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Intellia Therapeutics reported early, positive signs from its in vivo gene therapies for both transthyretin (ATTR) amyloidosis and hereditary angioedema Friday.

AstraZeneca’s rare disease subsidiary Alexion posted positive late-stage results from a potential complementary treatment for paroxysmal nocturnal hemoglobinuria.

The global cerebral palsy treatment market size is expected to reach USD 2.23 billion by 2025, according to laetst study by Emergen Research, expanding at a CAGR of 7.0% during the forecast period.

The U.S. FDA approved Mallinckrodt’s Terlivaz in adults with hepatorenal syndrome and is the only FDA-approved drug for that indication.

Becoming a chemist in the biopharma industry takes hard work, dedication and a willingness to continuously learn. Here’s what you need to know to become a biochemist.

Palisade Bio is laying off 20 percent of its workforce, the company announced Thursday. The cost-saving measures are expected to amount to over $1.5 million per annum.

The Lancet Commission published a lengthy criticism of the WHO’s COVID-19 pandemic response. In turn, the WHO offered a reply to a number of the complaints.

Neuronal Inflammation was found in two U.S. monkeypox patients, researchers short-list four drugs for potential study in autism and a new test can predict immunotherapy outcomes.

Five patients with systemic lupus erythematosus (SLE) are in remission after receiving CAR T cell therapy, according to a recent study.

BrainStorm Cell Therapeutics announced the publication of peer-reviewed data from a Phase II trial in progressive multiple sclerosis Thursday showing “preliminary evidence of efficacy.”

Biopharma and life science companies added changes to their leadership teams this week, with Cara Therapeutics, SOTIO Biotech, Agenus, and more filling several C-suite positions.

The Sept. 30 deadline for Congress to reauthorize the FDA’s prescription drug user fee is looming, while advocates and detractors are debating the merits of the funding source.

Expanded sweetener molecule development program aims to broaden access to plant-based sweetening solutions tailored to diverse customer food and beverage categories.

• $5.5 million financing that closed on April 2, 2026 together with $5.7 million in capital raised in Q1-2026, expected to provide Company with cash runway into 2027 • $4.5 million of accrued VAT refunds and R&D refundable tax credits from UK subsidiary begin maturing, including $1.9 million VAT cash refund expected in June 2026 • Successful EMA and Australia TGA (ATGA) meetings aligned around use of existing biomarker and pending release of 3-year overall survival data to support Conditional Marketing Authorisations (CMAs) for OST-HER2 in prevention or delay of recurrence in fully-resected, pulmonary metastatic osteosarcoma (“Metastatic Osteosarcoma”) • 2.5-year overall survival data expected to be released during ASCO 2026 • EMA and ATGA have aligned on key design aspects of confirmatory Phase 3 Metastatic Osteosarcoma protocol required to have commenced prior to grant of CMAs or FDA BLA, including alignment on 3-year overall survival as a primary clinical efficacy endpoint • Upcoming U.S. FDA Type C meeting and U.K. MHRA SAM meeting to align with EMA & ATGA prior to Australia Clinical Trial Notification submission for Phase 3, including alignment on 3-year overall survival as a primary clinical efficacy endpoint. U.S. FDA (“FDA”) Pre-BLA meeting to confirm alignment on surrogate and primary clinical efficacy endpoints with EMA/ATGA, including alignment on 3-year overall survival as a primary clinical efficacy endpoint, and seek decisions on rolling review acceptance and RMAT designation following the EMA and U.K. MHRA granting ATMP designation (EMA/MHRA RMAT equivalent) for OST-HER2 in Metastatic Osteosarcoma • OS Animal Health S-1 filing for proposed ‘Go-Public’ transaction and receipt of SEC comments