Key events of the year gone by
- Trump decided not to impose tariffs on pharma, which freed up unexpected, huge amounts of capital.
- The XBI saw its best trading year of the past six, finishing +36% up by the end of the year.
- The transition of FDA commissioners went smoothly, and even in the midst of some political tensions, the agency still approved 44 important new drugs for patients.
A world-expert in Stargardt disease has developed a pill to slow vision loss: why Tinlarebant could win FDA approval this year
After years studying Stargardt’s natural history, Belite Bio’s Dr. Hendrik Scholl is championing Tinlarebant as a practical early intervention - “Intervention is simply one pill a day - and we are able to slow down the progression of disease by more than one-third.”
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Belite Bio concludes phase 3 Stargardt trials with nearly 40% retinal cell preservation
Belite is positioning Tinlarebant for regulatory submission after Phase 3 Stargardt data, emphasizing the long-term safety burden of a systemic, lifelong oral therapy: “For a lifelong treatment, the safety bar is about as high as it gets.”
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Why Flatiron is betting on data quality, not hype, to power the next era of cancer care
With global oncology scale and nearly 2,000 scientific publications, Flatiron Health is positioning itself as the industry’s most trusted evidence partner by pairing AI-driven speed with uncompromising scientific rigor. “We don’t chase hype. We build trust, and in healthcare that’s non-negotiable.”
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“Efficacy with safety”: the north star behind Outpace’s next-gen engineered cell therapies
Outpace Bio is using AI-powered protein design to build “software and hardware” into engineered cells aimed at durable solid-tumor responses. For CEO Marc Lajoie, “efficacy is still the fundamental problem that needs to be solved - efficacy and safety.”
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From diagnostics to drug development: Scipher Medicine’s three-track strategy for high-impact immunology
As Scipher expands from predictive diagnostics into drug development, CEO Reginald Seeto stresses that technology must drive real clinical decisions: ‘You’ve got to bring technology to the point of greatest impact - otherwise even great innovations never get progressed.’
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Why Novellia thinks the best biopharma data strategy starts with patients, not pipelines
“Everything that’s good for patients delivers extraordinary value for biopharma - that overlap is the entire company,” says Shashi Shankar, CEO. From symptom tracking to AI-driven normalization, Novellia builds patient-first data infrastructure that scales into biopharma-grade intelligence.
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Mark Lappe’s lean biotech model: smaller teams, bigger wins
Inhibrx has built its drug discovery engine around tight, focused teams. The philosophy is simple: “We’re best when we’re smaller. When teams get too big, they stop innovating,” shaping how programs are advanced or spun out.
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The lab-data marketplace powering precision medicine’s next wave
As pharma’s demand for real-time lab intelligence accelerates, Prognos Health is leaning in - and after six or seven years of building, the company now sees “a real path to delivering 20 billion health insights by 2050”, says CEO Sundeep Bhan.
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Biotech's new superstructure: Vivodyne's bid to turn human tissues into scalable, searchable data
Vivodyne’s automated human-testing platform aims to deliver unprecedented scale, reproducibility, and biological fidelity in preclinical drug testing - powered by patient-derived tissues that “already know the instructions,” as CEO Andrei Georgescu puts it.
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Inside Fabian Gerlinghaus' Cellares and their $380M push to automate cell therapy manufacturing
With Bridgewater online and global facilities underway, Cellares is moving from platform development to commercial execution. CEO Fabian Gerlinghaus says, “Our factories can produce as many therapies as ten manual facilities - with just ten percent of the people.”
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Why Allucent says its global footprint is now a competitive edge in early-stage trials
Paula Brown Stafford argues that real differentiation now comes from pairing global operations with smart, disciplined innovation. “We're adding AI as a teammate, but we still need our team to have their own expertise to ask the right questions.”
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Building for zero failure: How 4G Clinical is redefining trial supply management
At 4G Clinical, David Kelleher articulates a leadership philosophy built on precision, flexibility, and the belief that execution failures in trials carry real human consequences: “We will never mis-randomize, stock out, miss dose, or compromise the blind.”
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How DNAnexus is enabling precision medicine from research to the point of care
DNAnexus is pushing precision medicine out of research silos to improve care delivery. As CEO Thomas Laur puts it, “The future isn’t moving data into one big bucket. It’s moving algorithms to federated data,” enabling distributed clinical decisions at scale.
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‘The moat is data’: Marc Tessier-Lavigne on building an AI-native drug company
As traditional drug discovery reaches its limits, Xaira is pushing toward a more predictive, AI-native model. CEO Marc Tessier-Lavigne says, “we’re turning this broken, artisanal endeavor into something much closer to an engineering discipline.”
