AstraZeneca has invested heavily in AI, primarily through collaborations, including an up to $5.3 billion partnership with China’s CSPC Pharmaceutical in June.
This latest FDA program aims to provide speedier reviews for generic drugmakers who produce their products in the U.S.
Rocket Pharmaceuticals’ strategic realignment initiative in July pulled funding from fanca-cel, which the biotech was developing for Fanconi anemia.
The centerpiece of the collaboration is the gene editor ABO-101, being developed for primary hyperoxaluria type 1, a rare disease that leads to severe kidney stones.
Jeanne Marrazzo, former director of the National Institutes of Allergy and Infectious Disease, was formally terminated Thursday after months on administrative leave, after filing a whistleblower report.
Expanded exemptions for orphan drugs could mean prolonged protections for top-selling drugs like Merck’s Keytruda, which was initially approved under this designation in 2014.
FEATURED STORIES
While a substantial portion of pipeline assets are externally sourced, many Big Pharmas are tapping into incubators and venture funds to uncover cutting-edge scientific trends, determine their future focus points and even carve out a niche in an emerging geographical hotspot.
From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to make CGTs commercially viable. But it is possible, experts agreed at a recent panel.
The primary focus in scaling up production should first be the adoption of lean manufacturing principles used in virtually every other industry.
After decades of limited progress—owing to the difficulty of treating the disease and resultant market risk—glioblastoma research is entering a new phase spurred by smarter trials, targeted funding and renewed interest from companies like Merck and Jazz Pharmaceuticals.
Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy families have turned to the news for answers. Meanwhile, the FDA insists it remains committed to notifying companies of any regulatory action before sharing information with the media or public.
Against steep odds and well-established paradigms, these four companies have successfully been commercializing their products on their own.
LATEST PODCASTS
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.
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.
In this bonus episode, BioSpace’s vice president of marketing Chantal Dresner and careers editor Angela Gabriel take a look at Q1 job market performance, layoffs and administration decisions impacting the workforce.
Job Trends
Astellas Pharma’s new life sciences center houses its first U.S.-based SakuLab, an incubator space for external partners, as well as its engineered small molecules unit.
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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 Alchemab deal will further strengthen Lilly’s early-stage pipeline for amyotrophic lateral sclerosis, coming less than a year after the pharma licensed QurAlis’ antisense oligonucleotide to correct a specific protein alteration in ALS. -
After multiple rounds of layoffs that cut Kronos down to just 10 people, the small molecule biotech has accepted a buyout offer from Kevin Tang’s Concentra Biosciences. -
Announcing first-quarter results, Biogen CEO Chris Viehbacher admitted that tariffs are “a new topic for us,” but said he does not expect major impacts—at least for 2025. -
Many companies have foreshadowed deals to come during earnings calls in recent days. The return of M&A would be a welcome sign for the biopharma ecosystem, which has been battered by macro headwinds such as tariffs and the possibility of new drug pricing pressures. -
Policy uncertainties are impacting biopharma dealmaking from continent to continent, with companies being asked to walk a tightrope on their relations with China.
WEIGHT LOSS
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The overturning of the FDA’s lab-developed tests rule is just the tip of the iceberg. With the loss of Chevron deference, power has shifted from federal agencies to the courts, with potential implications for everything from the FDA shortage list to CMS drug price negotiations. -
HHS Secretary RFK Jr. removes the COVID-19 vaccine recommendation for healthy kids and pregnant women—the latest in a string of changes to vaccine policies; judge issues an order to halt HHS’ reorganization and mass layoff plans; Rocket Pharmaceuticals’ pivotal Danon disease trial is on hold after a patient death; and President Trump has named Mehmet Oz to spearhead his Most Favored Nation drug pricing policy. -
The CMS last month declined to include anti-obesity medications in its Medicare coverage for Part D, a move that Lilly says could interfere with patients getting the appropriate medical care. -
China continues to be a source of innovation as Pfizer strikes biggest pact yet; HHS provides more info on Trump’s Most Favored Nation executive order; FDA Commissioner Marty Makary and CBER director Vinay Prasad reveal new COVID-19 vaccine strategy following Novavax approval; ODAC underway after chaotic planning; more.
