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Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of geneediting.
The collaboration with GenEdit, a South San Francisco startup developing new ways of delivering geneediting tools into the body, dates back to December 2020, but has recently begun to advance.
Scribe will receive $40 million upfront from Sanofi in a collaboration initially focused on developing an in vivo geneediting treatment for sickle cell disease.
Development of EDIT-101, the first CRISPR-based therapy to be used in a company-led trial to alter genes within a person's body, will now move forward under Editas' sole ownership.
A deal with biotech startup Capsida, which recently raised $140 million, gives CRISPR access to a Caltech technology that aims to improve the delivery of genetic medicines.
Formed as a diagnostics developer, Mammoth Biosciences, which Doudna started with two of her former students at the University of California, has accelerated plans to make geneediting medicines as well.
Regeneron Pharmaceuticals has collaborated with Mammoth Biosciences to develop in vivo clustered regularly interspaced short palindromic repeats (CRISPR)-based geneediting therapies.
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.
Vertex will license CRISPR technology to develop insulin-producing islet cells that are more resistant to immune rejection, adding to other efforts directed at Type 1 diabetes.
The geneediting company will focus on “in vivo” medicines, while seeking to license out or find a development partner for its clinical-stage treatment reni-cel.
A geneediting therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics can mute sickle cell disease’s most damaging symptoms. Yet treatment may not be as simple as its dramatic benefit makes it seem.
While the biotech will not develop the geneediting therapy further on its own, CEO Gilmore O’Neill claimed the data are still a meaningful demonstration of what CRISPR can accomplish.
The pharma will pay $75 million to access Precision’s geneediting technology, adding another program to the lengthening list of drug development efforts targeting the inherited blood condition.
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA geneediting therapies. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.
The geneediting company will take a 10% stake in SparingVision, a young biotech that will use Intellia's CRISPR technology to develop treatments for up to three ocular targets.
Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.
A freshly inked agreement gives Vertex the right to use tools from Arbor Biotechnology to develop "ex vivo" cell therapies for diseases like Type 1 diabetes and sickle cell, among others.
Shoreline will acquire a preclinical NK cell therapy Editas has been developing, as well as a license to use the CRISPR biotech’s geneediting technology.
Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo geneediting programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). The company will receive a combined upfront payment and equity investment of $60m.
Under the deal, the LentiPeak lentiviral vector technology platform and cell therapy production expertise of ElevateBio BaseCamp will be used by Affini-T to develop its investigational oncogenic driver programmes in the clinic. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.
The acquisition of Proof Diagnostics, which was formed to develop a COVID-19 test, gives Ginkgo a library of novel enzymes that can be used to make genetic medicines.
have received the green light from Health Canada for their Clinical Trial Application for VCTX210: an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D). CRISPR Therapeutics and ViaCyte, Inc.
have announced a strategic collaboration and option agreement for the use of Mammothâs CRISPR systems to develop in vivo gene-editing therapies. Bayer AG and Mammoth Biosciences, Inc.,
Ionis will pay the Bay Area gene-editing startup $80 million upfront as the companies work together to develop up to four gene-editing therapies, with the option to add four more targets. On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.
Expanding upon the CRISPR-Cas9 geneediting system, researchers at MIT have designed a new technique called PASTE geneediting that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE geneediting technique was recently published in Nature Biotechnology.
AMES, IA – Through CRISPR and other gene-editing technologies, researchers and developers are poised to bring dozens – if not hundreds – of new products to grocery stores: mushrooms with longer shelf lives, drought-resistant corn and bananas impervious to a fungus threatening the global supply.
Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA.
For example, once considered incurable and terminal, patients with sickle cell disease may reach new summits in their lives with geneediting technologies such as CRISPR to repair affected DNA and, in some cases, functionally cure the condition. There are also nuances for operational planning in cell therapy development programs.
In what will be a big moment for CRISPR technology, Intellia and development partner, Regeneron Pharmaceuticals will share the world’s first data from a therapy that edits flawed genes inside the body.
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.
Genomic studies of cancer patients have revealed thousands of mutations linked to tumor development. However, for the vast majority of those mutations, researchers are unsure of how they contribute to cancer because there's no easy way to study them in animal models.
WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated geneediting technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developinggeneediting therapies to treat genetic diseases.
Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.
Vita Therapeutics has raised $31m in a Series B financing round for the development of cell therapies for neuromuscular diseases and cancers. VTA-100 has been designed to be an autologous therapy which combines gene correction as well as induced iPSC technology for repairing and replacing muscle cells for LGMD2A patients.
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