Over the past decade, single-cell biology and spatial biology have revolutionized how researchers understand biological processes. Single cell has made, according to an editorial in Molecular Systems Biology, important contributions to our understanding of cell types, cell states, cell–cell interactions, and tissue architecture—while spatial biology also promises to transform biology and especially pathology by measuring physical tissue structure and molecular characteristics at the same time, as a recent review article in Science has noted.

Playing leading roles in the development of both technologies have been 10x Genomics and its co-founder and CEO, Serge Saxonov, PhD, who discusses his career in genomics and science on GEN’s Close to the Edge.

After discussing how he developed a lifelong interest in genomics and science, Saxonov shares how as founding architect and director of R&D at 23andMe, he defined the initial conception of its product, built many elements of the technology, and drove strategy and execution of R&D functions as the direct-to-consumer genetic testing company scaled.

As VP of applications at QuantaLife, which Bio-Rad acquired in 2011 for $162 million, Saxonov was responsible for building content, driving new applications, and identifying key diagnostics opportunities for the core droplet digital PCR (ddPCR) technology.

In 2012, Serge co-founded 10x Genomics with Ben Hindson and Kevin Ness. As the company’s CEO, Saxonov defined 10x’s vision and strategy, contributed to core inventions, and has led the company since its inception. In 2016, Saxonov was honored as one of Goldman Sachs’ 100 Most Intriguing Entrepreneurs of the Year.

Alex Philippidis is senior business editor of GEN. Jonathan Grinstein, PhD, is senior editor of GEN.

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In this episode of Close to the Edge from GEN, senior editor Jonathan D. Grinstein, PhD, speaks with Fyodor Urnov, PhD, a pioneer in the field of genome editing. Urnov is a professor of molecular and cell biology at UC Berkeley, the scientific director of technology and translation at the Innovative Genomics Institute in Berkeley, California, and a co-founder of Tune Therapeutics, one of several biotechs exploring epigenome editing. The conversation goes into great detail about Urnov’s background in epigenomics and genome editing, including his time at Sangamo, where his team coined the term “genome editing” in 2005. It then discusses his vision for “CRISPR cures on demand” and the obstacles that currently stand in the way. Urnov also provides insight into the origin story of Tune Therapeutics and how this epigenome editing company stacks up against more recent entrants.

The post Perfect Pitch: Genome Editing Pioneer Fyodor Urnov on Commercializing CRISPR Therapies and Epigenomic Tuning appeared first on GEN - Genetic Engineering and Biotechnology News.

As CEO of the Biotechnology Innovation Organization (BIO), King has focused in recent months on stabilizing the leadership and direction of the 1,000-plus member industry group, where the long tenure of Jim Greenwood was followed by the relatively short tenure of Michelle McMurry Heath, MD—about two years, ending last October.

King is also leading BIO’s ramp-up toward its 2023 BIO International Convention, to be held June 5-8 at the Boston Convention & Exhibition Center, and overseeing the organization’s response to a series of challenges to the industry. These challenges range from articulating industry’s perspective on pricing prescription drugs following enactment of the “Inflation Reduction Act” last year, to preserving access to the most widely used abortion pill, mifepristone in the face of a court challenge by opponents of abortion rights.

King is a former chair of BIO, and previously served as CEO of GlycoMimetics, a late clinical-stage biotechnology company she co-founded. GlycoMimetics focuses on discovering and developing glycobiology-based therapies for cancers and inflammatory diseases.

Before GlycoMimetics, King was a one-time Entrepreneur in Residence at venture capital firm New Enterprise Associates (NEA). Speaking with GEN senior business editor Alex Philippidis, Rachel recounts her multi-faceted career in biotech, and assesses the industry’s progress and continuing challenges in ensuring federal funding for basic research, advancing newer technologies such as synthetic biology and cell and gene therapy; and diversifying the industry’s workforce.

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For more than a month, Icahn has offered persistent criticism of Illumina’s current board led by chairman John W. Thompson, and the company’s management led by CEO Francis deSouza.

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Icahn’s arguments include:

• Illumina’s effort to acquire cancer blood test developer Grail despite opposition from U.S. and European regulators, which he says has drained the company of resources.
• Also, Illumina’s shrinking stock price—The company has lost some $50 billion in market capitalization—that’s the share price times the number of outstanding shares of a public company.
• He has also cited the board’s near doubling of CEO Francis deSouza’s total compensation last year, to nearly 27 million dollars, much of that increase based on stock options.

Icahn elaborates on these and other arguments at length, and discusses how he plans to move Illumina forward with GEN senior business Editor Alex Philippidis on a special episode of GEN’s “Close to the Edge.” Joining Icahn on the episode is one of his three nominees to Illumina’s board, Andrew J. Teno, a portfolio manager at Icahn Capital since October 2020.

Along with Teno, Icahn has also nominated to Illumina’s board Jesse A. Lynn, general counsel of Icahn Enterprises; and Vincent J. Intrieri, founder and CEO of VDA Capital Management, a private investment fund, and a former Icahn employee from 1998–2016.

Illumina has rejected the three as lacking the qualifications and experience it seeks in board members, and has defended its pursuit of Grail under deSouza.

