com

endpoints.news/grails-cance...

Chinese biotech claims promising brain cancer results using debated approach NeuExcell Therapeutics says it slowed an aggressive form of cancer in the first human test of a gene therapy technique that aims to coax the brain into healing itself.

A Chinese biotech says its gene therapy slowed aggressive brain cancer in an early human test, but the debated approach is already drawing skepticism from some researchers.
endpoints.news/neuexcell-th...

Can a Chinese drugmaker become a big pharma company? Hengrui is testing the waters With a valuation of about $61.2 billion, Hengrui is now larger than Takeda and Bayer. But there’s still one big item on its bucket list: selling its own treatments outside of China.

Can a Chinese drugmaker become Big Pharma? Hengrui is testing that idea, laying plans to move beyond dealmaking and sell its own drugs in the US and Europe as it grows into a global force.

Gene editing's search for a 'holy grail' has led to an explosion of tools, but few cures Former Intellia scientist John Finn reflects on gene editing's evolution and the closure of Tome Biosciences, highlighting the industry's focus on tools over medicines.

I'm late, but don't miss this piece from
@scienceboss.bsky.social:

Gene editing's search for a 'holy grail' has led to an explosion of tools, but few cures

Via @endpts.com

endpoints.news/as-gene-edit...

RFK Jr. adds conditions to newborn screening list, but future path unclear HHS Secretary Robert F. Kennedy Jr. added metachromatic leukodystrophy and Duchenne to a federal newborn screening list, reviving efforts that had been stalled under the Trump administration — though ...

RFK Jr. adds conditions to newborn screening list, but future path unclear

endpoints.news/rfk-jr-adds-...

China has a cheap, quick and quiet way to test novel therapies. Western genetic medicine makers want in IITs have grown increasingly popular among China researchers over the past decade, and have helped China’s fast-growing biotech sector catch up with rivals in the US and Europe. The very leniency that...

China has a cheap, quick and quiet way to test novel therapies. Western genetic medicine makers want in

endpoints.news/western-gene...

Via @leileiwu.bsky.social and @scienceboss.bsky.social

Alumni Notes: A Bounty of Books and Breaking News - Knight Science Journalism @MIT A recurring roundup of news about former KSJ Fellows, including a compendium of what the fellows have been writing.

Alumni Notes: From unchecked garbage fires to academic freedom, our alumni are telling the stories that link science to policy, and the everyday impacts on communities.

ksj.mit.edu/news/2025/12...

Nationwide Children’s gene therapy shows early promise after Taysha pullback Nationwide Children's Hospital delivers first gene therapy for SLC6A1 disorder to 8-year-old Maxwell Freed, showing early promise in treating seizures and developmental delays.

Nationwide Children’s gene therapy shows early promise after Taysha pullback

endpoints.news/nationwide-c...

Kansas County Agrees to Pay $3 Million Over Police Raid of Newspaper

Kansas County Agrees to Pay $3 Million Over Police Raid of Newspaper #Journalism #Kansas www.nytimes.com/2025/11/11/u...

We've been here before but let's see how FDA responds this time w/ Prasad wielding his influence. (I'm sure WSJ ed board + Loomer are already drafting their 2 cents...)

"Sarepta's Duchenne confirmatory trial fails, but biotech will ask FDA for full approval anyways"

endpoints.news/duchenne-con...

Post-Hoc: As China moves beyond ‘me-too,’ do Western regulators need to reconsider? Analysis: Chinese biotech dominates ESMO cancer conference with 3 major studies, including Kelun's Sac-TMT ADC showing 40% reduced death risk, sparking debate on FDA's stance on China-only data.

Good piece from @leileiwu.bsky.social:

Post-Hoc: As China moves beyond ‘me-too,’ do Western regulators need to reconsider?

endpoints.news/as-china-mov...

Grail reports fewer false alarms for blood cancer test ahead of FDA filing Grail’s blood test got better at telling when a positive result actually means cancer based on study findings, potentially strengthening the company’s case for a wider rollout, though questions remain...

Grail’s blood test got better at telling when a positive result actually means cancer based on study findings, potentially strengthening the company’s case for a wider rollout, though questions remain.

endpoints.news/grails-cance...

1A Listening to the news can feel like a journey. But 1A guides you beyond the headlines – and cuts through the noise. Let's get to the heart of the story, together – on 1A.Support NPR and get your news ...

Thank you everyone for making yesterday such an amazing, dream-come-true pub day for Lab Dog! The fun continues today with a live in-studio interview on @wamu.org.web.brid.gy 1A. Tune in or catch it later on the podcast! www.npr.org/podcasts/510...

D’Angelo, Grammy-winning R&B singer who became an icon with ‘Untitled (How Does It Feel),’ dies D’Angelo, the Grammy-winning R&B singer recognized by his raspy yet smooth voice and for garnering mainstream attention with the shirtless “Untitled (How Does It Feel)” music video, has died.

This one truly hurts. 💔
D'Angelo was a singular, once in a generation talent.
It feels cruel to lose him at a time when great artistry is sorely needed.

apnews.com/article/dang...

