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As a postdoctoral researcher at the Broad Institute of MIT and Harvard, Nicole Gaudelli was the co-inventor of a breakthrough in gene editing: the ability to target a single misspelling in DNA code. This invention, known as base editing, is a further advancement of the Nobel Prize-winning Crispr technology and has the potential to cure thousands of rare diseases caused by point mutations. She then joined Beam Therapeutics to, in her words, “make the dream of having that technology become a reality for patients really materialize.”
With clinical trials now underway at Beam for patients with sickle cell disease and a rare liver disease known as alpha-1 antitrypsin deficiency, Gaudelli hopes to embark on a new journey from the bench to the bedside. Today GV (formerly known as Google Ventures) announced Gaudelli will be the firm’s latest entrepreneur-in-residence in life sciences. “I just wanted to do it again,” she told Forbes. “I’m really motivated by having the ability to impact patients’ lives.”
GV was a natural fit, said general partner Issi Rozen, since Gaudelli’s base editor is being used by two of the fund’s portfolio companies – Beam and Verve Therapeutics, which is targeting cardiovascular disease. “It’s been a great way for us to bet on really talented people that we believe can start very interesting companies,” Rozen told Forbes. “We can take longer time horizons and take on more risky projects. Because it’s kind of like an incubation with a supportive family around you that helps you through the different steps, different decisions.”
Late last year, the FDA approved the first-ever Crispr-based therapy: Casgevy, a sickle cell disease treatment co-developed by Vertex and Crispr Therapeutics. Gaudelli says she continues to be “inspired and motivated” by the field of gene editing, predicting “a new wave of applications of this powerful tool.” She said she plans to explore opportunities for gene editing in new disease areas and also the possibilities for genetically engineering cells.
The Startup Behind The First Pig-Human Kidney Transplant Is Targeting Hearts And Livers Next
Last Thursday, surgeons transplanted a pig kidney into a living patient for the first time. The kidney was provided by eGenesis, a startup which has developed gene-edited pig organs to solve the problem of the severe shortage of organs available for donation. “We’ve shown that we actually have something that can help patients,” the company’s CEO Mike Curtis told Forbes. “To me it’s all about getting into this new era of science and the ability to help people that have very few treatment options.”
In addition to kidneys, the company also developed gene-edited hearts and livers for transplant. Curtis said he expects clinical trials for liver to start this year and kidneys next. He also hopes that, with more human data, gene-editing on organs can be refined to the point where patients don’t need to be on a regimen of medication to prevent organ rejection.
Read more here.
Pipeline & Deal Updates
Nutrition: Telenutrition platform Nourish, cofounded by alumni for the Forbes 30 Under 30 list, announced it has raised a $35 million series A round led by Index Ventures.
Brain Implant: NeuroOne Medical Technologies has begun a commercial launch of its OneRF system, which consists of a neural implant that can record the brain’s electrical activity and also treat patients through the ablation of brain tissue.
Big Pharma M&A: Novo Nordisk is acquiring RNA therapeutics company Cardior in a deal potentially worth up to $1.1 billion. Abbvie is acquiring autoimmune therapeutics company Landos Biopharma in a deal worth up to $212.5 million.
Oncology: Avenzo Therapeutics, which is developing treatments for metastatic breast cancer and other tumors, announced it’s raised a $150 million series A-1 round with an aim of advancing its therapeutic pipeline.
Mental Health: Brightside Health, which uses telehealth tools to treat patients with anxiety, depression and other mental health issues, announced it has raised a $33 million series C round led by S32.
FDA Approves Merck’s New Drug For A Rare Lung Disease
On Tuesday, the Food and Drug Administration approved a new drug to treat pulmonary arterial hypertension, a rare, progressive disease that primary affects women. The drug, called sotatercept, was developed and is being marketed by pharmaceutical giant Merck under the name Winrevair. This is the first new type of treatment for the disease approved in nearly a decade, and is considered by the FDA to be “first in class.” In a clinical trial, the drug reduced the risk of death or increased severity of the disease by 83%.
Read more here.
Other Healthcare News
Supreme Court justices suggested they’re unlikely to roll back federal approvals of abortion drug mifepristone during hearings this week, but it may be several months before they issue a final ruling.
Amazon Pharmacy is launching same-day prescription delivery in New York and Los Angeles.
U.K. searches for cancer on the public health system’s website surged by 373% following the news of Kate Middleton’s diagnosis.
Altimmune’s experimental weight loss drug pemvidutide minimized the loss of muscle mass while losing weight, according to results of a small trial.
With RxSense, Rick Bates tries to find consumers the best deals on their medicines, but now billionaire Mark Cuban is elbowing in.
Across Forbes
Here’s How Much Donald Trump Is Worth
The Rise Of Crypto’s Billion Dollar Zombies
This Deep-Sea Mining Company Will Sweep The Ocean Floor For Battery Materials—If It Doesn’t Go Broke First
What Else We are Reading
During the pandemic, were great vaccines bad business? A company-by-company review (STAT)
Overdosing on Chemo: A Common Gene Test Could Save Hundreds of Lives Each Year (KFF Health News)
How to make an old immune system young again (Nature)
