Bayer has licensed lipid nanoparticle (LNP) technology from Acuitas Therapeutics to develop in vivo gene editing and protein replacement programs in the liver. Bayer has joined forces with Acuitas Therapeutics, a Canadian biotechnology company specializing in LNP delivery of nucleic acid therapeutics. The partnership grants Bayer, and its affiliate Asklepios BioPharmaceutical (AskBio), access to Acuitas’ LNP technology. Bayer and AskBio will provide the gene editing technologies. “The collaboration will focus on developing and enabling in vivo genome editing CRISPR therapies for…

Johnson & Johnson (J&J) says it remains agnostic to modality type in favor of the end user despite its recent efforts in the cell and gene therapy (CGT) space. At last week’s Bernstein’s 39th Annual Strategic conference, Johnson & Johnson was asked about its strategy to evolve its pipeline, specifically with new technologies and modalities including CGTs, mRNA, gene editing, and gene silencing. “We are already in-house in cell therapy and in gene therapy,” stated CEO Joaquin Duato, referencing the…

As firms look beyond COVID-19, one of the most promising platforms is self-amplifying RNAs, says a report from Clarivate. The report by Clarivate, entitled “Where pharma are investing for the future of medicine”, said messenger RNA (mRNA) COVID-19 vaccines demonstrated “proof of concept for the technology” with BioNTech/Pfizer and Moderna releasing their  respective COVID-19 vaccines in 2020 and 2021. As life returns to normal, pandemic-related orders for COVID-19 vaccines have dropped in size –  Pfizer, for example, reported a 26%…

The $300 million Series A financing round will support development of an all-RNA delivery platform, says ReNAgade. Massachusetts-based start-up company ReNAgade claims its RNA platform combines delivery technologies, such as lipid nanoparticles (LNPs), with a wide range of coding, editing, and gene insertion tools to enable an all-RNA system to develop medicines. With $300 million raised in a Series A financing round led by biotech investors MPM BioImpact and F2 Ventures, a spokesperson for ReNAgade told BioProcess Insider,“the funding will…

Novartis will pay $87.5 million in cash to add the Phase I/II hematopoietic stem cell (HSC) gene therapy program to its advanced therapy pipeline. Novartis, through its acquisition of AveXis in 2018, is one of a small but growing number of players to bring a gene therapy to market: Zolgensma (onasemnogene abeparvovec) was approved in 2019 for spinal muscular atrophy (SMA). The Swiss pharma giant says it has over 20 gene therapy project in its pipeline and this week is adding…

With $50 million in Series A funding, NewBiologix aims to tackle challenges associated with the production of recombinant adeno-associated virus (rAAV).   The recently launched Lausanne, Switzerland-based company, NewBiologix, focuses on rAAV vectors, which is a typical delivery vehicle used for gene therapies. The firm is developing a DNA-based platform for the advanced engineering of cell lines used to produce cell and gene therapies (CGT) and it will be applied to human (HEK-293) and mammalian (CHO) cell lines. “By engineering…

Ascend Gene & Cell Therapies has launched to feed the demand for adeno-associated virus (AAV) vectors. Having raised $132.5 million in a Series A funding led by Abingworth and Petrichor, newly-formed contract development and manufacturing organization (CDMO) Ascend hopes to support cell and gene therapy players in the development and production of their products. Specifically, the firm is focusing on the development and manufacture of AAV by leveraging its split two-plasmid transfection system, something it says differentiates it from other…

Antibodies that cause allergic reactions also have potential as treatments for solid tumors, according to UCL researchers exploring the new field of allegro-oncology.  Immunoglobulin E (IgE) antibodies consist of two heavy chains and two light chains, with the ε chain containing four Ig-like constant domains. They are an important part of the immune response, particularly against parasites and infection by members of the helminth family of worms. However, IgE antibodies are more commonly known for their role in life-threatening hypersensitivity…

Advanced therapy manufacturing is still in its infancy says JIB’s Parviz Shamlou, who looks to digital processes and advanced AI to improve production going forward. Legacy biopharmaceuticals such as proteins and monoclonal antibodies are well-proven and relatively easy to scale-up. A single batch from a 20,000 L bioreactor, for example, will provide sufficient drug product to treat thousands of patients. However, cell and gene therapies represent a very different model, says Parviz Shamlou, executive director at the Jefferson Institute for…

Bristol Myers Squibb could pay Tubulis over $1 billion in milestone payments after inking a deal to develop differentiated antibody-drug conjugates (ADCs) for solid tumors. Bristol Myers will pay $22.75 million up front to Tubulis for the exclusive rights to access the German biotech’s payloads and P5 conjugation platform for the development of a selected number of ADCs to treat solid tumors. According to Tubulis, the P5 platform allows rapid generation of ultra-stable ADCs, supported by linker stability and chemical…