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Graphite Bio: gene editing blood stem cells for sickle cell disease


Ex vivo gene editing of hematopoietic stem cells using CRISPR–Cas9 and adeno-associated virus serotype 6 is ready for trials in people with sickle-cell disease.

Sickle cell disease has been a neglected area of drug development, with limited treatment options to prevent the associated debilitating pain and early mortality. With the maturation of cell and gene therapy, however, several cutting-edge treatments that have entered clinical testing aim to undo the damage caused by a mutation in the β-globin gene. Most existing gene therapy approaches supplement faulty production of β-globin with a compensating foreign gene—in the case of bluebird bio, for example, patients are given a lentiviral vector containing a T87Q-engineered form of the human β-globin gene that inserts at distant points in the genome and confers potent anti-sickling activity.


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Josh Lehrer, Graphite Bio CEO

June 25, 2021 09:37 AM EDTUpdated 10:41 AM


Graphite Bio leads this week's IPO squad, look­ing to turn the tide on sick­le cell dis­ease with gene edit­ing

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

Biotech’s IPO march continued earlier this week with Monte Rosa pricing on Thursday, and now Friday’s bunch heads to Nasdaq led by gene editing player Graphite Bio.

Located in South San Francisco, Graphite is going public Friday on the heels of a $238 million raise less than 10 months after getting started with a Series A. It’s been a rapid ascent, CEO Josh Lehrer told Endpoints News, but he feels the company is now at the point where it can focus both on drug development and further building out its platform.

Endpoints News

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October 11, 2021 06:00 AM EDT

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

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The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

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Omeros plunges deep­er af­ter FDA re­jects rare dis­ease drug, ask­ing for more in­for­ma­tion

Omeros practically warned investors that a complete response letter was coming when it disclosed that the FDA found deficiencies in its BLA for narsoplimab. But the agency did not elaborate on the specifics of those deficiencies for the drug, which was being positioned as a treatment for the rare but serious blood clotting disease known as hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).

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Reshma Kewalramani, Vertex CEO (YouTube)

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Endpoints News

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Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

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Senior Editor

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Through Graphite, Genus was quickly introduced to Helen Q., an innovation and strategy consultant with prior experience at McKinsey. With a strong background and experience in innovation across the corporate world, Helen proved to be the ideal candidate and helped Genus organize and implement a high-level strategy that transformed the organization.

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Introduction to Graphite Bio

Graphite Bio Launches with $45 Million Series A Financing

Next-generation gene editing company launched by Versant Ventures with additional backing by Samsara BioCapital

Core technology from Stanford founders Matt Porteus and Maria Grazia Roncarolo

Josh Lehrer joins as CEO

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Graphite Bio today announced a $45 million Series A financing led by founding investor Versant Ventures, together with Samsara BioCapital. Graphite Bio is a next-generation gene editing company focused on targeted DNA integration to precisely insert genetic payloads to treat a variety of severe diseases. The company was co-founded by pioneers in the fields of gene editing and gene therapy and has a rapidly maturing pipeline with an upcoming IND and several other candidates advancing toward the clinic in the next 12-18 months.

While many existing gene editing and gene therapy approaches are based on knocking out or randomly integrating genes, Graphite Bio is focused on correcting defective genes by high-efficiency site-specific integration of new genetic sequences. This technology has the potential to precisely repair a damaged portion of a gene, completely replace a malfunctioning gene while retaining normal regulatory control, or to insert a wide range of therapeutic genetic cargoes into precise regions of the genome.

In addition, Graphite Bio’s technology has the potential to provide for durable expression while minimizing toxicity from off-target insertions.

“Our flexible, site-specific approach is extremely powerful and could be used to definitively correct the underlying causes of many severe genetic diseases, and also is applicable to broader disease areas,” said Josh Lehrer, M.D., CEO of Graphite Bio. “With backing from Versant and Samsara, we look forward to progressing our novel medicines into the clinic for patients with high unmet needs.”

Dr. Lehrer joined Graphite Bio after six years at Global Blood Therapeutics (NASDAQ:GBT), where he most recently served as chief medical officer. At GBT, he played a key role in the clinical development and 2019 approval of the company’s Oxbryta therapy for sickle cell disease.

A next-generation gene editing platform

CRISPR-Cas9 gene editing has transformed the biotechnology landscape due to its ability to specifically induce double-stranded DNA breaks that can disrupt genes or genetic control regions, leading to novel treatment approaches for a range of diseases. However, it has been challenging to harness CRISPR-Cas9 for the high-efficiency insertion of new genetic cargo.

