Therapeutic Class

Cell and gene therapy exits from Sanofi and AvroBio

Sanofi will focus its cell therapy strategy on allogeneic products, terminating its partnership with Sangamo. Meanwhile, disappointing trial results have driven AvroBio to abandon its lead gene therapy candidate for Fabry disease. The cell and gene therapy space continues to evolve but two announcements demonstrate the vicissitudes that linger over this burgeoning sector. Firstly, French Pharma giant Sanofi has ended a long-term hemoglobinopathies partnership with genomics firm Sangamo Therapeutics. “In its notice to Sangamo, Sanofi indicated that its termination relates…

Amgen: Not treating biosimilars as ‘stepchild’ is key to success

Amgen says using the same development expertise and manufacturing capabilities for both its biosimilar and novel biologic programs sets it apart in the industry. Pharma firm Amgen is one of a handful of firms that has successfully invested in both innovator products and biosimilars – biologics that are very similar to reference biologics and for which there are no clinically meaningful differences in terms of safety, purity, and potency. With expected 2021 biosimilar revenues set to reach $2 billion, CEO…

New year, new gene therapy plants from Pfizer and Expression

Christmas came early for Pfizer and Expression Therapeutics, which cut the ribbons on viral vector manufacturing plants in North Carolina and Ohio, respectively. Pfizer’s gene therapy division celebrated the new year with an expanded manufacturing footprint, having opened the first of three facilities at its site in Durham, North Carolina on December 15. The 85,500 square-foot plant will support clinical manufacturing of Pfizer’s gene therapy pipeline and represents an investment of $68.5 million by the firm. The facility will create…

Novartis to boost gene therapy pipeline through $800m Gyroscope buy

Novartis will add a gene therapy candidate in Phase II trials for an advanced form of dry age-related macular degeneration through the addition of Gyroscope Therapeutics. Novartis will gain GT005, an Adeno-Associated Virus-2 (AAV)-based gene therapy candidate intended to be a one-time treatment for geographic atrophy (GA) – an advanced form of dry age-related macular degeneration (AMD) that leads to progressive and irreversible vision loss – through the proposed acquisition. Novartis is one of the leaders in the gene therapy…

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

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

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

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

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

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…