
FDA approves KRESLADI, first CIRM‑backed gene‑editing therapy
The FDA cleared KRESLADI, a gene‑editing treatment from Rocket Pharmaceuticals for severe leukocyte adhesion deficiency‑I in children, eliminating the need for a bone‑marrow donor. It is the first product directly funded by California’s Institute for Regenerative Medicine to receive approval.
Also developing:
By the numbers: Syneron Bio raises $150M Series B

New Fraser Institute research reveals that 1.4 million Canadians awaiting medically necessary procedures in 2025 collectively lost more than $4.2 billion in wages and productivity. On average, each patient forfeited about $3,043 of earnings while waiting. The study highlights a median wait of 28.6 weeks from primary‑care referral to treatment, among the longest recorded. The analysis excludes specialist appointment and diagnostic imaging delays, suggesting the true economic burden is even higher.
The UK Medical Training (Prioritisation) Bill 2026 passed, aiming to give preference to doctors trained within the country. Critics argue the bill’s wording unintentionally excludes refugee and asylum‑seeking physicians who have completed NHS‑funded training. Leaders of the UK National Refugee...

Today, I gave a virtual talk to a leading pharma company's team about the future of oncology. As they asked me to focus on AI's role in that, I'll describe real-life and some hypothetical examples of how the 5 levels...
https://open.substack.com/pub/pauloffit/p/the-v-word?utm_campaign=post-expanded-share&utm_medium=web More than one year passed before a public health official said the "V" word during a recent measles epidemic. Why?
Dyne Therapeutics reported that its exon‑skipping candidate z‑rostudirsen sustained respiratory and cardiac benefits through 24 months in the Phase 1/2 DELIVER study for Duchenne muscular dystrophy. The therapy maintained forced vital capacity, circumferential strain and left‑ventricular ejection fraction improvements compared with...

The UK Department of Health and Social Care has answered its five‑week consultation on NICE regulations by granting ministers a limited power to set the cost‑effectiveness threshold used in technology appraisal (TA) and highly specialised technology (HST) programmes. The change...

A new study in the Journal of the American College of Radiology shows radiology residency costs are climbing again after a pandemic‑induced dip. Diagnostic radiology applicants now spend about $3,800 on average, a 65% increase from the 2021‑2022 low, while...
SS Innovations announced an $18.6 million private placement to accelerate its global rollout, including a U.S. launch of the SSi Mantra surgical robot. The company filed for FDA 510(k) clearance in December, with a decision expected by mid‑2026, and aims to compete...
Junshi Biosciences has received acceptance from China’s National Medical Products Administration for its subcutaneous toripalimab injection (JS001sc), covering 12 cancer indications. The filing marks the first domestic anti‑PD‑1 monoclonal antibody in a subcutaneous formulation to reach the marketing application stage....
Alabama enacted House Bill 300, requiring insurers to cover medically necessary supplemental breast imaging—contrast‑enhanced mammography, ultrasounds, and MRIs—without patient cost‑sharing. The law, signed by Gov. Kay Ivey on March 5, joins roughly 30 states that have passed similar mandates. Advocacy groups,...

Uğur Şahin and Özlem Türeci announced they will leave BioNTech by the end of 2026 to launch a new company focused on next‑generation mRNA technologies. BioNTech will concurrently narrow its portfolio, concentrating on late‑stage therapeutic candidates and its existing vaccine...
AIAInow is hosting a deep‑dive session on using generative AI to accelerate healthcare research through Unity Health’s Gemini network. The program showcases Gemini’s ability to process massive clinical notes, automate ICD coding via the ICD Assist project, and generate synthetic...
A 16‑year longitudinal study of more than 66,000 women aged 25‑42 found that those who regularly slept fewer than seven to eight hours faced a markedly higher risk of developing hypertension. Participants with sleep difficulties also tended to have poorer...
The Scottish Government will launch Public Services Delivery Scotland (PSD Scotland) on 1 April 2026, merging NHS Education for Scotland and NHS National Services Scotland into a single body. PSD Scotland will lead workforce planning, infrastructure, innovation and, crucially, digital transformation across...

Healthcare organizations are grappling with “shadow AI,” the unsanctioned use of generative AI tools by staff. A Wolters Kluwer survey found 40% of workers encounter such tools and 17% use them, while Netskope reports enterprise‑managed AI adoption jumped from 12% to...

A new analysis shows the world’s largest pharmaceutical firms cut antimicrobial development by 35% over the past five years, dropping from 92 to 60 candidates. Only five of the 39 pipeline projects aimed at WHO priority pathogens include pediatric formulations...

