Biopharma tackles COVID-19, HIV and other viruses with gene and cell therapies Using gene editing to keep cholesterol down and heart attacks at bay One-and-done gene therapy wipes out Parkinson's symptoms in mouse models Sponsored: Partnering Early for Cell and Gene Therapy Manufacturing BioMarin's hemophilia gene therapy 'Roc solid' after 4 years New gene therapy technique shows promise in stem cell model of retinitis pigmentosa Stanford team deploys CRISPR gene editing to fight COVID-19 Treating age-related diseases with a CAR-T that targets 'senescent' cells Allogene reports 63% response rate in early trial of off-the-shelf CAR-T in lymphoma Generation Bio tees up $125M IPO to push next-gen gene therapies Gene therapy cuts fat and builds muscle in sedentary mice on unhealthy diets Bluebird nabs $200M in early payout from retooled Bristol Myers BCMA deal Novartis, Sarepta tap Dyno to unearth new gene therapy vectors Featured Story | Monday, June 29, 2020 Top players in gene and cell therapy have mobilized behind the mission of turning the new technologies against COVID-19. Their efforts to perfect these therapies could help combat this pandemic, prevent future ones and eliminate viruses that have resisted more traditional treatments. read more |
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| | Supplying Clinical Trials for Cell and Gene Therapies Planning the appropriate clinical supply strategy for your critical cell and gene studies is crucial. Catalent has the capabilities and expertise to support you with our specialized and integrated solutions, including clinical supply management, cold chain handling, specialized distribution services, and more. |
Top Stories Monday, June 29, 2020 What if we could replace the arsenal of drugs used to prevent heart attacks with one injection? Verve Therapeutics believes gene "base editing" is the key. In data presented this weekend, the company showed that the approach could switch off either the PCSK9 or ANGPTL3 gene—both of which play a role in heart disease—in monkeys. read more Wednesday, June 24, 2020 A UCSD team developed a gene therapy technique that cripples the ability of the PTB gene to produce a functioning protein. In mouse models of Parkinson’s, the gene therapy turned supportive cells called astrocytes into dopamine-producing neurons, erasing symptoms of the disease. read more Tuesday, June 23, 2020 The rapid expansion and advancement of the cell and gene therapy pipeline is forcing companies to rethink their early stage priorities. With nearly 1,000 companies competing for finite production capacity, forward-thinking leaders are recognizing the value of establishing manufacturing strategies far earlier than is typical for other modalities and engaging with external development and manufacturing partners to secure long-term supply capacity. read more Thursday, June 18, 2020 Four years on, BioMarin’s hemophilia A gene therapy is still going strong—it staved off bleeding episodes in patients with hemophilia A and reduced their reliance on infusions of a blood-clotting factor that they lack. read more Thursday, June 11, 2020 Scientists at Trinity College Dublin and University College London “mini retinas” with mutations in the RP2 gene that cause some forms of retinitis pigmentosa. Delivering a functional RP2 gene to the model eyes rescued the rod photoreceptor cells needed for proper vision, they reported. read more Friday, June 5, 2020 Stanford bioengineers teamed up with researchers at the Lawrence Berkeley National Laboratory to develop a CRISPR system that neutralizes SARS-CoV-2 by scrambling the virus's genetic code. They believe the technology could prove useful for combating several types of viruses, including influenza. read more Thursday, June 18, 2020 Many age-related diseases like liver fibrosis and diabetes have been partly attributed to cellular "senescence," a zombie-like state in which cells stop proliferating. A team at Memorial Sloan Kettering Cancer Center is targeting these diseases with CAR-T cells that are designed to clear senescent cells. read more Friday, May 29, 2020 Allogene released expanded data from a phase 1 trial of its off-the-shelf, CD19-directed CAR-T, ALLO-501, plus its antibody ALLO-647. The response rate wasn't quite as impressive as what the company reported two weeks ago, but the details could generate some excitement among Wall Street analysts. read more Tuesday, May 26, 2020 Another preclinical-stage biotech is taking to the public markets—Generation Bio filed for a $215 million IPO to advance gene therapies for hemophilia A and phynylketonuria and push one of them into the clinic. The deal comes on the heels of a $110 million venture round raised in January this year. read more Monday, May 11, 2020 A single injection of the gene that makes the protein follistatin caused a significant buildup of muscle mass in mice while also preventing obesity, reported Washington University researchers. The gene therapy could someday help people who are overweight and suffering from joint pain caused by osteoarthritis, they suggested. read more Monday, May 11, 2020 With its Bristol Myers-partnered CAR-T therapy under FDA review, bluebird bio is getting a $200 million payout. The Big Pharma is forking over the cash to get out of future payments on the treatment and its follow-up outside the U.S. The duo framed the move as a bid to focus on the impending U.S. rollout of the treatment, ide-cel. read more Monday, May 11, 2020 Although gene therapy has opened a new realm of possibilities, its delivery vehicles have held it back. Dyno Therapeutics is on a mission to change that—and it’s coming out of stealth with artificial-intelligence-powered technology and partnerships with Novartis and Sarepta Therapeutics that could net it up to $2 billion. read more Enrollment Showcase | Sponsored by: University of Florida College of Pharmacy University of Florida precision medicine leaders highlight current knowledge and trends to advance the future of health care in this quarterly webinar series. Register today. |
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Resources Sponsored by: PRA Health Sciences Collaboration across the industry will be key to meeting the demands of the RACE Act and ensuring pediatric patients receive the care and treatments they need. Sponsored by: Systech Get an inside look… Merck shares its next-generation approach to supply chain protection with new digital platforms, including Blockchain. Sponsored by: Automation Anywhere Built on advances in Artificial Intelligence (AI), data mining, and information integration into various systems at 100% accuracy, the new era of intelligent automation is integral to life sciences for devising new treatments faster and at a lower expense. Sponsored by: Research Solutions Learn four ways to squeeze more value from your limited R&D resources. Sponsored by: BioAnalytix Download this eBook and Learn the Advanced Analytical Approaches for Improved Development of Cell and Gene Therapies Sponsored by: Blue Matter, strategic consultants in the life sciences White paper describing 7 keys to success for any biopharma company that is entering (or thinking about entering) Europe. Sponsored by: Catalent Learn about a real-world example of how clinical supply management expertise was applied to overcome inventory challenges for a complex, global study. Sponsored by: Catalent Whether a study is simple or highly complex, forecasting and simulation reports serve as data driven communication tools to help minimize risks and keep the study on track. Learn more about forecasting solutions for informed decision making and supporting contingency planning for clinical study. Sponsored by: Catalent Learn how to drive excellence within the forecasting process and utilize forecasting throughout the clinical study to better plan clinical supply budgets and project timelines. Explore how to identify potential supply-related issues before they can negatively impact your study. Drug Development Boot Camp® 2020 November 18-19, 2020 | Register now! Pre-Boot Camp training ongoing now. | COVID19 provisions – training will be on-site as normal. | New – may be possible for some individuals to access the training virtually in real time. | 