Icahn is the CEO of Icahn Capital, LP, a wholly owned subsidiary of Icahn Enterprises LP, through which he manages various private investment funds. Icahn Enterprises is a Nasdaq-listed holding company engaged in businesses that include investment, energy, automotive, food packaging, real estate and home fashion.

The post Carl Icahn States His Case for Change at Illumina on “Close to the Edge” appeared first on GEN - Genetic Engineering and Biotechnology News.

Anzalone discusses his career in genome editing on GEN‘s Close to the Edge. He relates how he conceived the idea for prime editing, a “search and replace” gene editing approach, while he was a Jane Coffin Childs Memorial Fund postdoctoral fellow in the laboratory of David Liu, PhD.

The technology was first disclosed publicly in October 2019, when Liu and colleagues at the Broad Institute of MIT and Harvard published a paper in Nature that laid out a new mechanism for genome editing called “prime editing” that did not make double-strand breaks in the target sequence or use a donor DNA template. Liu and Anzalone co-founded a company formed to commercialize the technology, Prime Medicine, where Anzalone is the lead developer of prime editing, and the company’s scientific co-founder and head of its prime editing platform.

Despite a bear market for newly-public biotechs, Prime Medicine launched an upsized initial public offering (IPO) that raised $180.3 million in net proceeds. In its IPO prospectus, Prime disclosed a pipeline of 18 programs, all in discovery phases. Half are within its “immediate” strategic category, and include programs targeting blood, liver, eye, and ear disorders. The rest of Prime’s pipeline includes programs targeting Duchenne muscular dystrophy, cystic fibrosis, and seven repeat expansion disease-targeting programs.

The post Search and Replace: Andrew Anzalone Details How Genome Editing Went Prime Time on “Close to the Edge” appeared first on GEN - Genetic Engineering and Biotechnology News.

In the 2000s, deCODE expanded operations, moving into drug development and even launching a direct-to-consumer genetics business. But amidst a recession that hit Iceland particularly hard, deCODE declared bankruptcy in 2009. After restructuring, deCODE was acquired by Amgen in 2012 for a reported $415 million. It continues to operate with a scientific independence under the watchful eye of Stefansson. deCODE’s genomic prowess was on display in July 2022, when Stefansson and his colleagues published a major study on the whole-genome sequencing of more than 150,000 volunteers in the UK Biobank (UKB) in Nature.

In this special episode of Close to the Edge, correspondent Malorye Branca interviews Stefánsson on the implications of that landmark biobank study, deCODE’s relationship with Amgen, his views on deCODE’s legacy and the future study of human genetic diversity.

The post Decoding Genomic Diversity with deCODE Genetics CEO Kári Stefánsson on “Close to the Edge” appeared first on GEN - Genetic Engineering and Biotechnology News.

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In a wide-ranging conversation, Hopkins discusses his journey from South Wales to the University of Oxford, Pfizer to the University of Dundee, and the progress made by his Oxford-based company, which he launched in 2012. Hopkins is in no doubt that artificial intelligence is poised to transform not only the chemistry of drug discovery but also many other aspects of clinical and pharmaceutical research.

Headquartered in Oxford, Exscientia has claimed a number of firsts, including being the first company to have AI designed molecules entering clinical trials. After its initial public offering (IPO) in 2021, Exscientia’s total funding has surpassed $1 billion, with billions more promised if its pharma partnerships with the likes of Sanofi meet their goals.

The post From Knowledge, Drug Power: Andrew Hopkins Discusses AI-Based Development on “Close to the Edge” appeared first on GEN - Genetic Engineering and Biotechnology News.

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Shehnaaz Suliman, MD, MBA, MPhil, overcame South Africa’s apartheid policy to build careers in medicine, investment banking, and drug development that have brought her to the helm of ReCode Therapeutics, a developer of targeted therapies for life-limiting respiratory diseases.

On Episode 13 of “Close to the Edge,” Suliman shares how she came to lead ReCode following a career that began as a physician in her native South Africa, where she received her MD from the University of Cape Town Medical School.

Suliman pivoted to investment banking about two decades ago, earning an MBA at Oxford University, then holding positions at Lehman Brothers and later Petkevich & Partners. A few years later, she embarked on a successful career as a biotech business development executive. At Gilead, Suliman held management roles of increasing responsibility between 2005 and 2010 and played a significant role in expanding the company into new therapeutic areas, notably HIV, through M&A.

After Gilead, Suliman held positions at Genentech and its parent company Roche, as well as at Theravance, where she put together a $1 billion profit-sharing partnership with Johnson & Johnson’s Janssen Biotech for its lead JAK inhibitor program before moving to Alector Therapeutics as president and COO.

Suliman was appointed CEO of ReCode in January, succeeding David Lockhart, PhD, who has transitioned to chief scientific officer and remains the company’s president.

Suliman shares her perspectives on drug development, especially in respiratory diseases and the hot area of RNA-based therapeutics, as well as how ReCode has sought to address both through its selective organ targeting (SORT) platform—a platform of non-viral lipid nanoparticles (LNPs), designed to deliver treatments with precision, as well as safely and effectively. ReCode is also focused on developing therapies that apply gene editing/correction in target cells, including stem cells.

