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Venture philanthropy is a type of impact investment that takes concepts and techniques from venture capital finance and business management and applies them to achieving philanthropic goals. [1] [2] The term was first used in 1969 by John D. Rockefeller III to describe an imaginative and risk-taking approach to philanthropy that may be undertaken by charitable organizations. [3]
For example, in 2000 The Chicago Public Education Fund became the only venture philanthropy in the United States focused on a single urban school district, which served as a catalyst and strategic investment partner for Mayor Richard M. Daley and four Chicago Public Schools (CPS) administrations. [4] Other examples of this type of venture philanthropy are New Profit Inc., the Robin Hood Foundation, Tipping Point Community, Cure Alzheimer's Fund, The Redstone Acceleration & Innovation Network (TRAIN) initiative from FasterCures, [5] the Asian Venture Philanthropy Network (AVPN), Social Ventures Australia (SVA) in Australia, [6] the danone communities, [2] and the European Venture Philanthropy Association (EVPA).
In the late 1990s the Bethesda-based Cystic Fibrosis Foundation (CFF), wanting to take more direct action toward finding treatments for cystic fibrosis (CF) beyond its traditional approach of funding basic research at universities, invested in a small California biotechnology firm to help fund the discovery and development of the drug that twenty years later (January 2012) was approved as Kalydeco. [7] [8] Unlike other drugs that were available that just address symptoms of CF, the drug candidate was intended to address the underlying cause of CF. [7] The company in which CFF invested was Aurora Biosciences; CFF provided $30 million for Aurora to identify and develop up to three drug candidates. [9] [10] The unusual nature of the arrangement was widely noted. [8] [9] [10] [11] [12]
In 2001, Aurora was acquired by Vertex Pharmaceuticals, [13] but CFF continued to fund development of the CF drug candidates. That funding eventually grew to $150 million, [13] much of which was raised for the CFF by Joe O’Donnell, a Boston businessman whose son died of CF. [14]
When Vertex started selling Kalydeco, it priced it at about $300,000 a year and promised to provide it free to anyone in the US who was uninsured or whose insurance would not cover it. It justified the price—one of the highest in the world for any drug—by explaining that on the one hand, that it can only treat about 4% of CF patients, or about 3,000 people worldwide; as it was only approved for adults and children six and older, there are only about 2,400 people eligible to receive it; with that few people, it needed a high price in order to pay for the research to create it as well as its other programs, which include a drug candidate that could treat many more people with CF. It also pointed out the strong efficacy of the drug, and laid out the costs of managing CF that would be saved for people that the drug could treat; those costs include repeated hospitalizations and lung transplants. [15]
Nonetheless the high price led to sharp criticism of Vertex and the CFF. [7] [15] Twenty-nine physicians and scientists working with people with cystic fibrosis wrote to the CEO of Vertex Pharmaceuticals to plead for lower prices. [16]
CFF made the investment in exchange for a promise of royalties paid on sales of any drug it funded that made it to market; in 2014 it sold the future royalty stream to Royalty Pharma, a royalty fund investment company, for $3.3 billion, and said that it would use the funds to invest yet more in research and clinical trials for cystic fibrosis treatments. [12] [13]
In the course of working out the deal with Aurora in 2000, CFF included a clause in the agreement that allowed them to take control of the intellectual property if Aurora stopped developing any drug that had been discovered. CFF struck a similar agreement with the company, Altus Pharmaceuticals, to fund development of a recombinant enzyme that could treat pancreatic disease in people with cystic fibrosis. When Altus reported to CFF that it did not have funds to continue developing the drug, CFF seized control of the asset and eventually licensed it to Alnara Pharmaceuticals, which developed the drug further and was acquired by Eli Lilly in 2010. [17] [18]
UW-Madison Professor Emeritus Kenneth Zeichner wrote a paper criticizing the role of the NewSchools Venture Fund [19] in bringing deregulation and market-based practices into schools in the US. [20] Lois Weiner writing in Jacobin criticized teachers' unions for taking money from the Gates Foundation. [21] Others have argued that venture philanthropy often suffers from a lack of accountability, with projects prioritized more for their measurable metrics than for genuine long-term change, leading to a cycle of publicity-driven initiatives, in particular in education, that fail to address the underlying systemic issues facing society. [22]
Cystic fibrosis (CF) is a genetic disorder inherited in an autosomal recessive manner that impairs the normal clearance of mucus from the lungs, which facilitates the colonization and infection of the lungs by bacteria, notably Staphylococcus aureus. CF is a rare genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. The hallmark feature of CF is the accumulation of thick mucus in different organs. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. Other signs and symptoms may include sinus infections, poor growth, fatty stool, clubbing of the fingers and toes, and infertility in most males. Different people may have different degrees of symptoms.
