Jared Whitlock

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...

"In 25 years of covering the FDA, this would be a first," said @statnews.com Matt Herper commenting on the @endpts.com story about Makary today. See below #medsky #biosky

Exclusive: FDA's Makary sought rejection of KalVista’s drug in an unusual move by commissioner, sources say FDA Commissioner Marty Makary reportedly attempted to reject KalVista Pharmaceuticals' drug application, sources said. An HHS spokesperson calls the claim 'totally false.'

Great scoop from @andrewedunn.bsky.social:

Exclusive: FDA's Makary sought rejection of KalVista’s drug in an unusual move by commissioner, sources say

endpoints.news/sources-fda-...

Illumina to buy SomaLogic for $350M, pushing further into proteomics Illumina to acquire proteomics company SomaLogic for $350M upfront from Standard BioTools, expanding beyond DNA sequencing into protein analysis technology.

Illumina to buy SomaLogic for $350M upfront in proteomics push:

endpoints.news/illumina-to-...

Nobel laureate on how Trump's cuts are hurting science Neuroscientist, biologist and 2021 Nobel Prize winner Ardem Patapoutian joined Midday Edition to talk about how the Trump administration's effort to cut back on science spending is impacting researche...

I spoke with Jade Hindmon, host of @kpbssandiego.bsky.social Midday Edition, about the consequences of the current administration’s cuts to science funding. Please share to help spread the word and raise awareness.
www.kpbs.org/podcasts/kpb...

How a tailored ALS drug could help broaden rare disease therapies In 2020, Jaci Hermstad died at age 26 from a rare and aggressive form of ALS. But her story didn’t end there.

Can a drug developed for one person's rare form of ALS become a treatment for many others?

A treatment made just for a 26-year-old woman offers a glimpse into how customized therapies could be just a starting point.

endpoints.news/a-custom-als...

DNA giant Illumina spawned an ecosystem of competitors and customers For Illumina, its former employees are both a boon and a problem. In the 27 years since its founding, the DNA sequencing company unlocked new frontiers in biology, medicine and ...

@jaredwhitlock.bsky.social has written an article about the many new companies spun out/spawned by ex-Illuminites. In it he references the crowdsourced "Illumina Diaspora" database Millard Chan and I created to keep track of them all.
sandiegomics.com/illumina-dia...
endpts.com/how-illumina...

DNA giant Illumina spawned an ecosystem of competitors and customers For Illumina, its former employees are both a boon and a problem. In the 27 years since its founding, the DNA sequencing company unlocked new frontiers in biology, medicine and ...

DNA giant Illumina spawned an ecosystem. Now its former employees are both its customers and fiercest competitors.

My latest is an in-depth look at a different kind of corporate lineage story, via @endpts.com

endpts.com/how-illumina...

Big leases, bigger regrets: Biotech slump turns swanky offices from asset into liability Biotech companies face financial strain from expensive long-term leases signed during pandemic boom, with Ginkgo Bioworks, Prime Medicine & others struggling with high lease obligations amid market do...

During the pandemic boom years, young biotechs signed huge, long-term leases for space in a competitive market.

Now, many of them are being dragged down by those big obligations... and can't get rid of them,
@andrewedunn.bsky.social reports

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Exclusive: Drug compounder Empower was built on risky shortcuts, ex-employees and inspections allege Empower Pharmacy has used poor-quality ingredients, skirted regulations and pushed out or sued employees who raised concerns, an Endpoints News investigation found.

I'm late to the party but don't miss this investigation from @shelbylivingston.bsky.social into Empower, a compounding pharmacy that produces weight loss medications and other drugs.

endpts.com/pharmacy-emp...

Trade Journalism Award Finalists Recognizing excellence in writing for a specialized audience

I'm thrilled to be a finalist for the NIHCM trade journalism award alongside so many amazing reporters whose work I really admire (hi @kakape.bsky.social and @jaredwhitlock.bsky.social !)

Thank you NIHCM!
nihcm.org/awards/trade...