ReCode says its delivery platforms can be customized to treat different respiratory diseases and targeted organs. The company’s pipeline is anchored by preclinical inhaled and IV lead programs for cystic fibrosis (CF) caused by nonsense mutations, as well as an inhaled treatment for primary ciliary dyskinesia (PCD). The CF treatment focuses specifically on the 10–13% of patients who have nonsense mutations that cannot fully benefit from current small molecule therapies.

Suliman concludes the wide-ranging conversation by discussing her status as one of a relatively small number of women CEOs in biotech, as well as assessing ReCode’s major goals and hopes, and hers over the next three to five years.

The post Shehnaaz Suliman, MD, MPhil, Discusses Targeted Respiratory Therapies on “Close to the Edge” appeared first on GEN - Genetic Engineering and Biotechnology News.

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Helen Sabzevari, PhD, president and CEO of cell and gene therapy developer Precigen, began her study of biology as a high school student in Iran, and continued her education in the U.S. after the Iranian Revolution of 1979.

On Episode 12 of “Close to the Edge,” Sabzevari recounts her life and career, which has seen her advance from a postdoc at Scripps Research Institute, to a researcher at the NIH’s National Cancer Institute, to positions at Merck & Co. and Compass Therapeutics before joining Precigen as it refocused on drug development.

As Precigen rebranded from Intrexon, it tapped its head of R&D Sabzevari to run the company. Precigen has since grown its pipeline to seven clinical-phase and nine preclinical cell and gene therapy candidates focused on cancer, as well as autoimmune disorders and infectious diseases, notably chronic Hepatitis B.

Sabzevari shares her perspective on the key challenges to cancer drug development, especially in immuno-oncology, and how Precigen has sought to address those challenges. Among challenges she discusses are those of manufacturing product and containing costs.

The company recently shared positive news on the clinical progress of five pipeline candidates. Three were developed using Precigen’s Ultra-CAR-T platform: PRGN-3005 being developed for advanced, recurrent platinum resistant ovarian cancer; PRGN-3006 for relapsed or refractory acute myeloid leukemia; and PRGN-3007, for which the FDA has cleared IND application to launch a Phase I/Ib open-label trial in advanced ROR1⁺ hematological and solid tumors.

The other two promising candidates are off-the-shelf immunotherapies developed using Precigen’s AdenoVerse platform, including PRGN-2009 in recurrent or metastatic human papilloma virus (HPV)-associated cancer, and PRGN-2012, which is directed against cells infected with HPV 6 or HPV 11 for treatment of recurrent respiratory papillomatosis (RRP).

Sabzevari concludes the wide-ranging conversation assessing the major goals and hopes she and Precigen have for the next five to 10 years.

The post Helen Sabzevari, PhD, of Precigen Connects Cancer and the Immune System on “Close to the Edge” appeared first on GEN - Genetic Engineering and Biotechnology News.

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Most guests on “Close to the Edge” are CEOs of companies in the biotech space, but Episode 11 features a slight exception: George Church, PhD, of Harvard Medical School has not only been a cutting-edge researcher for more than four decades, but also a serial entrepreneur who has co-founded more than three dozen companies, in fields that include genomics, gene editing, gene therapy, organoids, synthetic biology, and de-extinction.

On “Close to the Edge,” Church discusses the scope and scale of his famed lab, some of its current areas of research, and whether any of its numerous projects are being pursued despite an apparent lack of commercial potential.

Church also explains the drive that has prompted him to partner with others in co-founding 38 companies spun out from his lab to commercialize discoveries, many of which he discusses during our interview. They include:

• Colossal Laboratories and Biosciences, launched in September with $15 million in seed funding and a mission of de-extinction for the woolly mammoth and preservation of endangered Asian elephants
• Dyno Therapeutics, a developer of AI-based adeno-associated virus (AAV) vectors for gene therapy that launched potentially multi-billion-dollar collaborations with two biopharma giants, Novartis and Sarepta Therapeutics.
• Editas Medicine, a developer of genome editing therapies which Church co-founded with CRISPR pioneer and Nobel laureate Jennifer Doudna, PhD, among others.
• eGenesis Bio, which focuses on “xenotransplantation”—creating human-compatible organs through its multiplexed gene editing platform.
• GRO Biosciences (GRObio), which recently raised $25 million towards its goal of developing protein therapeutics using an expanded amino-acid alphabet. The company’s co-founders first connected with Church as postdoctoral students working in his lab.

Later on, Church offers his take on some of the ongoing challenges in molecular biology, including gene therapy dosing as well as CRISPR and other gene editing technologies. He concludes the interview by sharing his major hopes and goals for the next five to 10 years.

Church is a pioneer of genomics with numerous accomplishments in the areas of genome mapping, sequencing, editing, and writing. Since 1986, Church has been on the faculty at Harvard, where he is a professor of genetics at Harvard Medical School and a founding member of the Wyss Institute, for which he oversees the directed evolution of molecules, polymers, and whole genomes to create new tools with applications in regenerative medicine and bio-production of chemicals.

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