Article digest · Oct 3 AM

AI & Rare and Intractable Diseases — what each story says

Every story in this issue with a short summary, so you can decide what to read before leaving the site. Summaries are written by AI (Claude) only from each publisher's own description and checked by Jev for anything not in that description. They are not full translations. Follow "Read the original" for the full story.

21 stories14 with summaries9 topics

🔎 Diagnosing rare disease3

simplywall.st10/02 12:44 UTC

What Is Johnson & Johnson (JNJ) Signaling With Gene Therapy And AI Repurposing?

Johnson & Johnson advanced its gene therapy candidate Botaretigene sparoparvovec for X-linked retinitis pigmentosa in ongoing regulatory and clinical processes. The company expanded drug repurposing programs that use AI tools to reanalyze existing compounds for oncology and rare diseases. Management also took a leading role in global collaborations on AI-driven compound repurposing.

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simplywall.st10/01 05:22 UTC

BioMarin Pharmaceutical (BMRN) Could Be 35% Undervalued Following Profluent AI Collaboration

Profluent's new collaboration with BioMarin puts AI-designed enzyme therapies at the center of its research effort, with BioMarin screening AI-generated candidates for its rare disease pipeline. BioMarin's share price has fallen recently, with a 7-day return of 3.55% down and a 30-day return of 8.02% down. Its 1-year total shareholder return is positive at 9.37%.

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simplywall.st10/02 12:44 UTC

What BioMarin Pharmaceutical (BMRN)'s AI Drug Discovery Deal Means For Shareholders

In late September 2026, Profluent announced a collaboration with BioMarin to apply AI protein design models to identify novel enzyme therapy candidates. In the same period, BioMarin entered a global settlement and royalty-bearing patent license with Ascendis Pharma covering TransCon CNP and related products.

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🧬 Genomics1

simplywall.st09/25 02:51 UTC

Tempus AI (TEM) Faces A Valuation Test As It Expands Into Whole Genome Sequencing

Tempus AI outlined a push into whole genome sequencing, planning a research platform of 100,000 fully sequenced genomes tied to clinical records for AI-driven disease analysis. Its shares have a 30-day return of 19.66% and a 90-day move of 46.49%. The 1-year total shareholder return is 6.35%.

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🎀 Breast cancer4

pharmaphorum10/02 11:32 UTC

Everlywell rolls out Clairity AI for breast cancer in US

Digital health firm Everlywell is rolling out Clairity AI across the US. The tool can assess the risk of developing breast cancer from a mammogram.

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🫁 Lung cancer3

Select Science10/02 06:28 UTC

CellCarta and Imagene AI expand CDx collaboration

CellCarta and Imagene AI are expanding AI-powered biomarker and companion diagnostic (CDx) programs for clinical trials and NSCLC.

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🩺 Colorectal and GI1

Bioengineer.org10/02 14:09 UTC

AI Reads Scans to Predict Hidden Spread in Colorectal Cancer Before Surgery

Whether a colorectal tumor has already spread cells beyond its original site is one of the hardest questions to answer before surgery. The article describes AI that reads scans to address it.

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🔬 Rare cancers2

simplywall.st10/01 05:22 UTC

BioMarin Pharmaceutical (BMRN) Could Be 35% Undervalued Following Profluent AI Collaboration

Profluent's new collaboration with BioMarin puts AI-designed enzyme therapies at the center of its research effort, with BioMarin screening AI-generated candidates for its rare disease pipeline. BioMarin's share price has fallen recently, with a 7-day return of 3.55% down and a 30-day return of 8.02% down. Its 1-year total shareholder return is positive at 9.37%.

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🖥️ Diagnosis and pathology5

diagnosticimaging.com10/02 17:05 UTC

Keys to Post-Deployment AI Monitoring in Radiology

In an interview with Diagnostic Imaging, Joseph Cavallo, MD, MBA, shared insights on post-deployment monitoring of AI in radiology.

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Manufacturing Chemist10/02 12:22 UTC

Leica Biosystems brings Tempus pathology products to Aperio AI Store

Expanding the Aperio AI Store gives researchers more ways to study cancer through AI-enabled tools. The tools sit within a connected digital pathology ecosystem.

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Imaging Technology News10/01 16:27 UTC

Study Shows AI Tool Can Help Identify Training Gaps for Radiology Residents

A recently published study found that using AI to track which pathologies radiology residents saw each day made it possible to identify gaps in their clinical exposure. Supplemental, targeted teaching cases could then be provided. The study authors said this helped ensure residents were exposed to the full breadth of pathology needed for training.

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simplywall.st10/01 12:00 UTC

3 Australian AI Healthcare Stocks With Revenue Growth Up To 49%

Rising global bond yields have pushed borrowing costs higher for governments and companies, which puts pressure on healthcare budgets and hospital funding. The article breaks down three ASX AI healthcare stocks from a screener that could benefit if cost control becomes the main priority.

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💊 Treatment and drug discovery1

finance.biggo.com10/02 07:08 UTC

Valo's Brian Alexander: AI Won't Fix Drug Discovery Until Human Biology Moves Upstream

Flagship Pioneering-founded Valo is betting that the AI revolution in drug discovery has been aiming at the wrong target. CEO Brian Alexander makes this argument.

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⚖️ Regulation and approvals1

MedTech Dive10/02 15:25 UTC

FDA to prioritize guidance on AI, surgical robots next year

The FDA outlined its A-list and B-list guidance priorities for the 2027 fiscal year.

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The analysis and visual summary are in the Oct 3 AM issue. Japanese version here.