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Astel­las signs on pluripo­tent stem cell part­ners at Ex­CellThera; Lit­tle Uni­cy­cive brings home $25M af­ter float­ing a small IPO

Astellas has signed up on rights for a batch of pluripotent stem cells from Montreal-based ExCellThera, centering on the in vitro use of their UM171 compound.

Astellas is handing over an unspecified upfront payment and promising a slate of milestones to get the collaboration underway.

Guy Sauvageau, CEO and founder of ExCellThera, noted:

While ExCellThera has focused on developing our core strength in other therapeutic areas, Astellas has invested significantly in PSC-related programs and is absolutely the right partner for us in that regard.

WHO is­sues new guide­lines for gene edit­ing; Doud­na-backed Cari­bou launch­es its first al­lo­gene­ic CAR-T tri­al

Three years after news of the birth of two gene-edited babies shocked ethicists, regulators and biologists around the world, World Health Organization experts called for the establishment of a new whistle-blowing mechanism to help prevent future unsafe or unethical uses of CRISPR and other gene editing technologies.

The recommendation was one of a series the WHO panel issued Monday in a pair of reports designed to guide global governance of gene editing. The panel was convened in December 2018, weeks after Chinese scientist He Jiankui announced two babies were born from embryos he edited, and follows a similar effort from the International Commission on the Clinical Use of Human Germline Genome Editing, which released a series of recommendations last year.

With TransCode go­ing pub­lic, in­dus­try sur­pass­es $10B IPO mark; Galect­in's in­hibitor shows PhIb suc­cess in can­cer treat­ment

TransCode Therapeutics has priced its IPO at $4 a share, and while that number might be on the smaller end of IPOs this year, it signifies the passing of the $10 billion mark for the industry.

With $10 billion raised in public offerings so far this year, the biotech market is well on track to surpass its record totals for last year. (For more on biotech’s red-hot IPO space, bookmark our tracker here.)

Bridge­Bio adds 3 more in­sti­tu­tions to its ros­ter of col­lab­o­ra­tors; Ex­sci­en­tia lands Gates Foun­da­tion mon­ey for Covid-19 pill 

BridgeBio chief Neil Kumar is adding to his long list of research partners.

The biotech CEO announced this morning that the company’s hunt for new drugs to treat genetic diseases and cancers with clear genetic drivers has sparked new alliances with researchers at Stanford, MUSC Foundation for Research Development and the University of Pittsburgh.

BridgeBio — which employs a hub and spoke company structure — counts 23 institutions on its list of collaborators. That appears to be a work in progress, with more on the way. — John Carroll

Ar­row­head touts Phase Ib RNAi da­ta for ccR­CC; PureTech lines up PD-1 from BeiGene for up­com­ing com­bo study

Just days after getting walloped by the news that animal tox data forced them to halt an early-stage cystic fibrosis study, Arrowhead is touting some initial snapshots from their RNAi treatment for clear cell renal cell carcinoma.

Most of the positive signs they’re pointing to relate to biomarker activity, with an average 48% reduction in HIF2α protein among nine evaluable biopsy samples. Those reductions varied widely, though, starting at 9% and growing to 82%.

Chemo­Cen­tryx gets new PDU­FA date af­ter ma­jor amend­ment; Nim­i­um launch­es with eye on obe­si­ty

Following talks with the FDA, ChemoCentryx on Monday filed an amendment to its application for avacopan as a treatment of anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, resulting in a new PDUFA goal date of Oct. 7.

While the company did not explain what was contained in the amendment filing, its stock spiked by more than 10% on the news. How the agency will ultimately decide on the drug remains a mystery. The agency’s Arthritis Advisory Committee in May voted 9-9 on whether the efficacy data from a small Phase III trial support approval of avacopan, 10-8 in favor of the drug’s safety profile, and 10-8 that the benefit-risk profile is adequate to support approval.

Sar­to­rius buys ma­jor­i­ty stake in Ger­man man­u­fac­tur­ing play­er; Cerev­el Ther­a­peu­tics ends the week on a high note, with $350M raise

Sartorius Stedim Biotech acquired a majority stake in Germany-based CellGenix GmbH, the companies announced Friday.

Sartorius has acquired 51% of the company for $118.3 million and announced the plan to acquire the remaining shares of the company at the beginning of 2023 and 2026.

CellGenix was started at the University Medical Center of Freiburg in 1994 and currently employs 70 people. The company generated $23.6 million in sales in 2020.

Kin­tara of­fers peek at mid-stage da­ta in re­lapsed glioblas­toma pa­tients; Jazz wins quick ap­proval for pe­di­atric can­cer drug

Among the dozens of cancer types, glioblastoma has proven one of the hardest to crack with little clinical success over the years. Now, a San Diego biotech is rolling out mid-stage data it claims could be pointing its drug in the right direction in relapsed patients. But there are several caveats.

A high dose of Kintara’s VAL-083 spurred a median OS of 8 months in patients with recurrent glioblastoma who had been previously treated with chemotherapy temozolomide, according to topline data from an open-label Phase II study conducted at MD Anderson Cancer Center in Houston.

3 gene ther­a­py com­pa­nies se­cure VC mon­ey; No­valis clos­es near­ly $30 mil­lion fund

A European life sciences VC firm will invest in three more startups specializing in rare genetic diseases as a part of its Sofinnova Telethon Fund, the company announced Wednesday.

AAVantgarde Bio, Alia Therapeutics and Borea Therapeutics will receive seed funding worth a total of $7.1 million. The fund is dedicated to biotechs in Italy, and led by Lucia Faccio and Paola Pozzi, Partners at Sofinnova Partners.

Io­vance's TIL ther­a­py makes ear­ly head­way in lung can­cer; An­tios and Ar­bu­tus join hands on HBV com­bo treat­ment

Iovance has posted a first look at its tumor infiltrating lymphocytes as a treatment for non-small cell lung cancer, paving the way for a pivotal study.

Among 28 patients enrolled in Cohort 3B of the IOV-COM-202 basket study — 24 of whom were evaluable — investigators recorded an overall response rate of 21.4% at the interim, with 1 complete response and 5 partial responses. All patients in the group had progressed on prior immune checkpoint inhibitor therapy, and some had received tyrosine kinase inhibitors. The six responders also underwent chemotherapy.