Regulatory

Gen1e Lifesciences receives US FDA designations for GEn-1123

Tue, 09 Jun 2026 09:00:00 -0400

Gen1e Lifesciences Inc. has obtained both orphan drug and rare pediatric disease designations from the U.S. FDA for GEn-1123 for the treatment of Duchenne muscular dystrophy (DMD).

Moderna’s mRNA-4194 cleared for clinic for Lynch syndrome

Tue, 09 Jun 2026 09:00:00 -0400

The University of Oxford and Moderna Inc. have announced authorization by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study of mRNA-4194, Moderna’s investigational mRNA-based cancer vaccine for Lynch syndrome.

China deals highlight biopharma evolution, reports LEK

Tue, 09 Jun 2026 08:00:00 -0400

In 2025, China’s outbound life sciences and biopharma deals reached nearly $100 billion, about 12 times the level seen in 2021, underscoring how quickly global appetite for Chinese assets has accelerated, according to a recent report by L.E.K. Consulting.

Competitive approach better than restricting deals with China?

Mon, 08 Jun 2026 12:00:00 -0400

The recent introduction of the bipartisan Biotech Investment National Security Act in the U.S. House has industry and venture capitalists urging lawmakers to take a breath, step back, and consider a less invasive approach than restricting U.S. biopharma deals with Chinese innovators.

China’s NMPA clears Hansoh’s HS-10541 for clinic

Mon, 08 Jun 2026 09:00:00 -0400

Hansoh Pharmaceutical Group Co. Ltd. has obtained clinical trial approval from China’s National Medical Products Administration (NMPA) for HS-10541 tablets.

Amphista’s BRD9 degrader gains IND clearance for AML

Mon, 08 Jun 2026 09:00:00 -0400

Amphista Therapeutics Ltd. has obtained IND clearance from the FDA for AMX-883, an orally bioavailable degrader of BRD9, for the treatment of acute myeloid leukemia (AML). A phase I trial in patients with relapsed or refractory AML and high-risk myelodysplastic syndrome is expected to begin in the second half of this year.

FDA gets earful on CNPV pilot

Fri, 05 Jun 2026 12:00:00 -0400

The U.S. FDA’s year-old Commissioner’s National Priority Voucher (CNPV) pilot program played to mixed reviews at the agency’s June 4 listening session intended to get various stakeholders’ perspective on the ultra-accelerated review process being offered to qualifying drugs.

SCOTUS breathes new life into skinny labels

Thu, 04 Jun 2026 12:00:00 -0400

The U.S. Supreme Court handed Hikma Pharmaceuticals and the entire generic industry a big victory June 4 with its 9-0 opinion ensuring the future of skinny labels and correcting the Federal Circuit’s flawed understanding of infringed inducement.

Sotio Biotech’s SOT-106 designated orphan drug for osteosarcoma

Thu, 04 Jun 2026 09:00:00 -0400

Sotio Biotech AS’s SOT-106 has been granted orphan drug designation by the FDA for the treatment of osteosarcoma. SOT-106 is a next-generation antibody-drug conjugate targeting leucine-rich repeat-containing 15 (LRRC15), a clinically validated target broadly expressed across sarcoma subtypes and in tumor-associated stroma.

Rubio reopening door with Gavi

Wed, 03 Jun 2026 12:00:00 -0400

Nearly a year after Health and Human Services Secretary Robert Kennedy announced the U.S. was cutting off funding for Gavi, a global vaccine alliance, Secretary of State Marco Rubio said his department is reengaging with the organization in light of the ongoing Ebola outbreak in central Africa. In testifying before a June 2 Senate Foreign Relations Committee hearing, Rubio said the State Department made the decision to reengage a few weeks earlier with Gavi. He provided no detail of what that engagement looks like.

