CELL AND GENE THERAPY
Tune Therapeutics | December 03, 2021
Tune Therapeutics, a biotechnology company pioneering the creation of epi-therapeutic medicines, launched today with its powerful and precise genetic tuning platform, TEMPO. This cutting-edge technology dials gene expression up or down to desired levels – with the potential to reverse pathways of cancer, genetic disease, and aging by changing cell fate and function at will.
"Genetic medicine is at a tipping point. We now understand that the driving force of human health and disease is not our genes, but the epigenomic elements that shape and control them. Until now, scientists and bioengineers lacked the combined understanding, clinical expertise, and technology needed to make epigenomic therapies a practical reality. Now, we have all three."
Matt Kane, CEO of Tune Therapeutics
Tune's proprietary TEMPO platform can rapidly target and adjust the epigenomic machinery of the cell, which shapes DNA and controls gene expression. By varying specific control modules in an iterative process, TEMPO can fine-tune expression toward healthy levels – even in diseases involving multiplex or polygenic interactions.
Unlike genome editing, the tuning process does not generate double- or single-strand breaks in DNA and makes no permanent changes to the DNA sequence. This de-risks the precise targeting of entire gene networks, allowing Tune to simultaneously turn silenced genes on and dial over-expressed genes down, in a practical, therapeutic context.
Tune has already shown that TEMPO can locate epigenomic elements involved in several intractable genetic conditions – revealing targets and networks that would be invisible or inaccessible to gene editing approaches. Moreover, Tune can optimize TEMPO to command expression of individual genes or networks with remarkable specificity and precision. This opens the door to an entirely new class of epi-therapeutics.
"The exciting challenge in front of us is taking these transformative advances in technology and extending their potential for our greater society," said Charlie Gersbach, PhD, Acting Chief Scientific Officer, Tune Therapeutics. "From proof of concept in rare, single-gene disorders to common conditions that aren't linked to a single gene mutation – but are treatable through epigenomic control and constitute the vast majority of human diseases."
Veteran Genomic Medicine Leadership Team
Tune is launching with a veteran leadership team, endowed with deep expertise in gene and cell therapy, genome editing, and epigenetics.
Matt Kane, Chief Executive Officer
Akira Matsuno, Co-Founder, President and Chief Financial Officer
Charlie Gersbach, Ph.D., Co-Founder, Acting Chief Scientific Officer
Fyodor Urnov, Ph.D., Co-Founder, Scientific Advisory Board
Heidi Zhang, Ph.D., Executive Vice President, Head of Technical Operations
Blythe Sather, Ph.D., Vice President, Head of Research
In addition, Tune's Board of Directors includes Mr. Kane, Dr. Gersbach, Ali Behbahani, M.D., (New Enterprise Associates), and co-founder Dan McHugh
Drawing upon deep, local talent pools in Durham and Seattle, Tune has assembled two highly seasoned discovery and development teams, secured foundational intellectual property from Duke University, and raised $40 million from top-tier investors – including co-leads New Enterprise Associates and Emerson Collective, with Hatteras Venture Partners, Mission BioCapital, and others joining the round. This financing will enable Tune to rapidly advance its preclinical research, attract top-tier talent, and further develop its therapeutic platform.
"Tune is effectively pioneering a brand-new therapeutic modality," said Dr. Behbahani. "With the unbound potential of this approach, and their collective successes in the field, Tune is primed to become a transformative presence in modern biomedicine."
About Tune Therapeutics
Tune Therapeutics, Inc. is orchestrating the power of the epigenome to treat complex, pervasive diseases that have long eluded treatment. With its versatile and powerful TEMPO epigenomic control platform, Tune's experienced team is pioneering a new therapeutic modality that can fine-tune any gene network. In breaking free from the limitations of traditional gene and cell therapies, Tune is developing solutions for even the most challenging and intractable diseases – and building the capacity to transform human health and medicine.
Stemcyte | March 23, 2022
On January 7th, 2022, the FDA received the BLA (Biologics License Application) for the "HPC-Cord Blood" product. On March 8th, 2022, StemCyte was formally informed that the submission was acceptable to join the biologics license quality review process.
The "HPC-Cord Blood" is an umbilical cord blood hematopoietic stem cell product intended for unrelated donor hematopoietic progenitor cell transplantation procedures in patients with hematopoietic system disorders an appropriate preparative regimen for hematopoietic and immunologic reconstitution. There have been over 40,000 successful umbilical cord blood transplants globally to treat disorders of the hematological and immunological systems and genetic metabolic diseases since the first successful use of umbilical cord blood to treat Fanconi Anemia patients in 1988.
StemCyte has donated more than 2,200 cord blood units for transplantation to 1 in every 20 patients with umbilical cord blood transplants globally during the last 20 years. In addition, at least 350 transplant centers around the world, including well-known medical centers such as UCLA Medical Center, Duke University Hospital, Taiwan Chang Gung Memorial Hospital, and National Taiwan University Hospital, consistently meet the quality standards of international accrediting bodies and are recognized and trusted as safe and effective by StemCyte's products.
Avantor | June 24, 2022
Avantor, Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries has entered into an agreement with GeminiBio to deliver custom hydrated solutions and cell culture media to the biopharma industry.
Custom hydrated solutions and cell culture media are critical components of the bioproduction workflow, especially for emerging modalities such as gene therapy. This collaboration will complement Avantor's proven ability to serve manufacturing platforms with GeminiBio's small-to-medium scale offering, enabling customers to utilize custom cGMP products through the full development lifecycle – including early-stage research, scale-up and commercialization.
This new capability along with Avantor's recent acquisitions and site investments, support a growing portfolio of proprietary offerings, demonstrating Avantor's commitment to supporting customer workflows in both upstream and downstream processing for the biopharma industry.
With the imperative to meet patient needs quickly, our customers require custom solutions to accelerate their bioproduction goals, Customized media is critical in optimizing cell line productivity, and hydrated solutions offer efficiency to biomanufacturers as they optimize their downstream processes. Our collaboration with GeminiBio will drive increases in product yields, helping bring efficiency and speed to our customers as they scale. Together, we can drive science forward to bring therapies to the patients who need them most."
Dr. Ger Brophy, Executive Vice President, Biopharma Production for Avantor.
Enabling customers to optimize their upstream and downstream liquid manufacturing workflows by offering custom solutions, built around a strong quality foundation, has been an essential part of GeminiBio's success. Collaborating with Avantor, who shares our focus on quality, will allow us to expand our ability to address customer needs for both small and large volume process liquids."
Brian Parker, CEO of GeminiBio.
Avantor®, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries. We set science in motion to create a better world.