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RNA sector set for progress thanks to decades long research drive

The RNA drug sector has overcome some significant hurdles according to Alynlam Therapeutics CEO John Maraganore, who says industry efforts to work with these challenging molecules have laid the foundations for growth. “The scientific obstacles are best defined here, small interfering RNA molecules are large. They’re charged. They’re susceptible to biological degradation. They’re also prone to activated immune sensor systems. And so the challenge of making drugs out of small interfering RNAs was really a formidable scientific challenge that needed…

BrainStorm’s cell therapy: Catalent high on NurOwn supply

by Dan Stanton

BrainStorm Cell Therapeutics is eying the potential launch of NurOwn and has selected CDMO Catalent to produce the autologous cell therapy from its site in Texas. Known as NurOwn, MSC-NTF is cell therapy candidate being investigated in Phase III trials for Amyotrophic Lateral Sclerosis (ALS) by BrainStorm. The potential therapy is made from bone marrow-derived mesenchymal stem cells (MSCs) taken from the patient, which are expanded and differentiated ex vivo before being converted into MSC-NTF cells that secrete high levels…

Internal capabilities ‘the backbone’ of Pfizer’s gene therapy strategy

by Dan Stanton

With a $500 million facility in North Carolina set to come online this month, Pfizer has lauded its approach to gene therapy development and manufacture. Big Pharma peers Roche and Novartis (via the acquisitions of AveXis and Spark, respectively), have achieved regulatory success in the gene therapy field, and Pfizer hopes to replicate similar achievements in the coming months and years. To get there, the firm is following a “three-pronged strategy,” according to Robert Smith – SVP of Global Gene…

WuXi ATU opens Philly cell and gene therapy testing center

by Gareth Macdonald

WuXi ATU has opened a testing facility in Philadelphia, US to cater for growing demand for the cell and gene therapy sector. The new test center expands the campus that WuXi ATU – a subsidiary of WuXi AppTec – established late in 2020. A company spokesman told us “The new Philadelphia facility will enable us to triple our capacity for critical tests related to the characterization of lentivirus and adeno-associated-virus (AAV) products such as replication competency (RCL and RCAAV assays)…

Doggybone DNA ‘ideal tech’ for pandemic vaccine response, says Touchlight

by Dan Stanton

Doggybone DNA vaccines can overcome the costly manufacturing limitations of plasmid DNA and the complex formulations and cold-chain issues of mRNA, says Touchlight’s chief research officer. With the successful rollout of Moderna and Pfizer/BioNTech’s respective COVID-19 vaccines, nucleic acid vaccines have shown overwhelming success for pandemic response due to their rapidity of manufacture and induction of robust immune responses, but limitations remain. “Most nucleic acid vaccines are mRNA-based, which induce high titers of neutralizing antibodies (nAb) but lower induction of…

Regeneron moving fast to assess Ab cocktail for Omicron variant

by Dan Stanton

Regeneron says it can adapt its COVID-19 antibody cocktail to be effective against the Omicron variant if necessary, thanks to lessons learned the first time around. Omicron was the word of the day at the Evercore ISI HealthCONx Conference yesterday, as drug companies assess the potential impact of the recently discovered variant. One company that spoke about its efforts against the new strain was Regeneron, which has been at the forefront of therapeutic development against COVID-19 with its antibody cocktail…

Scaling-up AAV production? Take your time to develop best processes

by Dan Stanton

The lack of standardization surrounding gene therapy production means process design can be more important than speed to clinic, says Oxford BioMedica’s CCO. The complexities of manufacturing monoclonal antibodies (mAbs) has been ironed out somewhat over the 30+ years of their presence commercially. But for the burgeoning gene therapy sector, there are no standardized mammalian-based platforms, for example, nor tried-and-tested upstream and downstream processes. “Viruses and viral vectors come in many shapes and sizes, each with their own characteristics,” Dave…

Novo Nordisk to add RNAi engine in $3.3bn Dicerna buy

by Dan Stanton

The acquisition of development partner Dicerna Pharmaceuticals will accelerate Novo Nordisk’s position in the RNA interference (RNAi) space. In 2019, Novo Nordisk paid $175 million upfront to collaborate with Dicerna in the discovery and development of RNAi therapies for liver-related cardiometabolic diseases using Dicerna’s proprietary GalXC RNAi platform technology. Two years on, and the Danish drugmaker has entered an agreement to buy the partner outright, offering $38.25 per share and valuing the firm at around $3.3 billion. “The acquisition of…

eBook: Vaccines Revisited — The Past, Present, and Future of Nucleic-Acid Vaccine Production

by Dan Stanton, Millie Nelson and Gareth Macdonald

The quarterly BioProcess Insider eBook series launched in 2021 to investigate specific modalities and business strategies driving the biopharmaceutical sector based upon trends and discussion points presented in the Insider’s pages. Two of the four quarterly publications — this one included — have focused on the vaccine industry. If the series had launched in 2019, cell and gene therapies might have warranted a double edition, or perhaps investments in antibodies and antibody fragments would have driven a bispecific focus. Five…

List forks out $125m+ for microbiome Indiana plant

by Millie Nelson

List Therapeutics will increase manufacturing capacity for live biotherapeutics products (LBPs) with its $125+ million investment in Indiana, US. List Therapeutics, whose parent company is South Korean firm Genome & Company, will invest in the city of Fishers, Indiana to build a 110,000 square-foot outsourcing facility. According to the firm, the facility will be commissioned by the end of 2023 and will be located at the Fishers Life Science and Innovation Park. The plant expands List’s offering of end-to-end manufacturing…