| .png) | Is your MSC process optimized? The path to optimized MSC manufacturing is often bottlenecked by the time and labor required for comprehensive screening. Read this app note to learn about streamlining your process development and setting the design space for future scale-up. Access Now |
- Regenerative medicine nears banner year with $14.1B cash infusion, regulatory milestones and a well-stocked pipeline
- Roche's gene therapy plans take Shape with $3B-plus biobucks deal
- Moderna is ready to spend billions in COVID-19 cash, and the famed biotech has sights set on gene editing
- Vertex layers on bluebird bio pressure with $1.2B Arbor gene-editing pact
- CRISPR pinpoints new leukemia target and a 'pocket' that could make it druggable
- Gilead's Kite signs 2nd blood cancer pact, this time with Appia Bio for up to $875M
- Astellas to pay Minovia $20M upfront, with $420M on the table per product, for mitochondrial cell therapies
- Improving CAR-T cell therapy with more sensitive tumor identification
- CRISPR pioneer Feng Zhang's latest work delivers mRNA, gene therapy with a human protein
- FDA lifts clinical hold on Rocket gene therapy trial, creating launchpad for start of pediatric dosing
- Coya Therapeutics eyes IPO, but first they need some midstage data for ALS hopeful
- Genevant pens $303M biobucks pact with Takeda, aiming at rare liver diseases using gene therapy
- Sonoma Bio bags $265M to rev up cell therapies for arthritis, diabetes
- GentiBio banks $157M to fuel Treg treatment for Type 1 diabetes
- T-knife Therapeutics bags $110M to sharpen solid tumor cell therapy push into the clinic
- Novartis fine-tunes gene therapy candidates with a drug that acts as a 'dimmer switch'
Featured Story | By Annalee Armstrong For a long time, gene and cell therapies were a treatment option for the future. Now, the pace of development is moving at breakneck speed, with a number of firsts and $14.1 billion in financing collected over the initial half of the year. The Alliance for Regenerative Medicine has documented the “acceleration on all fronts” in a new report looking at the first half of 2021. read more |
| |
|---|
|
Top Stories By Amirah Al Idrus Roche may market the first FDA-approved in vivo gene therapy, but the company isn’t resting on its laurels. The Swiss pharma is teaming up with Shape Therapeutics on next-generation gene therapies for Alzheimer’s, Parkinson’s and rare diseases in a deal that could exceed $3 billion. read more By Annalee Armstrong Moderna has found a direction to volley its mountain of COVID-19 vaccine cash: gene editing. Executives revealed during a second-quarter earnings call Thursday that Moderna is ready to “expand our horizons” with external technologies or products. Read: They’re ready to start buying. read more By Kyle LaHucik Months after first-in-human gene-editing results made a splash around the world, Vertex is making a $1.2 billion edit to its CRISPR work through a new research pact with existing partner Arbor Biotechnologies. After dishing out $900 million upfront to CRISPR Therapeutics for sickle cell disease and beta thalassemia in April, Vertex is doubling down in its attempt to beat bluebird bio to market with a gene-editing therapy for those blood diseases. read more By Arlene Weintraub Using CRISPR, a team at Penn’s medical school discovered that an epigenetic regulatory protein called ZMYND8 governs the expression of genes that are critical for the growth and survival of AML cells. They uncovered a way to target it with drugs and to predict which patients are likely to respond to ZMYND8 inhibition. read more By Kyle LaHucik Gilead's Kite is helping Appia Bio fly a little higher with a blood cancer research and development deal that could be worth $875 million if the wind blows in the right direction. The Culver City, California-based biotech will lead preclinical and early clinical research on two off-the-shelf cell therapies. read more By Kyle LaHucik Atellas Pharma and Minovia Therapeutics are linking arms on researching and developing novel cell therapies for diseases caused by dysfunction of the mitochondria. The deal sees Astellas pay Minovia $20 million upfront with the potential for up to $420 million in biobucks for each product. read more By Angus Liu Many cancer patients may progress or relapse after being treated with CAR-T cell therapies, because tumor cells expressing low levels of the target antigen could survive and escape detection. Scientists at the Fred Hutchinson Cancer Research Center have designed new CARs with better antigen sensitivity, and they have shown promise in mouse studies. read more By Angus Liu COVID-19 mRNA vaccines and existing gene therapies, including those built with the CRISPR-Cas9 gene-editing tool, are delivered into cells with viral vectors or lipid nanoparticles. A research team led by CRISPR pioneer Feng Zhang of the Broad Institute has developed a new mRNA delivery system that harnesses a human protein called PEG10. read more By Nick Paul Taylor Rocket Pharmaceuticals’ gene therapy trial is back on track. With the FDA lifting the clinical hold after Rocket changed the study protocol, the biotech is now on course to start dosing children in the third quarter. read more By Kyle LaHucik Coya Therapeutics is planning a phase 2b trial to support an FDA approval for an ALS candidate as the biotech positions for another round of funding by year's end and a potential IPO down the road. The journey to a green light will be funded by a crossover financing round this year ahead of a public offering, but first Coya will need to put up some data to prove itself. read more By Ben Adams Forever biotech friend Takeda is signing up to a new deal with Genevant as it doubles down on the company. read more By Amirah Al Idrus After a modest $40 million debut in early 2020, Sonoma Biotherapeutics is grabbing a whopping $265 million to bankroll a pipeline of cell therapies for autoimmune diseases, including Type 1 diabetes and rheumatoid arthritis. read more By Amirah Al Idrus The Treg field is heating up. A week after Sonoma Bio snagged a meaty $265 million round to advance its regulatory T cell (Treg) programs, GentiBio is getting off a $157 million series A to do the same. read more By Nick Paul Taylor Investors have flocked to T-knife Therapeutics, which has been taking aim at solid tumors, an area where cell therapies have struggled to perform as well as they have in some blood cancers. Just last year, the biotech raised €66 million ($78 million) and now has reeled in a further $110 million to support a multifront push into clinical trials. read more By Angus Liu Novartis has licensed a technology that uses its investigational RNA splicing modulator branaplam as a modulator of gene therapy. The "dimmer switch," developed at Children’s Hospital of Philadelphia, could fine-tune levels of proteins expressed by gene therapies, improving a wide range of treatments, the researchers said. read more Resources Sponsored by: Box Read "Get to Know Cloud Content Management for Life Sciences" and learn how to eliminate silos across all workflows, from R&D to commercialization. Sponsored by: Evidera, a PDD business Learn about key post-marketing safety study solutions, including registries and REMS, and how they can most effectively help meet regulatory requirements and maximize patient access. Sponsored by: Cytiva A one-stop hub for Cytiva resources to support process intesification Sponsored by: Remarque Systems Remarque Systems consolidates data, then layers on visualization and automated metrics to generate consistent, actionable data insights in real-time. Click here for more info. Sponsored by: Catalent Learn about drug product process development challenges and risks, the difference between innovator and biosimilar drug product process development, and more during the drug product process development phase. Sponsored by: Catalent Get insights on key considerations for a successful tech transfer process for manufacturing a biologic drug product, including a case study on overcoming challenges in a process transfer for a sterile diluent. | 
