Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging trends” in efficacy.
Kygevvi is indicated for patients with thymidine kinase 2 deficiency whose symptoms arise by 12 years of age. The disease manifests as muscle weakness and can become life-threatening in severe instances.
BioSpace has named 50 life sciences companies to its 2026 Best Places to Work list. AbbVie, Amneal Pharmaceuticals and BridgeBio executives share what makes their organizations special.
Less than 24 hours after resigning his post as the FDA’s head drug regulator, George Tidmarsh is reportedly reconsidering his decision and vowing to fight for his name and credibility amid a probe into his “personal conduct” at the agency.
At the center of the licensing deal is an NLRP3 inhibitor that has shown “encouraging efficacy in acute inflammation models,” according to TransThera, indicating its potential in various metabolic and inflammatory diseases.
Manifold will use its tissue-targeting shuttle technology to help Roche develop new therapeutics for diseases of the central nervous system.
FEATURED STORIES
Amid a season of regulatory and scientific advances, experts reveal a culture of data hoarding among cell and gene therapy developers that is reinforcing fragmentation, stalling innovation and delaying access to treatments.
Earlier this summer the FDA asked Moderna for more efficacy data on its flu vaccine before it could review an mRNA-based combination shot that targets both influenza and COVID-19. Now, the entire vaccine sector is sizing up a new regulatory world, companies’ next steps uncertain.
Participants in trials of BrainStorm Cell Therapeutics’ NurOwn filed a Citizens’ Petition with the FDA earlier this month seeking a new review of the stem cell therapy that was rejected in 2022 based on real-world data and 90% survival in an expanded access program.
From Wall Street to real estate to a “big, ugly” pharma building, Mayo Venture Partner Audrey Greenberg reflects on a career defined by taking a leap at just the right moment.
Some of the most high-profile acquisitions in recent years have involved women-fronted biotechs. BioSpace reviews five of the most notable here.
Seven biotech unicorns are advancing AI-powered drug discovery and development—but must contend with a difficult investing environment where competition is steep and the usual roads to exit are uncertain.
LATEST PODCASTS
In this episode, presented by the Genscript Biotech Global Forum 2025, BioSpace’s Head of Insights Lori Ellis talks to Tom Whitehead, co-founder of the Emily Whitehead Foundation, about how standard care, cell and gene therapies and their impact on patients.
Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees more action with new company launches, IPOs and fresh data; and experts get ready for an important era in the Duchenne muscular dystrophy space.
Biopharma executives shared their thoughts on the potential impacts of the new administration; Annalee Armstrong recaps JPM and her talks with Biogen, Gilead, Novavax and more; Wegovy’s higher dose induces more weight loss; AstraZeneca and Daiichi Sankyo’s Dato-DXd scores its first FDA approval.
Job Trends
Kite, a Gilead Company, announced results from three new analyses for Yescarta® in relapsed/refractory large B-cell lymphoma, including both new clinical research and real-world evidence highlighting manufacturing and product characteristics of Yescarta, and outpatient administration of both Yescarta and Tecartus® at the 2024 European Hematology Association Annual Congress, June 13-16, Madrid.
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SPECIAL EDITIONS
In this deep dive, BioSpace investigates China’s rise as a biotech powerhouse.
In this deep dive, BioSpace explores the next big thing in obesity.
BioSpace did a deep dive into biopharma female executives who navigated difficult markets to lead their companies to high-value exits.
DEALS
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The pharma giant inked its third T cell engager deal of 2025 Wednesday—this time with Xilio Therapeutics for tumor-activated immunotherapies. -
Just a few months after Vir Biotechnology lost an emergency authorization for its COVID-19 antibody, Marianne de Backer stepped in as CEO to answer a critical question: What’s next? -
Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies. -
M&A was already on the upswing in 2024, and the new Trump administration may support that trend. But if data aren’t handled properly, acquisitions won’t reach their full potential. -
With just one asset in weight loss moving through the clinic, Pfizer targets the space for potential dealmaking, as well as bringing assets over from China.