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Currently trailing Eli Lilly and Structure Therapeutics in the oral weight loss space, Novo Nordisk strikes a deal with Septerna to put new discovery-stage programs into play.
POLICY
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The Department of Health and Human Services is terminating around $500 million in BARDA contracts associated with mRNA vaccine development, a move that will affect several pharma companies, including Moderna, Pfizer, Sanofi and AstraZeneca. -
The regulatory environment is placing extreme pricing pressure on pharmaceutical manufacturers. Their success in the market depends on mounting an agile response. -
Albert Bourla confirmed that he called President Donald Trump after receiving a letter asking Pfizer and a clutch of other pharmaceutical companies to lower drug prices or face consequences. -
Earlier this year, the Centers for Medicare and Medicaid Services scrapped a previous proposal, from the Biden administration, to include anti-obesity medications in Medicare Part D coverage. -
The FDA has denied that it plans to combine the Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research into one entity.
Dry promotions include new titles and responsibilities without higher pay. What should you do if you’re offered this type of promotion?
By incorporating soft skills in your resume, you showcase your ability to work well with others, which is important to employers.
It’s important to quit your job the right way so you can exit on a positive note, which could benefit you in the future.
The FTC’s final rule banning most new noncompetes should go into effect later this year, although it faces opposition from the U.S. Chamber of Commerce.
By understanding the different types of difficult bosses, you can shift your perspective and approach the situation from a less stressful, more effective angle.
Despite the benefits of hiring foreign-born STEM employees, some companies avoid it largely due to unfamiliarity with the visa process, according to two recruitment experts.
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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In May, Revolution Medicines projected its cash and equivalents of $2.1 billion would last into the second half of 2027. With new funding from Royalty Pharma, the biotech has withdrawn that runway end date. -
In combination with Roche’s PD-L1 blocker Tecentriq, zanzalintinib bested Bayer’s Stivarga. Exelixis is positioning the drug candidate as a successor to cabozantinib, which is set to lose patent exclusivity in 2030. -
Venclexta, when combined with azacitidine, elicited an overall survival benefit below 10% in patients with myelodysplastic syndromes. -
The deal gets NextCure the rights to Simcere’s novel ADC for solid tumors outside of China. -
Analysts at Truist Securities called J&J’s CAR T readout “compelling,” noting that the efficacy figures could position the cell therapy as a formidable competitor to the current standard of care, Gilead’s Yescarta.
NEUROSCIENCE
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While ALTO-203 missed its depression-related endpoints, improvements in EEG biomarkers, attention and wakefulness point to signals of drug activity, William Blair said, though the analysts pointed to other indications as potentially more promising for future development. -
Although the company withheld detailed findings from the study of treatment-resistant depression, analysts at Stifel called COMP360’s efficacy “more than good enough” for registrational purposes. -
The U.K.-based biotech is set to enter mid-stage studies for its depression drug this year, while two other GABAA modulators are poised for clinical trials in 2026. -
Jefferies analysts said on Tuesday that Ventyx could leverage its mid-stage data for VTX3232 to position the oral drug candidate as a treatment for other neurodegenerative diseases, including Alzheimer’s. -
Analysts at Jefferies give Roche and Prothena’s Phase III study just a 25% to 40% probability of success.
CELL AND GENE THERAPY
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Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry. -
In a roundtable event on Thursday, HHS Secretary Robert F. Kennedy Jr. said his office will work to eliminate barriers that keep cell and gene therapies from the market. -
Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according to BMO Capital Markets analysts. -
The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug development and review process, particularly for rare diseases. -
J&J has a multi-year head start, but Gilead believes it can win market share by delivering a drug with better safety and at least as good efficacy.
