Therapeutic Class

ReNAgade takes off with $300m+ to tackle RNA challenges

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 adding cystinosis gene therapy candidate from AvroBio

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…

NewBiologix launches with $50m to develop CGT platform

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 launches with $130m to tackle manufacturability of AAVs

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…

IgE antibodies could be potential solid tumor treatments, says UCL

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…

The future of CGT production? Automation, AI, and academic capacity

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…

Tubulis deal sees Bristol bolster ADC commitment

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…

Pfizer touts mRNA as alternative to current flu vaccines

Pfizer says its mRNA-based influenza program, in collaboration with BioNTech, has potential to succeed current vaccination technologies. With influenza causing 650,000 global deaths every year, developing a more flexible and accurate vaccine is in the interest of global health. Last September, Pfizer initiated a Phase III study of its messenger RNA (mRNA) based flu vaccine, following a 2018 partnership with a then little-know German biotech BioNTech. In 2020, both BioNTech and mRNA were thrown into the global spotlight due a…

CPhI NA: CGT big picture ‘strong’ despite investment slowdown

The advanced therapy sector will weather the current financing storm, but thousands of skilled workers are still needed for it to achieve its potential, says JIB’s Parviz Shamlou. In 2019, the Jefferson Institute for Bioprocessing (JIB) opened its doors in Philadelphia, Pennsylvania to support the sector, providing training across all functions of bioprocessing including for cell and gene therapies (CGTs), which the region is renowned for. The Institute is currently undergoing a facility expansion program that will add pilot-scale production…

UK Gov sets up £10m CGT manufacturing center

The National Health Service has opened a £10 ($12.5) million cell and gene therapy manufacturing plant in Bristol that will increase UK capacity for plasmids and vectors. The Government-funded Clinical Biotechnology Centre (CBC) is designed to expand the UK’s ability to make the clinical grade products required for the research and development of new cell and gene therapies. The center will manufacture both plasmids and viral vectors. It will support early phase clinical trials and pre-clinical work. In time, the…