WEIGHT LOSS
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FDA Commissioner Marty Makary talks about his plans to revamp drug development and reduce ‘conflicts of interest’ between the agency and pharma industry; Roche and Regeneron jump on the U.S. manufacturing train as Trump’s tariffs loom; and Eli Lilly scores a big win for orforglipron while Novo Nordisk reveals it has applied for FDA approval of its oral semaglutide. -
Novo Nordisk filed for approval of an oral, 25-mg formulation of its weight loss blockbuster “earlier this year,” according to a company spokesperson. -
President Donald Trump in February threatened top pharma leaders, including Eli Lilly CEO David Ricks, with tariffs unless they reshore their manufacturing operations. -
Eli Lilly’s shares shot up 11% pre-market on Thursday after orforglipron became the first small-molecule GLP-1 drug to ace a late-stage study in type 2 diabetes, eliciting significant reductions in body weight and improvements in glucose control. -
Donald Trump takes biopharma on a tariff-themed rollercoaster ride; J&J kicks off the Q1 earnings season; experts express concern about the FDA’s future; Pfizer’s obesity setback could be Viking’s gain; and BioSpace reveals the highest paid pharma CEOs.
POLICY
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As an office of the executive branch, the Department of Health and Human Services “does not have the authority” to implement sweeping changes to the structure of the agency as created by Congress, a judge wrote. -
Kennedy wants to expand the injury compensation program to include COVID-19 vaccines, while also stretching the “statute of limitations” to more than three years. -
In an open letter addressing the Trump administration’s proposed budget cuts to HHS, the executives urged Congress to continue “robust federal funding” for scientific research, which they say will help maintain U.S. biotech leadership globally. -
The revamped and “more anti-vax skewed ACIP committee” at the CDC “has a bone to pick with mRNA vaccines,” according to Truist Securities analysts. Meanwhile, the FDA moves forward on having Pfizer/BioNTech and Moderna update labels for their COVID vaccines. -
Susan Monarez, already acting director of the CDC, said during her confirmation hearing that she sees no causal link between vaccines and autism.
Whether a job search is voluntary or not, months of interviews, wrangling over pay and benefits and evaluating options has a weighty psychological toll.
The role of a clinical research nurse can be rewarding, but it’s not without challenges. Find out more about the role of a clinical research nurse and what it takes to become one in our guide.
Find out everything you need to know about research and development, including the skills it requires and what you should do if you’re ready to make the transition into R&D.
At the foundation of every clinical trial are the professionals who ensure its successful execution. Read on to find out more about how to know if clinical research is right for you.
Q1 is the time when many employers are actively recruiting new talent. Because it takes an average of 60 days to fill a job opening, Q4 might be the best time to apply for jobs in the life sciences.
Being laid off from your job can be difficult and confusing. To help you in your job search, we’ve explained how to address a layoff in your cover letter to help you land your dream job.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
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At the intersection of radiation and precision, Novartis, Bayer, AstraZeneca and more hope to cash in on a radiopharmaceuticals market that could top $16 billion by 2033. -
After losing its powerhouse partner, IGM Biosciences closed “most” of its labs and offices and initiated a strategic review of potential strategic alternatives and options for the business. -
The cell engineering company, co-founded by oncologist and writer Siddhartha Mukherjee, does not see a path forward for its pipeline of early-stage cell therapies for two different types of cancer. -
The Massachusetts biotech will focus its efforts and resources into cemsidomide, an oral drug candidate being trialed for multiple myeloma and non-Hodgkin lymphoma. -
Keytruda is set to lose exclusivity in 2028, meaning Summit may face competition from cheaper biosimilars. Meanwhile, other branded drugmakers are also seeking to improve on the blockbuster checkpoint inhibitor.
NEUROSCIENCE
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Despite a dip in sales and a recent schizophrenia stumble, the company drew an optimistic outlook for sales for the rest of the year, even as the specter of pharmaceutical tariffs looms. -
With a new raise provided by Flagship Pioneering, the new company is aiming to find “the silent window” before disease symptoms set in. -
Cobenfy’s late-stage flop is BMS’ second high-profile failure in as many weeks. The pharma announced last week that Camzyos was unable to improve disease burden in non-obstructive hypertrophic cardiomyopathy. -
The Health and Human Services Secretary said that he will find and eliminate the cause of autism by September, an idea that suggests how little he knows about the condition. -
Jefferies analysts predict Annexon’s tanruprubart could be approved by mid-2026.
CELL AND GENE THERAPY
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Adaptimmune is rolling out its T cell therapy Tecelra for synovial sarcoma, recording $1.2 million in sales since its approval in August 2024. Nevertheless, it is pausing development of two oncology assets to save money. -
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do. -
The biotech is exploring opportunities for a reverse merger or other business combinations. CFO and now interim CEO Anup Radhakrishnan will take charge of these negotiations. -
After a patient taking the Duchenne muscular dystrophy gene therapy Elevydis died of liver injury, Sarepta will update the label to reflect the safety signal. -
The gene therapy world is in turmoil, but Arbor, armed with more than $1 billion in partnerships and raises, is going forward.
