The White House Office of Science and Technology Policy (OSTP) said Tuesday that "altering the human germline for clinical purposes is a line that should not be crossed at this time" in a statement outlining the Administration's position on acceptable uses for gene editing technology.
In the statement, OSTP director John Holdren expressed concern over the unknown consequences of heritable genetic alterations for future generations. Holdren also emphasized the need for global cooperation in establishing guidelines for gene editing applications.
In April, NIH said it would not fund research involving gene editing in human embryos, but would continue to fund research using human somatic or germline cells unless they would be used to create human embryos for research purposes. OSTP's statement does not address specific research uses for gene editing technologies; OSTP did not respond to requests for comment (see BioCentury Extra, April 29).
Last week, the National Academy of Sciences and the National Academy of Medicine said they would hold an international meeting this fall to discuss scientific, ethical and policy implications of human gene editing.
A Chinese research group published a study last month characterizing the use of CRISPR/Cas9 (CRISPR-associated protein 9) in non-viable human embryos (see BioCentury Extra, April 23).
Five Prime Therapeutics Inc. (NASDAQ:FPRX) gained $3.78 (19%) to $23.45 in early after-hours trading Tuesday after post-market news that it partnered with bluebird bio Inc. (NASDAQ:BLUE) to develop chimeric antigen receptor (CAR) T cell therapies.
Five Prime will grant bluebird exclusive rights to preclinical-stage human antibodies against an undisclosed target. bluebird will use its lentiviral gene therapy platform and existing CAR T capabilities to develop therapies against the target.
Aron Knickerbocker, Five Prime's CBO, told BioCentury the target has been validated in hematologic cancers and solid tumors.
Five Prime will receive an upfront payment of $1.5 million and is eligible for $130 million in milestones per product, plus tiered royalties. bluebird will fund development, regulatory and commercial activities.
bluebird has a separate collaboration giving Celgene Corp. (NASDAQ:CELG) options to license a pipeline of CAR T candidates after Phase I studies. Those assets are in preclinical development (see BioCentury Extra, March 21, 2013).
Five Prime shed $0.38 to $19.67 during Tuesday's trading, while bluebird gained $5.31 to $183.69.
Amgen Inc. (NASDAQ:AMGN) and AstraZeneca plc (LSE:AZN; NYSE:AZN) will halt clinical studies of investigational psoriasis therapy brodalumab, including a Phase III trial to treat psoriatic arthritis, Amgen spokesperson Trish Hawkins told BioCentury.
Amgen had said Friday it would exit a collaboration with AZ to develop the IL-17 inhibitor due to concerns that the compound will require restrictive labeling based on suicidal ideation and behavior seen in clinical trials.
The companies released data last year from three Phase III studies of the human IgG2 mAb against IL-17 receptor (IL17R; IL17RA) to treat moderate to severe plaque psoriasis. A Phase II trial to treat axial spondyloarthritis was due to begin in June. Amgen previously said it planned to submit a BLA and an MAA mid-year for brodalumab to treat moderate to severe plaque psoriasis.
AZ said it will evaluate data from brodalumab trials and decide how to proceed with the program (see BioCentury Extra, May 22).
Amgen lost $4.57 to $159.01 on Monday. AZ fell 100p to 4,373.50 in London and lost $1.77 to $67.68 in New York.
Synthetic biology company Synlogic Inc. (Cambridge, Mass.) named Jose-Carlos Gutierrez-Ramos president and CEO. Gutierrez-Ramos was group SVP and global head of biotherapeutics R&D at Pfizer Inc. (NYSE:PFE).
Infectious disease play Nabriva Therapeutics AG (Vienna, Austria) hired Elyse Seltzer as CMO. She was VP of global clinical sciences and operations at GlaxoSmithKline plc (LSE:GSK; NYSE:GSK). Former CMO William Prince will become SVP of clinical science.
Sanofi (Euronext:SAN; NYSE:SNY) named Suresh Kumar EVP of external affairs, a newly created position. Kumar was a partner at Oliver Wyman, where he led the firm's public sector practice and was a member of its health and life sciences team.
Drug delivery company Delcath Systems Inc. (NASDAQ:DCTH) promoted Jennifer Simpson to president and CEO. Simpson had been interim president and CEO since September 2014.
Diagnostic play Cancer Genetics Inc. (NASDAQ:CGIX) named Mandar Kulkarni chief technology officer of its Indian subsidiary. Kulkarni was head of molecular biology and genomics at Sandor Lifesciences Pvt. Ltd (Hyderabad, India).
Aegerion Pharmaceuticals Inc. (NASDAQ:AEGR) said lomitapide met the primary endpoint in a Japanese open-label Phase III trial to treat homozygous familial hypercholesterolemia (hoFH).
The primary endpoint of the nine-patient trial was the mean percent change in LDL-C at the maximum tolerated dose compared to baseline after 26 weeks of treatment.
Aegerion plans to submit an NDA for lomitapide in Japan in 4Q15 or 1Q16 based on the 26-week data. The company intends to submit 56-week data while the NDA is under review.
Aegerion markets the small molecule microsomal triglyceride transfer protein (MTTP; MTP) inhibitor as Juxtapid in the U.S. and in the EU as Lojuxta. Aegerion reported $57.3 million in lomitapide sales in 1Q15.
Aegerion lost $0.22 to $20.16 on Tuesday.
PureTech Ventures (Boston, Mass.) intends to raise about $160 million in an IPO on the London Stock Exchange in June. The firm plans to invest the proceeds to develop its existing portfolio companies' pipeline candidates and invest in new candidates. Jefferies and Peel Hunt are underwriters.
Spokesperson Ben Atwell said PureTech is choosing to list in London rather than the U.S. because U.K. investors have experience with companies with similar business models. He cited as an example Allied Minds plc (LSE:ALM), which invests and creates subsidiaries based around early stage researrch emerging from U.S. universities, federal research institutions and laboratories. Allied Minds' share price has more than tripled to 601.50p since the company listed last June.
PureTech's portfolio includes Vedanta Biosciences Inc. (Boston, Mass.), which is developing therapies derived from the microbiome, and Gelesis Inc. (Boston, Mass.), which is developing an encapsulated device that swells in the stomach and small intestine to control appetite.
Separately, PureTech named Joichi Ito chairman; he joined the firm as a senior partner last October. Marjorie Scardino, former CEO of Pearson plc (NYSE:PSO) and chairman of the MacArthur Foundation, joined PureTech's board.
MPM Capital named Pablo Cagnoni managing director. Cagnoni was president of the Onyx Pharmaceuticals Inc. subsidiary of Amgen Inc. (NASDAQ:AMGN).
Longitude Capital promoted Sandip Agarwala to managing director from principal.