In China, a low-cost push to rival a life-saving, $2M medicine Indian veterinarian Nirnay Murthy sought Lantu Biopharma's experimental gene therapy for his son's spinal muscular atrophy after being unable to afford Novartis's $2.1M Zolgensma treatment.

ICYMI:

In China, biotech startups are racing to make low-cost gene therapies, challenging multi-million dollar price tags and aiming to bolster global reach.

endpoints.news/chinese-biot...

In China, a low-cost push to rival a life-saving, $2M medicine Indian veterinarian Nirnay Murthy sought Lantu Biopharma's experimental gene therapy for his son's spinal muscular atrophy after being unable to afford Novartis's $2.1M Zolgensma treatment.

“We will show the world that there is a better, more responsible, and economically more sensible way of drug development,” -- Chinese biotech offering a low-cost version of a gene therapy that saves the lives of small children but at a cost of $2m

endpoints.news/chinese-biot...

In China, a low-cost push to rival a life-saving, $2M medicine Indian veterinarian Nirnay Murthy sought Lantu Biopharma's experimental gene therapy for his son's spinal muscular atrophy after being unable to afford Novartis's $2.1M Zolgensma treatment.

For families, a $2.1M medicine can feel like both a miracle and an out-of-reach reality.

Now, biotechs in China are racing to develop lower-cost alternatives:

endpoints.news/chinese-biot...

HHS says ‘the science speaks for itself’ on mRNA cuts. Some of the scientists it cites disagree Some of the studies that underpin the Trump administration’s decision to veer away from mRNA vaccine research are being misapplied, co-authors of the work say, muddling the move by HHS and Secretary R...

HHS says ‘the science speaks for itself’ on mRNA cuts. Scientists it cites disagree

Good piece from @maxbayer.bsky.social

endpoints.news/researchers-...

Roche doubles down on its Alzheimer's ambitions as next-gen amyloid-lowering drug excites scientists Roche revealed the latest cut of data from its closely-watched Phase 1/2 study of trontinemab, an amyloid-targeting antibody designed to latch onto receptors that yank it across the protective blood-b...

The session at #AAIC25 on Roche's next-gen #Alzheimers drug #trontinemab was jam packed and despite one patient's death, the excitement for this drug was palpable. Read more about the dramatic amyloid reduction and surprisingly low ARIA in @endpts.com - endpoints.news/roche-double...

Breaking: FDA will ask Sarepta to stop selling Duchenne treatment, following patient deaths FDA to request Sarepta halt shipments of DMD drug Elevidys after two patient deaths, HHS official says. Trading of $SRPT shares paused after decline.

We are closely reporting on this story. If you have any tips, please contact me on Signal at jaredwhitlock.73

endpoints.news/fda-asks-sar...

Sarepta patient dies in limb-girdle study, latest death after earlier patient safety concerns Patient dies from liver failure in Sarepta Therapeutics' gene therapy trial for limb-girdle muscular dystrophy, following two deaths in company's Elevidys DMD program.

Sarepta Therapeutics said that a patient died in a clinical trial testing one of its gene therapies for limb-girdle muscular dystrophy, the latest death for a company that’s already under heightened scrutiny for safety concerns.

endpoints.news/sarepta-pati...

Fascinating story today from @levfacher.bsky.social about a rehab center where patients are currently being treated for substance use disorder with GLP-1 drugs — even as clinical trials measuring their effectiveness as anti-addiction medications remain years from reading out data.

The Trump administration disbanded a newborn screening panel. Advocates now face a harder path atient groups face a harder and unpredictable path going state-by-state to boost screening for rare but treatable conditions after the Trump administration disbanded a federal advisory committee on ne...

In Focus: The Trump administration disbanded a federal newborn screening panel, and patients now face a hard and unpredictable path to diagnosis and treatment.

The Trump administration disbanded a newborn screening panel. Advocates now face a harder path atient groups face a harder and unpredictable path going state-by-state to boost screening for rare but treatable conditions after the Trump administration disbanded a federal advisory committee on ne...

The Trump administration disbanded a newborn screening panel. Advocates now face a harder path.

“It’s not acceptance. It’s next-level rage," one parent told me.

endpoints.news/the-trump-ad...

China’s rise splits US biotech investors: Cash in, or counter? US investors debate response to China's rise in drug development, with some seeing a national security threat while others embrace collaboration. FDA and policymakers consider reforms to maintain comp...

As China’s drug industry expands, US biotech investors are divided: cash in or push back? Some, like Curie.Bio’s Zach Weinberg, are calling for action to limit China’s growing role in R&D.

China’s rise splits US biotech investors: Cash in, or counter? US investors debate response to China's rise in drug development, with some seeing a national security threat while others embrace collaboration. FDA and policymakers consider reforms to maintain comp...

One biotech VC calls China a threat. Another says Americans won’t forgive politicians who impede life-saving drugs.

Inside the investor split on China’s ascent in drug development.

w/ @andrewedunn.bsky.social + @kylelahucik.bsky.social, @maxbayer.bsky.social

endpoints.news/us-investors...