Had it been asked to, the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee likely would have voted yesterday to recommend approval of Vertex Pharmaceutical Inc.’s exagamglogene autotemcel, or exa-cel, as a one-time transformative treatment for severe sickle cell disease. But that’s not what the panel was asked to do, the FDA’s Nicole Verdun reminded the group when the conversation veered in that direction. Acknowledging that there’s no question of the efficacy of the autologous, ex vivo CRISPR/Cas9 gene-edited therapy or the significant unmet need for curative treatments in sickle cell, Verdun redirected the conversation to additional studies that could be conducted, especially in the postmarket setting, to tease out any safety concerns from potential off-target edits. Saying the off-target analyses Vertex conducted were reasonable, Alexis Komor, a gene editing expert on the panel, cautioned against “expecting perfection at the expense of progress.”

Astrazeneca makes an equity stake in Cellectis in $245M deal

Astrazeneca plc is making a $220 million equity investment and tossing in $25 million up front to Cellectis SA as part of a new collaboration agreement. The cash payments are part of a deal that sees Astrazeneca make a 22% equity stake in Cellectis while using its gene editing technologies and manufacturing prowess to develop cell and gene therapies. Ten candidates could be selected, with Cellectis in line to receive an IND option fee plus development, regulatory and sales milestone payments that could range from $70 million to $220 million. Cellectis’ stock (NASDAQ:CLLS) was surging strongly at midday, with shares trading 190% upward at $2.80 each.

$60M series A fuels Gate effort targeting extracellular proteins

Gate Bioscience Inc. made its debut with $60 million in series A money. Versant Ventures and A16z Bio + Health led the round. The firm is developing molecular gates, small molecules that eliminate disease-causing extracellular proteins. Arch Venture Partners and GV took part in the financing as well.

BCI flourishes despite widespread stock declines

The BioWorld Cancer Index (BCI) saw its highest point of the year from April through July, when it had a run above 30%, before falling to 23.5% at the end of August. It closed Q3 up 22.96% for the year, a sharp divergence from the more extensive Nasdaq Biotechnology Index and Dow Jones Industrial Average, which are tracking down 6.16% and up 1.09%, respectively. 

Israel tops up budget for struggling biotechs amid continuing war challenges

Israel’s finance ministry has increased the amount of funding high-tech companies with a short runway, including biotechs, can apply for under a fast-track scheme run by independent, publicly funded agency The Israel Innovation Authority, in response to the country’s war against Hamas. The program has been boosted by an extra ₪300 million (US$74.7 million), bringing the total budget to about ₪400 million, and from this month will assess applications from startups facing financial difficulties. The move reflects the raft of pressures the political situation in the country is placing on pharma and biotech firms as they grapple to continue doing business in an environment of conflict and uncertainties. 

Biolinerx shares climb after $270M+ motixafortide deal in Asia with Gloria Bio

Biolinerx Ltd. will hand off the rights to its stem cell mobilizer, motixafortide, in Asia to China’s Gloria Biosciences Co. Ltd. via an out-licensing deal worth up to $280 million, news that sent stocks soaring nearly 13% on Oct. 31. Motixafortide (GLS-010), approved by the U.S. FDA as Aphexda in combination with filgrastim (granulocyte colony stimulating factor) in September 2023, is a novel CXCR4 inhibitor that incites stem cell mobilization for autologous stem cell transplantation in patients with multiple myeloma.

Ausbiotech 2023: Australia invests in mRNA vaccines as biotech industry celebrates DNA milestones

Australia has a lot to celebrate when it comes to vaccines. The University of Queensland is where Ian Frazer invented the human papillomavirus vaccine Gardasil, and now Australia is projected to be the first in the word to eliminate cervical cancer, Queensland Deputy Premier Steven Miles said during the Ausbiotech 2023 conference held Nov. 1-3 in Brisbane, Australia.

Also in the news

3sbio, Abbvie, Acelink, Acumen, Adaptimmune, Aileron, Aldeyra, Amarin, Annovis, Apnimed, Apollomics, Atara, Atossa, Atyr, Biora, Bridge, Bristol Myers Squibb, Calidi, Centessa, Cessation, Chimeric, Cocrystal, Corcept, Cstone, Ctrl, Cybin, Engene, Engine, Eterma, Galera, Generate, Havah, Huidagene, Inhalon, Ionctura, Jasper, Khondrion, Kintara, Kyverna, Lakewood-Amedex, Lexicon, Lianbio, Ligand, Lung, MBX, MEI, Menarini, Mersana, Mindbio, Nimbus, Nouscom, Novartis, Novelmed, Ocuphire, Pierre Fabre, Porosome, Processa, Rayzebio, Reneo, Retinalgenix, Roche, RS, Sanofi, Sarepta, Selecta, Sensei, Shionogi, Shorla Oncology, Sitryx, Sosei Heptares, Sparx, Spero, Spexis, Spyglass, Stemline, Storm, Theratechnologies, UCB, Ultimovacs, United, Vaccinex, Verge Genomics, Virpax, Vtv, X4, Xoma, YS