Therapeutic Class

CMC and staffing must be resolved if CGTs are to go ‘supersonic’

CMC deliverables must be addressed to continue CGT momentum, but overcoming talent gaps will give firms the competitive edge, says former Spark Therapeutics CTO Cynthia Pussinen. With 10 approvals in the US and Europe last year, there is little doubt the cell and gene therapy (CGT) sector is blossoming. Consultant Cynthia Pussinen described the sector as having “lifted off and in flight” at Phacilitate’s Advanced Therapies Week earlier this month, with much progress including “truly rewriting how patients are treated…

Catalent addressing CGT challenges with supply chain service

Catalent has designed a case management service, which aims to tackle challenges associated with the safety and timeline of cell and gene therapy programs. Contract development manufacturing organization (CDMO) Catalent says the complex nature associated with delivering doses of cell and gene therapies (CGTs) means the products need to be managed correctly and efficiently to reduce the risk of production schedule disruption and administration of the therapy to the individual. The launch of the company’s case management service looks to…

MD Anderson touts ‘entirely new modality for delivering mRNA’

Researchers led by the University of Texas MD Anderson Cancer Center have developed a delivery system for mRNA using extracellular vesicles (EVs). The study, published in Nature Biomedical Engineering, details how the researchers used EV-encapsulated messenger RNA (mRNA) to produce collagen for numerous months in the cells of photoaged skin. The researchers claim this is the first therapy that represents a proof-of concept for using EV mRNA therapies. EVs are small structures formed by cells that transport biomolecules and nucleic…

CGT myth-busting: ‘The process is NOT the product’

Deeper characterization and quality-by-design (QbD) approaches demonstrate the cell and gene therapy space has outgrown the lazy ‘process is the product’ rhetoric, say experts. Journalists and industry alike have clung onto the concept that ‘the process is the product’ when it comes to cell and gene therapies (CGTs). For years, the catchy phrase has been banded around convention centers and company boardrooms but as the field matures – 10 CGTs won approval in the US and Europe last year –…

WuXi ATU wins tech disruptor at #ATW awards in Miami

A transfection-free and scalable AAV technology has bagged WuXi ATU the Manufacturing Tech Disruptor of the Year Award at the first Advanced Therapies Awards ceremony. Miami, Florida’s famous Fontainebleau Hotel hosted the integral Advanced Therapies Week (ATW) Awards event last week, honoring progress and success in cell and gene therapies. Highlights of the evening including the presentation of the Lifetime Achievement Award to Carl June – the cell therapy pioneer and Time Magazine ‘Person of the Year’ runner-up 2016, who…

10 CGT approvals in 2022 brings ‘Gottlieb’s math’ close to realization

With cell and gene therapies coming of age, experts at Advanced Therapies Week looked to reducing COGs, novel payer models, and even biosimilars to tackle their spiraling price tags. In 2022, there were 10 approvals between the US and Europe for nine cell and gene therapy products: Adstiladrin, Breyanzi, Carvykti, Ebvallo, Hemgenix (approved by both FDA and EMA), Roctavian, Skysona, Upstaza, Zynteglo. As such, optimism was high at Phacilitate’s Advanced Therapies Week, held this week in Miami, Florida. “We’ve gone…

Novartis at JPM: ‘7-day CAR-T process an alternative to allogeneic’

With its next-generation CAR-T manufacturing platform, Novartis told JP Morgan Conference delegates it has no plans to move into the allogeneic space. Novartis is one of the pioneers in the chimeric antigen receptor (CAR) T-cell space, bringing the first therapy to market in the US in 2017: Kymriah (tisagenlecleucel), a treatment for B-cell acute lymphoblastic leukemia (ALL). Since then, a handful of other cell-based gene therapy products have been approved by the US Food and Drug Administration (FDA) but all…

Tides in! Deals and manufacturing news from Lilly, Merck, Cordenpharma, and more

BioProcess Insider turns the tides with news from Eli Lilly and Merck & Co., CordenPharma, and Aurisco in this oligonucleotide and peptide round-up. First up, and PeptiDream has garnered Big Pharma attention over the past couple of weeks, with both Eli Lilly and Merck & Co. (known as MSD outside north America) inking separate deals with the Japanese firm on the same day. PeptiDream uses its Peptide Discovery Platform System (PDPS) technology to produce highly diverse non-standard peptide libraries for…

Sarepta expands DMD gene therapy CDMO deal with Catalent

Catalent will supply commercial doses to Sarepta Therapeutics if its Duchenne muscular dystrophy (DMD) gene therapy delandistrogene moxeparvovec (SRP-9001) wins approval. In November, the US Food and Drug Administration (FDA) accepted the submission from Sarepta for its potential DMD gene therapy SRP-9001. The Agency has given an action date of May 29, 2023, and thus the firm has expanded its partnership with contract development and manufacturing organization (CDMO) to prepare for the potential of commercial production. “Sarepta is working as…

Vector suppliers need to improve production processes

Gene therapy developers need access to high quality viral vectors and to meet this need suppliers need to optimize manufacturing processes, says Genemagic Biosciences’ CEO, Jingmin Zhou. Viral vectors are empty virus particles that can be used to deliver selected sections of genetic material into target cells. They are a key component of modern molecular biology and have been used in everything from cell line development through to crop engineering. In addition, viral vectors are vital to the cell and…