CSPC’s SYS-6063 gains Chinese trial clearance for SLE

Wed, 03 Jun 2026 09:00:00 -0400

CSPC Pharmaceutical Group Ltd. has obtained clinical trial clearance from China’s National Medical Products Administration (NMPA) for SYS-6063, an mRNA-LNP-based dual-target CAR T-cell injection for the treatment of relapsed or refractory systemic lupus erythematosus (SLE).

BMS-Hengrui deal strikes panic in Washington

Tue, 02 Jun 2026 12:00:00 -0400

On the heels of the multibillion-dollar licensing deal between Bristol Myers Squibb Co. and Jiangsu Hengrui Pharmaceuticals Co. Ltd., Chairman John Moolenaar (R-MI), of the House Select Committee on the Chinese Communist Party, sent a letter to Treasury Secretary Scott Bessent urging him to add biotechnology as a prohibited technology under the Comprehensive Outbound Investment National Security (COINS) Act of 2025.

China launches long-awaited drug data protection rules

Tue, 02 Jun 2026 12:00:00 -0400

China’s National Medical Products Administration has issued final implementation measures for drug trial data protection that mark a significant step toward aligning China’s pharmaceutical intellectual property framework with global standards.

Fulcrum drops work on SCD drug pociredir; stock plummets

Tue, 02 Jun 2026 12:00:00 -0400

Shares of Fulcrum Therapeutics Inc. (NASDAQ:FULC) fell 54% after the company said it is discontinuing work on sickle cell disease (SCD) candidate pociredir, its only clinical-stage candidate, and reviewing strategic alternatives in the wake of the U.S. FDA’s safety concerns regarding the drug target.

FDA advises leveraging what’s already known in gene therapy R&D

Tue, 02 Jun 2026 12:00:00 -0400

Rather than reinventing the wheel for every gene therapy that uses genome editing, the U.S. FDA is advising sponsors on leveraging existing knowledge, be it publicly available or platform-based, to more efficiently advance their products across multiple stages of development.

FDA approves IND for Iovance’s TIL therapy IOV-5001

Tue, 02 Jun 2026 09:00:00 -0400

Iovance Biotherapeutics Inc. has received IND clearance from the FDA for IOV-5001, a next-generation interleukin-12 (IL-12)-tethered tumor-infiltrating lymphocyte (TIL) therapy.

Voyager’s tau-targeted gene therapy VY-1706 gains IND approval

Tue, 02 Jun 2026 09:00:00 -0400

Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.

FDA clears Shionogi’s Xocova as first COVID-19 prevention pill

Tue, 02 Jun 2026 08:00:00 -0400

The U.S. FDA approved Shionogi & Co. Ltd.’s Xocova (ensitrelvir) as the first oral post-exposure prophylactic option in the U.S. to prevent COVID-19, with the decision coming ahead of a PDUFA target date of June 16.

Policy, aka politics, to have bigger role in US grants?

Mon, 01 Jun 2026 12:00:00 -0400

The Trump administration’s efforts to ensure U.S. federal grants align with its policies may soon be coming to fruition. The White House Office of Management and Budget released a proposed rulemaking to revise its Guidance for Federal Financial Assistance.

China launches long-awaited drug data protection rules

Fri, 29 May 2026 12:00:00 -0400

Replimune tries again with melanoma drug post FDA shakeup

Fri, 29 May 2026 12:00:00 -0400

Industry watchers will be tracking closely Replimune Group Inc.’s latest attempt to secure U.S. FDA approval of its advanced melanoma candidate, RP-1 (vusolimogene oderparepvec). The company announced Friday it had reached alignment for resubmitting the twice-rejected BLA with the agency, which it said has pledged to prioritize the review.

BMS-Hengrui deal strikes panic in Washington

Fri, 29 May 2026 09:00:00 -0400

FDA accepts IND for Rest Therapeutics’ early PTSD candidate

Fri, 29 May 2026 09:00:00 -0400

The FDA has accepted the IND application from Rest Therapeutics SAS for RST-101, the company’s lead investigational candidate for the early treatment of post-traumatic stress disorder (PTSD).

TROP2 ADCs progress into first-line for lung, breast cancers