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The long game in cancer screening: Freenome’s data-first strategy
“If we don’t detect cancer early and intervene, it’s very hard to talk about a cure.” Freenome is betting that data scale, commercial discipline, and blood-based screening can finally close oncology’s early-detection gap.
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Why Trevi Therapeutics’ Jennifer Good believes treating cough means targeting the brain
For these patients, cough isn’t just a symptom - it rules their day. Trevi Therapeutics is targeting triggers in the lung as well as cough’s neurological roots,” says CEO Jennifer Good.
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Inside nChroma Bio’s push to deliver a functional cure for Hepatitis B
Epigenetic silencing is emerging as one of biotech’s boldest bets, and nChroma Bio is racing to prove it in the clinic. ‘Functional cures have been elusive, but epigenetic silencers are uniquely suited to the biological challenge for hepatitis B patients,’ Jeff Walsh says.
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How Genomenon finds order in 11 million scientific papers
Biology’s data problem isn’t scarcity, it’s structure. Genomenon is addressing it by converting millions of scientific papers into usable genomic intelligence. “If Google had gone to school to get a PhD in molecular biology, that's what Mastermind is,” says CEO Mike Klein.
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The physics mindset behind one of biotech’s fastest-growing diagnostics companies
BillionToOne’s CEO, Dr. Oguzhan Atay, argues that “we are approaching biological problems from physics-based principles,” a mindset driving the company’s rapid growth, long-term focus, and pursuit of ultra-sensitive cancer and prenatal testing.
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The first inning of a new research paradigm: Emulate, Inc. and the rise of organ-on-a-chip technology
As regulators, pharma, and researchers search for more predictive tools, Emulate, Inc’s CEO, Jim Corbett, argues that Organ-on-a-Chip technology is still early. “If this were a nine-inning baseball game, we’re in the first inning - but the direction is very clear.”
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Lexicon’s next act: betting big on first-in-class cardiometabolic medicine
“We’re really on the precipice of moving the company into that next stage of maturity,” Dr. Mike Exton
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“Speed is survival”: inside 4D Molecular Therapeutics’ bid to scale genetic medicine
Driven by the precision of viral biology, 4D Molecular Therapeutics is redefining gene therapy’s reach - scaling a once ultra-rare field into broad, sustainable markets.
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Affinia closes $40m Series C: CEO Rick Modi explains how low-dose gene therapy capsids could cure cardiomyopathy
“Over the past year, we have made tremendous progress…” Rick Modi says, as Affinia Therapeutics pushes its BAG3-DCM program toward first-in-human testing, aiming to prove that next-generation capsids can restore heart function at dramatically lower doses.
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How Epicrispr are leading the first epigenetic editing trial for muscular dystrophy
“We can regulate any gene, anywhere,” says CEO Dr. Amber Salzman, whose Epicrispr team is pioneering epigenetic editing - a precise, non-cutting approach - to transform neuromuscular disease treatment through durable, targeted control of gene activity.
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Four years of progression-free survival: Nuvation Bio wins FDA approval for lung cancer
With a median progression-free survival nearing four years in ROS1 lung cancer, Nuvation Bio’s breakthrough therapy signals a turning point for precision oncology - validating Dr. David Hung’s patient-first vision.
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From stealth to spotlight: with $70m Series A, ‘total allergic control’ is Excellergy's goal
Backed by $70 million in Series A funding, Excellergy emerges from stealth with a mission to lead a new era in immunology - targeting the full IgE pathway to redefine how allergic diseases are treated.
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New CEO of Kindbody David Stern is in the business of building families
“Fifteen to twenty years ago, a woman under 35 had a 20% chance of live birth per IVF cycle. At Kindbody, today, it’s greater than 50%,” David Stern credits lab innovation and AI for rewriting fertility’s odds.
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A Legend in the making: how Ying Huang is scaling CAR-T to global markets
“After just one single infusion, about a third of patients remained in remission for five years. Some physicians even called it a functional cure,” Ying Huang underlines why CARVYKTI is reshaping expectations in oncology.
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A world-expert in Stargardt disease has developed a pill to slow vision loss: why Tinlarebant could win FDA approval this year
After years studying Stargardt’s natural history, Belite Bio’s Dr. Hendrik Scholl is championing Tinlarebant as a practical early intervention - “Intervention is simply one pill a day - and we are able to slow down the progression of disease by more than one-third.”
Read sponsor >
Belite Bio concludes phase 3 Stargardt trials with nearly 40% retinal cell preservation
Belite is positioning Tinlarebant for regulatory submission after Phase 3 Stargardt data, emphasizing the long-term safety burden of a systemic, lifelong oral therapy: “For a lifelong treatment, the safety bar is about as high as it gets.”