New research by Google, Imperial College London and the NHS, published in Nature Cancer, demonstrates that an AI‑based mammography system can identify 25% of interval cancers previously missed by radiologists. In a study of 125,000 women, the AI also detected...

The FDA has eliminated the longstanding requirement for two pivotal clinical trials, adopting a single‑trial default to speed drug approvals. The agency pairs this change with a mandate for more rigorous post‑market surveillance to catch safety signals after launch. Pharmaceutical...
Primary care physicians are feeling the squeeze from projected Medicaid cuts and low insurer reimbursement rates, prompting practices like Valley Medical Group in western Massachusetts to lay off 40 staff members, roughly 10% of its workforce. To regain bargaining power...

NewcelX has entered a collaborative research agreement with Eledon Pharmaceuticals to advance its NCEL‑101 cell therapy for type‑1 diabetes. The partnership integrates NewcelX’s off‑the‑shelf islet replacement product with Eledon’s anti‑CD40L monoclonal antibody, tegoprubart, which has been used in over 100...

Paperpal for Life Sciences, Cactus Communications' AI platform, has completed a HIPAA readiness assessment and can now offer Business Associate Agreements for handling PHI and PII. The certification underscores the platform’s secure‑by‑design architecture and data‑governance framework. Paperpal claims its AI...

The author uses a murder‑mystery framework to expose how the gradual erosion of abortion rights was driven by obscure legislators and policy architects rather than obvious villains. By tracing tragedies such as Rosie Jimenez’s 1977 death and Becky Bell’s 1984...
Ferring Pharmaceuticals reported revenue above €2.5 billion for 2025, a 7% increase at actual exchange rates and 10% at constant rates. Growth was powered by its flagship reproductive medicine Menopur and the U.S. ramp‑up of gene‑based bladder‑cancer therapy Adstiladrin. Operating profit...

A medical council fitness‑to‑practice committee found Dr C guilty of poor professional performance after he inappropriately exposed a Defence Forces officer’s breast during a September 2022 consultation at the Curragh Camp. The panel also determined he failed to document the exam...
Eight‑year‑old Jonah’s severe tooth infection forced his family into two emergency‑room visits, highlighting a national surge in pediatric dental emergencies. ER visits for non‑traumatic dental issues among children rose roughly 60% from 2019 to 2022, with some hospitals reporting increases...

Researchers at Texas Children’s Duncan Neurological Research Institute and Baylor College of Medicine demonstrated that skipping exon e2 of the MECP2 gene boosts MeCP2 protein production by 50‑60%, rescuing neuronal function in Rett syndrome mouse models and patient‑derived cells. The study...

The Independent Public Regional Hospital in Szczecin, Poland, was hit by a ransomware‑style cyberattack on the night of March 7‑8, 2026, encrypting key parts of its electronic medical record system. With digital access blocked, the hospital shifted to a fully paper‑based...
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Raj Shah, head of healthcare at Nordic Capital, argues that Asia is becoming a pivotal arena for healthcare private equity. Demographic pressures, especially aging populations and rising middle‑class incomes, are creating a surge in demand for medical services. Investors, both...

The FDA released draft guidance encouraging the use of Bayesian statistics in drug and biologic clinical trials, aiming to shorten development timelines and lower costs. By allowing external data—known as priors—to be incorporated, the approach promises more efficient, adaptive studies,...

The episode challenges the conventional view of rubella, arguing that the virus was never definitively isolated and that the disease is historically mild. It critiques the 1941 Australian study linking rubella to congenital cataracts and the subsequent vaccine rollout, suggesting...

In this episode, Peter Lee, President of Microsoft Research, recounts the evolution of AI from early neural networks to today’s large language models, describing how Microsoft recognized OpenAI’s potential early on and invested heavily despite industry skepticism. He explains the...

Rapport Therapeutics has granted Tenacia Biotechnology exclusive rights to develop and commercialize its TARPγ8‑specific AMPA receptor negative allosteric modulator RAP‑219 in Greater China, covering indications such as focal onset seizures and bipolar mania. The agreement provides Rapport with a $20 million...
Amid rising mental‑health crises, policy attacks, and AI‑driven discourse, author Mike Magee argues that a liberal‑arts education offers a vital counterbalance to the divisive Trump era. He highlights how institutions like Le Moyne College embed ethics, history, and critical thinking into...

