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
Analysts believe that Gilead’s new PrEP drug Yeztugo could reach peak sales of $4.5 billion. Not if GSK has anything to say about it.
Cell and gene therapy leaders say the agency’s decision to remove the Risk Evaluation and Mitigation Strategies that had been attached to approved CAR T cancer therapies reflects “thoughtful consideration of real-world evidence” and “regulatory trust.”
Big Pharmas like Eli Lilly, Sanofi and Novartis headed back to the dealmakers table multiple times, with 32 total deals counted across the industry for the first half.
Changing how biopharmas package their products, how regulators review new drugs and how mutated genes are fixed could make ultrarare disease treatments possible.
The FDA delivered two notable approvals for RSV immunization, UroGen overcame a negative advisory committee vote to secure an approval in bladder cancer, and more key regulatory nods from the past month.
In the race to make the most tolerable obesity drug, there seems to be no clear winner—at least not according to analysts parsing the data presented at the American Diabetes Association annual meeting this week.
LATEST PODCASTS
GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis, Miguel Forte and Ali Pashazadeh discuss how a slow and steady pace is a continuation of the pattern we have seen throughout the last three years.
Novartis, Gilead, Roche and Takeda commit to new partners in a spate of mid-sized collaborations this week. Meanwhile, Applied Therapeutics’ stock tanks 80% after govorestat is denied approval, Intra-Cellular Therapies seeks to expand Caplyta into major depressive disorder and the FDA investigates the safety of bluebird bio’s Skysona.
Job Trends
Eli Lilly and Company announced that Melissa Seymour will join the company as executive vice president of Global Quality and member of the company’s Executive Committee, effective July 22, 2024.
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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 deal follows a $1.06 billion U.S. contract in July 2024 and a $1.24 billion agreement with an Asia-based pharma a few months later. -
On the company’s Q4 earnings call where an eyepopping $88.8 billion in full-year sales were revealed, leaders shifted focus away from enormous takeovers to single-digit billion buy outs. -
With an eye toward advancing a novel antibody-drug conjugate for gastrointestinal cancers, ArriVent is the latest biopharma player to ink a deal with a Chinese biotech. -
Five years ago, Gilead signed a massive deal with Galapagos. After a restructuring, the pharma is still hunting for the potential it saw at the original signing. -
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.
WEIGHT LOSS
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Compounded versions could make up as much as 40% of the semaglutide market, said Novo Nordisk CEO Lars Fruergaard Jorgensen on Thursday, but the company hopes to win patients over. -
Lexicon’s LX9851 targets ACSL5, a liver enzyme involved in fat metabolism that helps moderate fat accumulation and slow down gastric emptying. -
Deloitte urged pharma executives to “be bold” in a new report tracking the top 20 pharmaceutical companies’ R&D performance. -
Eli Lilly and Novo Nordisk are in a global battle for dominance in the weight loss space. BioSpace takes a look at the territory covered and what’s to come. -
With crucial lessons learned from the manufacturing shortages of injectable GLP-1s, experts say securing adequate supply of the upcoming oral options will be the sector’s next great challenge.
POLICY
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District Judge William Young, a nominee of Republican President Ronald Reagan, blasted the Trump administration’s NIH cuts as discriminatory and “bearing down on people of color because of their color.” -
HHS Secretary Robert F. Kennedy Jr.’s actions in recent months have raised concerns that he is taking a heavy-handed and unilateral approach to vaccine policy in the U.S. -
At a satellite kickoff event to the annual BIO meeting, investment bankers and VCs gave reasons for optimism amid a ‘volatile’ period for the industry. -
Scientists and analysts express concern that the newly appointed ACIP members—which include known anti-vaxxers—could relitigate recommendations that have already been made. Many are imploring Sen. Bill Cassidy to step up. -
The rehired staff, who number around 460, work with the CDC’s viral disease prevention efforts and sexual health testing labs, among others. The reinstatements are a ray of light in an acrimonious week that also saw protests and the complete overhaul of the agency’s vaccine advisory committee.