Graphite Bio’s gene editing platform includes several complementary technologies that enable targeted and permanent DNA integration at very high efficiency. The platform builds on seminal work led by Dr. Danny Dever in the laboratory of Dr. Matt Porteus at Stanford University demonstrating an increase in integration efficiency from less than 1% to greater than 50% across diverse genetic lesions in a wide range of cell types.

“Achieving high-efficiency targeted gene integration has been a critical objective of gene editing for more than 15 years, but only now is this technologically possible,” said Jerel Davis, Ph.D., Managing Director at Versant and a Graphite Bio board member. “As the founding investor of CRISPR Therapeutics, Versant has seen first-hand the rapid evolution of the gene editing field. Our collaboration with Matt Porteus on CRISPR Therapeutics was highly productive. We are thrilled to be working with him on Graphite Bio, alongside Maria Grazia, Josh and Danny.”

The efficiency improvements enable clinically meaningful levels of targeted integration for therapeutic applications. The company’s medicines therefore can replace defective genes, insert genetic cargo into specific loci and significantly limit undesirable, indiscriminate transgene expression.

Graphite Bio founders and operating plans

The company’s scientific founders include:

Matthew Porteus, M.D., Ph.D., Professor of Pediatrics and Stem Cell Transplantation and the Associate Director of Center for Definitive and Curative Medicine at Stanford University. Dr. Porteus is a pioneer in the gene editing field and has been involved in the space for more than 20 years. He is an academic founder of CRISPR Therapeutics, and advisor to a number of companies in the field as well as the FDA. He has advanced several of Graphite Bio’s technologies and programs at his Stanford lab. As a pediatric hematologist, Dr. Porteus treats patients with severe genetic diseases.
Maria Grazia Roncarolo, M.D., Ph.D., Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine, Director of the Center for Definitive and Curative Medicine, and Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine at Stanford University. She is a global leader and pioneer in gene therapy, having served as the director of the Telethon Institute for Cell and Gene Therapy at the San Raffaele Scientific Institute in Milan. There, she advanced multiple gene therapy products into the clinic. This work ultimately led to the first approved gene therapy, Strimvelis.
Based on progress to date, Graphite Bio expects to commence Phase 1 studies of its first development candidate in early 2021. The company’s lead program specifically corrects the single nucleotide point mutation in the sickle β globin gene. In cells from patients with the disease, Graphite Bio has shown that its gene correction approach efficiently restores healthy hemoglobin protein and eliminates sickle cell hemoglobin.

In addition, Graphite Bio is advancing several other programs to the clinic, each with the potential to lead to a cure for serious unmet needs.

“It is gratifying to see our work on new gene editing approaches being translated into novel therapies,” said Dr. Porteus. “I’m very excited to be working with Versant again on a start-up and I look forward to collaborating with Samsara and the Graphite Bio team to bring a new generation of genetic treatments to patients.”

In connection with the financing, Abe Bassan, Vice President at Samsara BioCapital, and Carlo Rizzuto, Ph.D., Partner at Versant, joined Graphite Bio’s board.

About Graphite Bio

Graphite Bio is a next-generation gene editing company focused on targeted DNA integration to precisely insert genetic payloads to treat a broad range of severe diseases. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, and is backed by Versant Ventures and Samsara BioCapital. For more information, please visit

About Versant Ventures

Versant Ventures is a leading healthcare venture capital firm committed to helping exceptional entrepreneurs build the next generation of great companies. The firm’s emphasis is on biotechnology companies that are discovering and developing novel therapeutics. With $3.2 billion under management and offices in the U.S., Canada and Europe, Versant has built a team with deep investment, operating and R&D expertise that enables a hands-on approach to company building. Since the firm’s founding in 1999, more than 75 Versant companies have achieved successful acquisitions or IPOs. Versant is currently investing out of its seventh fund, Versant Venture Capital VII, a $600 million global biotech fund closed in December 2018. In parallel the firm co-invests out of its Canadian strategic fund Versant Voyageurs I and its later-stage biotech opportunity fund Versant Vantage I. For more information, please visit

About Samsara BioCapital

Samsara BioCapital is a new breed of biotech investment fund focused on translating cutting-edge biology into new therapies to treat patients with unmet medical needs. Founded in 2016 by Srinivas Akkaraju, MD, PhD, our team of scientists, investors, and entrepreneurs takes a long-term view to value creation across all stages of public and private life science companies. We believe in a collaborative, hands-on approach, working closely with entrepreneurs to harness exciting scientific advances and build leading companies. Samsara actively manages approximately US$410 million in assets on behalf of endowments, foundations, and family offices. For more information, visit

Katherine Vega Stultz, COO
[email protected]


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Intro to Graphite Bio Part 2

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