The "Happy Mum Happy Baby" podcast’s Season 17 delivers a diverse lineup of episodes that explore parenting realities, from Steve Backshall’s reflections on fatherhood to raw conversations about postpartum psychosis, breastfeeding pressures, and celebrity motherhood stories. Notable milestones include the 300‑episode...
At HIMSS26, the pre‑conference highlighted how healthcare organizations are deploying digital technologies and artificial intelligence to transform care delivery. Sessions showcased AI‑driven diagnostics, telehealth expansion, and data‑interoperability initiatives across hospitals and health systems. Demonstrations illustrated real‑world use cases, from predictive...

New research from NYU Stern finds private‑equity‑owned nursing homes have 11% higher resident mortality, 4.4% fewer nurses, 25% more hospital complications, and a ten‑fold increase in bankruptcy risk. The study links these outcomes to aggressive debt‑laden buyouts, sale‑leaseback deals and...
Researchers at Icahn School of Medicine created the first sex‑specific atlas of GLP‑1 expression in the mouse brain using RNAscope, mapping the peptide across 25 nuclei. The atlas shows pronounced differences between females and males, especially in hindbrain nuclei of...

In this episode, Professor David Fernández‑Rivas discusses the role of engineers as problem‑solvers and the necessity of interdisciplinary collaboration, especially between physics, chemistry, and bioengineering. He explains microfluidics—manipulating fluids at the micrometer scale—and its parallels with microelectronics, then delves into...
David Creel, PhD, RD, a clinical psychologist and dietitian at the Cleveland Clinic, emphasizes that lasting weight‑loss hinges on behavioral psychology rather than isolated diet or exercise prescriptions. He outlines a framework that blends collaborative communication, self‑monitoring, skill‑building, and relapse‑prevention...

The U.S. Department of Health and Human Services, under new leader Robert F. Kennedy Jr., released a video that mimics Nintendo’s Smash Bros. Melee to announce an inversion of the traditional food pyramid. The spoof places Kennedy himself in the game’s style,...
On the opening day of HIMSS26, industry leaders debated AI ethics and regulation in healthcare. Sessions emphasized deploying AI responsibly, with a human‑centered approach that safeguards patient data and outcomes. Speakers highlighted the gap between rapid AI innovation and the...
CEO Hal Wolf received a tribal honor on the opening day of HIMSS26. The award recognized his leadership in advancing digital health solutions for federally recognized tribes. The ceremony took place during the conference’s pre‑conference forums, underscoring HIMSS’s emphasis on...
Maternal‑fetal specialist Dr. Cara Dolin confirms pregnant people can drink coffee, but only in moderation. The American College of Obstetricians recommends keeping caffeine below 200 mg per day, roughly one 12‑ounce cup of coffee. Exceeding this limit has been associated with...
Exercise is generally safe for most cancer patients and can be a powerful adjunct to treatment. Medical oncologists emphasize that even modest activity—walking, yoga, or light strength work—helps lower inflammation, fatigue, and improves sleep and mood. The American Cancer Society...
Merck released phase III data from its Litespark‑011 trial, showing a potent HIF‑2α inhibitor that could reshape renal‑cell carcinoma therapy. Researchers at INSERM uncovered hypothalamic tanycytes as a primary mechanism for clearing pathological tau, opening new avenues for Alzheimer’s treatment....

Aquila has stepped out of stealth mode, securing state and federal contracts to deploy a healthcare data integration platform aimed at cross‑jurisdiction epidemiological and population‑health tracking. Led by Dr. Jaime Bland, the startup targets the 30% of U.S. hospitals that...

A royal commission report released in March 2026 concluded that New Zealand’s Covid‑19 response was among the world’s most effective, crediting low death rates and aggressive elimination tactics. The inquiry focused on the 2021‑2022 shift from elimination to a suppression strategy,...
Duke University researchers discovered that resident tissue macrophages (RTMs) are essential for maintaining intraocular pressure (IOP) in mice. Fluorescent tagging showed that selective removal of RTMs clogged the eye's outflow pathway, causing fluid buildup and elevated IOP, while depletion of...
Another example of how RFK Jrs version of MAHA is totally incoherent. --> he wants to end 'GRAS' chemicals in food (generally regarded as safe) --> his FDA opens door to flavored e-cigs (which use GRAS chemicals - NEVER TESTED FOR INHALATION...

Great evening with Frontera’s mgmt team and Denver area founders. Frontera is using AI to impact behavioral disorders, esp Autism. No holds barred panel w/ friends Ben Hemani (Bison Ventures, ex Gates Ventures), Galym Imanbayev (Lightspeed) and Amol (Frontera, ex...