Read on to learn about the different nursing positions available in the biopharmaceutical industry, as well as the skills and qualifications you need to be a successful biopharma nurse.
Finding a new job can be a daunting task. From updating your resume to preparing for interviews, there are a lot of moving parts. To help, here are five interview techniques that actually work.
BioSpace spoke with three CEOs: Alto Neuroscience’s Dr. Amit Etkin, Omega Therapeutics’ Mahesh Karande and Rain Therapeutics’ Avanish Vellanki about their companies’ employment growth.
The life science industry is growing rapidly, and many companies have announced expansions and job creation. Still, others have been forced to cut costs and slash jobs. For that and more, continue reading.
BioSpace spoke with Jay Johnson, the Director of Talent Acquisition for Orthopedics at Stryker, to find out what it takes to land a job in medical sales.
Since 2020, the life sciences industry has worked to rebound from the COVID-19 pandemic. But one sector’s growth has far outpaced the others–the biotech industry.
HOTBEDS
IN CASE YOU MISSED IT
Perfecting microbial manufacturing processes means taming its greatest asset: speed. This rapid process for creating biologics also means a race against time, where success hinges on conquering three key challenges: designing a robust microbial strain, optimizing fermentation for industrial scale, and ensuring consistent high-yield product yield.
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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The targeted drug release device TAR-200 shows promising response and disease-free survival rates in specific populations of patients with non-muscle-invasive bladder cancer. -
The FDA is currently reviewing Merck’s sBLA for Keytruda in head and neck cancer, with a target action date of June 23. -
The transaction is expected to close in the second half of 2025. With the deal, Merck KGaA is adding to its rare disease and oncology pipelines. -
With ivonescimab’s data coming solely from China, its prospects in the U.S., where Summit owns the rights, remain up in the air. -
Presentations at this year’s American Association for Cancer Research meeting could have a broad impact on the treatment landscape for head and neck and lung cancer, and implications for specific drug modalities like TIGIT and VEGF.
NEUROSCIENCE
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After some high-profile crashes, the one-time biotech darling is inching toward success with its Hunter syndrome treatment, which today began a rolling BLA for accelerated approval. -
Biopharma leaders react to the forced resignation of CBER Head Peter Marks as RFK Jr.’s promised job cuts begin at the FDA; Novo Nordisk presents mixed results from oral semaglutide in cardiovascular disease; the EU’s Committee for Medicinal Products for Human Use declines to recommend Eli Lilly’s Alzheimer’s drug; and pharma R&D returns grew in 2024.
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The European Union’s CHMP said that the benefits of the drug, already approved in the U.S., do not outweigh the risk of potentially fatal brain swelling and bleeding. -
WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to file for accelerated approval of the candidate in 2026. -
Eisai’s new fiscal 2027 forecast for Leqembi is roughly 50% lower than its projections a year ago.
CELL AND GENE THERAPY
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The companies were two years into a four-year, $400 million agreement aimed at developing and marketing gene therapies together. -
As FDA seeks to rehire some fired employees, Donald Trump threatens to enact tariffs on pharma companies unless they reshore manufacturing; another lawsuit hits the complex GLP-1 compounding space as Eli Lilly offers expanded Zepbound options; and struggling gene therapy biotech bluebird bio goes private in an attempt to stay solvent.
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At the 2025 National Biotechnology Conference, gene therapies, bispecific antibodies and other novel modalities—relative newcomers to medicine—will be much discussed. In this curtain raiser, BioSpace speaks with conference chair Prathap Nagaraja Shastri of J&J about these highly anticipated topics. -
The treatment, called DB-OTO, is one of several early-stage gene therapies being developed to treat relatively straight-forward causes of genetic deafness. -
The proposed acquisition by global investment firms Carlyle and SK Capital Partners could net shareholders $3 per share plus potential CVR dollars and provide bluebird bio with primary capital to expand the commercial reach of its gene therapies.
