Boston has long been known for its hospitals and universities, but today it is also the beating heart of biotech innovation. Here, scientists are rewriting the rules of medicine: designing drugs with artificial intelligence (AI), correcting genetic errors, and even reversing the biology of aging. The city’s unique ecosystem of talent, capital, and clinical expertise is powering breakthroughs that could transform patient care across the globe.

Reversing liver fibrosis with a once-monthly shot

In Boston’s biotech hub, companies are advancing therapies with the potential to impact millions of lives. One such promising therapy comes from GlaxoSmithKline (GSK) in Cambridge, Mass., where researchers are developing efimosfermin, a once-monthly injection designed to reverse liver fibrosis and transform treatment for fatty liver disease.

Efimosfermin, a long-acting protein therapy, has shown striking results in phase 2 studies and is ready for phase 3. 

“Efimosfermin has the potential to rapidly and significantly reverse liver fibrosis and stop disease progression in moderate-to-severe metabolic dysfunction–associated steatohepatitis (MASH), with a manageable tolerability profile,” says Kaivan Khavandi, GSK’s senior vice president and global head of respiratory, immunology, and inflammation research and development.

MASH is one of the most severe forms of steatotic liver disease, which affects around 5 percent of the global population and is a leading cause of liver transplants in the United States. Unlike glucagon-like peptide-1 (GLP-1) therapies that primarily address metabolic drivers of disease, efimosfermin acts directly on fibrosis. It helps the liver handle fats and sugars, reduces inflammation, and regulates scar tissue buildup.

The potential impact is significant: Preventing cirrhosis and end-stage liver disease could save the US health care system an estimated $40–100 billion over the next two decades. With its extended half-life and low immunogenicity, efimosfermin also offers the convenience of a once-monthly injection.

“We strongly believe efimosfermin can set a new standard of care for patients with fibro-inflammatory liver disease,” Khavandi says.

GSK’s Boston location plays a central role in the company’s work. “It’s a critical hub for biotech innovation and world-class academic expertise,” Khavandi says. The company recently partnered with Boston University’s Centre for Regenerative Medicine to accelerate discovery.

Powering precision oncology

Other Boston companies are tackling cancer, one of medicine’s most urgent challenges.

MOMA Therapeutics was founded on a bold idea: to create drugs for a class of highly dynamic proteins once thought too difficult to target. Known as molecular machines, these proteins power essential cellular processes, from normal housekeeping to energy production that can be co-opted to promote cancer growth.

“MOMA is really all about making transformative medicines for patients,” says CEO Asit Parikh. “If we can drug these targets once, each subsequent project becomes easier, giving us insights at scale.”

Five years ago, MOMA existed only on paper. Today, it has two medicines in clinical trials and a rich discovery pipeline supported by partnerships with Roche and Bayer Pharmaceuticals. Its lead program, MOMA-313, is a first-in-class inhibitor of DNA polymerase theta, now in phase 1 testing for tumors with homologous recombination deficiency (HRD) mutations, including breast, ovarian, prostate, and pancreatic cancers. The therapy is being studied in combination with PARP (poly ADP-ribose polymerase) inhibitors, a $10 billion drug class that extends life for many HRD patients but often loses effectiveness within months.

“When you put the two together, you get unprecedented synergy in preclinical models,” Parikh says. “If patients could stay on therapy for years instead of months, just imagine the impact on their lives.”

Based in Boston, MOMA thrives on the region’s unique ecosystem of world-class science, venture capital, and talent. “The quality of the talent we have at the company is exemplary, and I think the talent in the Boston area is second to none,” Parikh says.

Editing RNA to treat disease

Some teams are going deeper into human cell biology by editing the RNA instructions themselves.

In 2012, the same year the gene-editing technology CRISPR was first published, one of AIRNA’s founders pioneered RNA editing. His lab showed it was possible to guide ADAR, a natural enzyme in our cells, to make precise RNA edits using an oligonucleotide drug. ADAR is already active in humans, making small RNA edits as a part of antiviral defenses.

“I kind of think of it as the organic version of editing,” AIRNA CEO Kris Elverum says.

AIRNA, headquartered in Cambridge with labs in Germany, has built a diverse, global team united by the mission of using RNA editing to treat both rare and common diseases. Its first medicine, AIR-001, targets alpha-1 antitrypsin deficiency, a genetic liver disease that affects about 100,000 people in the US. A single RNA “letter” error prevents patients from making a key protein, often leading to emphysema and liver disease. Current treatments require weekly plasma infusions that don’t address the root cause. AIR-001 aims to fix the error in the patients’ own liver cells, allowing them to make the correct protein naturally.