Read sponsor >
Inside Fabian Gerlinghaus' Cellares and their $380M push to automate cell therapy manufacturing
With Bridgewater online and global facilities underway, Cellares is moving from platform development to commercial execution. CEO Fabian Gerlinghaus says, “Our factories can produce as many therapies as ten manual facilities - with just ten percent of the people.”
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Affinia closes $40m Series C: CEO Rick Modi explains how low-dose gene therapy capsids could cure cardiomyopathy
“Over the past year, we have made tremendous progress…” Rick Modi says, as Affinia Therapeutics pushes its BAG3-DCM program toward first-in-human testing, aiming to prove that next-generation capsids can restore heart function at dramatically lower doses.
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How Piramal Pharma plans to become a multi-billion dollar business
Peter DeYoung lends insights on the strategic approach behind Piramal Phama’s expansion and growth.
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We all want our hospital records in one place. Is Novellia the app to do it?
Shashi Shankar explains how his app unifies patients' scattered medical records all together, and what that has to do with the 21st Century Cures Act.
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Backed by AstraZeneca, this company is helping radioligand cancer therapies hit the shelves
"Think of us as a critical partner in the ecosystem, ensuring that brilliant innovation doesn’t get stuck in the development phase but actually reaches the patients who need them."
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CAR-T can only treat 3% of cancer patients. Mongoose Bio is building for the other 97%
Neil Warma explains how Mongoose Bio is pioneering TCR-T therapies that may last longer, tackle more cancers, and require less from patients.
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This biotech is targeting the 'immortality switch' to fight blood cancers
Harnessing Nobel Prize-winning insights on telomeres, Geron’s CEO Dr. John A. Scarlett is taking on some of hematology’s toughest challenges—and winning FDA backing in the process.
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NVIDIA’s Biggest Bet Yet: How Recursion Is Powering a New Era of AI-Driven Drug Discovery
“Almost all of the data that we use comes from our own laboratories - data generation doing real experiments in human biology… The AI side of things will be commoditized eventually, and the winners will be those who have the strongest data moat in life sciences.”
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From Grammys to Gene Therapy: Gaurav Shah’s Rocket-Fueled Revolution in Rare Diseases
A Grammy-winning CEO is taking gene therapy to new heights.
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Is neuromodulation the breakthrough that Alzheimer's has been waiting for?
Sinaptica's precision neuromodulation combines TMS and EEG to target memory networks, offering new hope in Alzheimer's care.
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Mayo Clinic-backed, Halle Berry-approved: Pendulum’s Dr. Colleen Cutcliffe is tackling type 2 diabetes before it even starts.
As clinical trials progress, Pendulum hopes their medical evidence can beat the status quo and encourage Americans to take preventative care into their own hands.
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Blood tests for cancer are here and Guardant Health is leading the charge
Guardant Health has seen a string of consistent firsts - most recently FDA approval for colorectal cancer in July, 2024
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GERD affects millions of Americans. This drug is the first major innovation in three decades.
GERD affects 65 million Americans, yet innovation in treatment had stagnated. Phathom Pharmaceuticals' FDA approval of VOQUEZNA® (vonoprazan) is a big moment for patients.
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Let there Belite: the prospect of an oral pill for vision loss
If left untreated, Stargardt disease and Geographic Atrophy almost always lead to vision loss and blindness - but a groundbreaking oral treatment in the last stage of clinical trials offers new hope.
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How ImmunityBio is transforming cancer - a treatment in just 30 seconds
“I think I'm blessed with truly the best team in all of pharma… we are doing something that's bigger than all of us!”
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Ocugen has a cure in sight... will it get FDA approval soon?
“The cost of gene therapies can reach millions of dollars per patient… Our goal is developing cost-effective treatments that can be accessible globally.”
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Cell therapy used to be expensive, then Cellares changed everything
The CEO of Cellares sits down to tell us how the Cell Shuttle, an engineering marvel of 21st Century biotech, was invented and the lives that it could save...
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Stephen Gillett, CEO of Alphabet's Verily explains the future of precision and public health
We spoke with Stephen Gillett, Alphabet’s health-tech boss on life, leadership and AI.
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Sarah Boyce's Avidity: A deep dive into one of the fastest growing biotechs of all time
Avidity Biosciences has achieved successful data readouts for all three of its programs in Phase 1/2 clinical trials, initiated its first Phase 3 trial, and raised over $1.1 billion this year.
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Inside the world’s most innovative square mile: MassBio leads the life sciences
Kendalle Burlin O’Connell tells us about the privilege of heading up MassBio - the organization that represents the extraordinary life sciences sector in Massachusetts.
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