In addition to safety questions about its COVID-19 vaccine, Johnson & Johnson (J&J) is facing manufacturing challenges as a key partner, Emergent Biosolutions Inc., has yet to get FDA approval to manufacture or distribute the vaccine or its components in the U.S. That approval could be awhile in coming. On April 21, a day after completing its investigation of Emergent’s Bayview plant in Baltimore, Md., the FDA released the Form 483 from the inspection, showing nine observations related to cross-contamination issues, lack of employee training and poorly maintained facilities. During the nine-day inspection, the FDA investigator noted peeling paint and residue on floors and walls and saw employees throwing unsealed bags of “special medical waste” into a service elevator in the manufacturing area.

Business as usual: Versant raises $950M for three new funds

Versant Ventures has closed three new funds with an aggregate raise of $950 million to allocate to the next wave of innovative start-ups across North America and Europe. The money is new, but everything else is more or less unchanged. “It’s the same strategy, same team, same geography, same operational model,” Versant chairman and managing director Brad Bolzon told BioWorld.

Gene therapy continues to attract investors

The development of gene therapy has come a long way over the past two decades after getting off to a very rocky start following the death of a young patient as a result of being treated with an experimental therapy. Since that time continuing scientific progress has enabled the development of a robust product pipeline of promising therapies that could lead to, according to FDA estimates, 10 to 20 cell and gene therapy products a year within the next five years. The renaissance of the sector has also attracted record amounts of investment capital and significant business development.

In the wake of dashed hopes for a Huntington’s therapy, efforts continue

Two more soldiers in the campaign for a viable Huntington’s disease (HD) treatment fell to trial failures in March. Roche Holding AG quit dosing its phase III Generation HD1 study with antisense therapy tominersen, licensed from Ionis Pharmaceuticals Inc., based on a data monitoring committee’s findings. A week later, Wave Life Sciences Inc. rolled out data from the Precision-HD2 and Precision-HD1 phase Ib/IIa trials that tested the mRNA-targeting antisense oligonucleotides WVE-120102 and WVE-120101, respectively. Precision-HD2’s core study showed no dose responses; the open-label extension turned up inconsistent results. Precision-HD1 results proved similar, so Wave stopped development of both candidates. Last September, Vaccinex Inc. said its SEMA4D inhibitor pepinemab showed encouraging trends but missed statistical significance in the phase II effort called Signal, but the firm is continuing to work with the compound and hopes to find a partner for phase III. An array of players remain busy in the potentially lucrative HD space.

Edigene secures $62M in series B+ to advance hematopoietic stem cell therapy

Genome editing startup Edigene Inc. closed a ¥400 million (US$62 million) series B+ round to expand its operation to more cities and advance its lead product ET-01 into clinical trials. The financing follows the $67 million series B that closed in October 2020.

Peptron signs global rights for ADC candidate to Qilu

HONG KONG – Qilu Pharmaceutical Ltd. has inked a deal for the global rights to Peptron Inc.’s antibody-drug conjugate (ADC) candidate PAb001-ADC for the treatment of various cancers. Seoul, South Korea-based Peptron stands to receive an up-front payment of $3 million and $539 million in milestone payments, as well as royalties. “These funds will help us to focus on the development of other antibody moieties as well as new molecular entity programs,” a Peptron spokesman told BioWorld.

Also in the news

Agenebio, Allogene, Alterity, Antios, Apic, Atriva, Ayala, Bharat, Biomea, Brii, Chiesi, China Biologic, Claritas, Cohbar, Edigene, Enzychem, Evelo, Excellthera, Hemoshear, Humacyte, Kadmon, Kancera, Khondrion, Marinomed, Moderna, Morphosys, Nasus, Novo Nordisk, Oasmia, Ocugen, Oncotelic, Organicell, Ossium Health, Pharvaris, RDIF, Recursion, Retrotope, Revolution, TG, Trevena, Vaccinex, Valneva, Willow