“One of the things that’s really exciting for me and for the patient community is that we should be able to get a sense pretty quickly of how well this works,” says Elverum. “When we correct that single letter, the cell immediately makes the right protein.”

AIRNA expects to begin phase 1 clinical trials of AIR-001 in early 2026.

And the promise of RNA editing goes beyond one disease.

“As a species, one of the most powerful things we’ve developed is the ability to change the letters in the genome,” Elverum says. “Long term, RNA editing lets us not just repair harmful mutations, but also learn from the variants that give people superior health and share those benefits more widely.”

“As a species, one of the most powerful things we’ve developed is the ability to change the letters in the genome.”
– AIRNA CEO Kris Elverum

Accelerating drug discovery with AI

Generate Biomedicines is harnessing generative AI to create protein therapeutics from scratch.

“Generative biology is all about using machine learning at scale to understand the principles of biology,” says CEO Mike Nally. “With the power of these technologies, we’re now able to disentangle complexity and recombine it to create novel therapeutics in ways we couldn’t have done before.”

One of the field’s biggest breakthroughs is de novo protein design, using computers to generate entirely new proteins that bind to biological targets without borrowing from nature. 

“Rather than trial and error, we’re moving drug discovery into a more deterministic, engineerable domain,” Nally explains. “That means higher-quality medicines at lower cost, discovered far faster.”

Generate’s first programs focus on oncology, immunology, and infectious disease. Its most advanced therapy, an antibody for severe asthma, has completed early trials and is expected to enter phase 3 testing later this year. Instead of monthly injections or daily inhalers, the molecule is designed for dosing just once every six months or so. Generate has three programs in the clinic today and expects five by early 2026. The asthma program in particular highlights the speed of the platform: it moved from conception to Phase 3 in just four years.

“That’s about as fast as I’ve seen in my 20 years in the industry,” Nally says. “If you think about all the medical progress we’ve made over the past 100 years, I believe AI could enable a comparable leap in the next decade. That’s what gets us out of bed in the morning: the potential for massive impact on humanity.”

“If you think about all the medical progress we’ve made over the past 100 years, I believe AI could enable a comparable leap in the next decade. That’s what gets us out of bed in the morning: the potential for massive impact on humanity.”
– Generate Biomedicines CEO Mike Nally

Restoring youthful cell function

Push further still, and you can reach the software of aging: the epigenetic code.

Life Biosciences, co-founded by Harvard Medical School professor David Sinclair, pioneers cellular rejuvenation therapies to reverse and prevent age-related diseases. The company focuses on partial epigenetic reprogramming.

When an organism is young, their epigenetic code — chemical markers on DNA that regulate gene activity — keeps genes properly switched on or off so the right proteins are made and cells function smoothly. As the code becomes disorganized with age, genes misfire, contributing to age-related diseases.

The company is working to reverse this process using OSK, a set of transcription factors — gene-controlling proteins — that can reset the code and restore youthful cell function. Its current focus is on diseases of the eye: glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION), a sudden loss of vision in one eye. Together, glaucoma and NAION are the most common chronic and acute causes of vision loss in older adults, yet neither has treatments that protect or restore the retinal ganglion cells at the root of the conditions.

Preserving sight could help individuals maintain independence, activities, social connection, mental health, and cognitive acuity. “Fixing vision has this potentially tremendous value for aging,” says Sharon Rosenzweig-Lipson, chief scientific officer at Life Biosciences.

Life Biosciences has already demonstrated OSK can safely improve visual signals, retinal structure, and epigenetic markers in non-human primates. Now it’s preparing to launch its first human clinical trial in the first quarter of 2026. The treatment, called ER-100, uses a dual-vector system delivered by a single injection into the eye. OSK remains “off” until patients take an oral pill to switch it on, giving doctors precise control over the treatment.

This innovation is just the beginning, Rosenzweig-Lipson says. “What we can do in the eye, we can do elsewhere, and we’re super excited about that data.”

For all the science and technology in the Boston area, what drives its biotech community is something more human. “People here are resilient, creative, and eager to tackle hard problems,” says MOMA’s Parikh, “and all of it is motivated by helping patients.”