The Cystic Fibrosis Trust is a UK-based national charity founded in 1964, dealing with all aspects of cystic fibrosis (CF). It funds research to treat and cure CF and aims to ensure appropriate clinical care and support for people with cystic fibrosis.
Vertex Pharmaceuticals Incorporated is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headquarters in Boston, Massachusetts, and three research facilities, in San Diego, California, and Milton Park, Oxfordshire, England.
Delta Phi Epsilon is an international sorority founded on March 17, 1917 at New York University Law School in Manhattan. It is one of 26 social sororities that form the National Panhellenic Conference (NPC). It has 110 active chapters, three of which are located in Canada, making the sorority an international organization.
The Cystic Fibrosis Foundation (CFF) is a 501(c)(3) non-profit organization in the United States established to provide the means to cure cystic fibrosis (CF) and ensure that those living with CF live long and productive lives. The Foundation provides information about cystic fibrosis and finances CF research that aims to improve the quality of life for people with the disease. The Foundation also engages in legislative lobbying for cystic fibrosis.
Cystic Fibrosis Canada is one of national charitable but not-for-profit corporation established in 1960. Cystic Fibrosis Canada's mandate is to help individuals with cystic fibrosis, principally by funding cystic fibrosis research and care. The organization also provides educational materials for the cystic fibrosis community and the general public; undertakes advocacy initiatives with, and on behalf of Canadians with cystic fibrosis, and raises funds to support its programs.
Ivacaftor is a medication used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, who account for 4–5% cases of cystic fibrosis. It is also included in combination medications, lumacaftor/ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor which are used to treat people with cystic fibrosis.
Lumacaftor (VX-809) is a pharmaceutical drug that acts as a chaperone during protein folding and increases the number of CFTR proteins that are trafficked to the cell surface. It is available in a single pill with ivacaftor; the combination, lumacaftor/ivacaftor, is used to treat people with cystic fibrosis who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the defective protein that causes the disease. It was developed by Vertex Pharmaceuticals and the combination was approved by the FDA in 2015. As of 2015, lumacaftor had no medical use on its own.
Moderna, Inc. is an American pharmaceutical and biotechnology company based in Cambridge, Massachusetts, that focuses on RNA therapeutics, primarily mRNA vaccines. These vaccines use a copy of a molecule called messenger RNA (mRNA) to carry instructions for proteins to produce an immune response. The company's name is derived from the terms "modified", "RNA", and "modern".
A royalty fund is a category of private equity fund that specializes in purchasing consistent revenue streams deriving from the payment of royalties. One growing subset of this category is the healthcare royalty fund, in which a private equity fund manager purchases a royalty stream paid by a pharmaceutical company to a patent holder. The patent holder can be another company, an individual inventor, or some sort of institution, such as a research university.
Lumacaftor/ivacaftor, sold under the brand name Orkambi among others, is a combination of lumacaftor and ivacaftor used to treat people with cystic fibrosis who have two copies of the F508del mutation. It is unclear if it is useful in cystic fibrosis due to other causes. It is taken by mouth.
Aurora Biosciences was a biotechnology company founded in 1995 in San Diego to commercialize fluorescence assays based on Roger Y. Tsien's discoveries concerning green fluorescent protein and its uses in basic research - work for which Tsien eventually won the 2008 Nobel Prize in chemistry along with two other chemists.
Joshua S. Boger is an organic chemist and the founder of Vertex Pharmaceuticals Incorporated. He is considered a pioneer in the field of structure-based rational drug design. Drugs developed include amprenavir, an HIV protease inhibitor; telaprevir, a protease inhibitor for treatment of hepatitis C; and Kalydeco, for the treatment of cystic fibrosis. In 2003, Vertex was listed as one of forty worldwide Technology Pioneers by the World Economic Forum. As of 2012, Boger became executive chairman of Alkeus Pharmaceuticals.
Tezacaftor is a drug used for the treatment of cystic fibrosis (CF) in people six years and older, who have a F508del mutation, the most common type of mutation in the CFTR gene. It is sold as a fixed-dose combination with ivacaftor under the brand name Symdeko. It was approved by the U.S. FDA in 2018. The combination of elexacaftor, tezacaftor, and ivacaftor is being sold as Trikafta.
Jeffrey Leiden is an American physician, scientist and businessman who is the executive chairman of Vertex Pharmaceuticals, a biotechnology company based in Boston, Massachusetts. He was initially appointed to the board of directors of the company in 2009 and was CEO and president from February 2012 to March 2020.
Elexacaftor/tezacaftor/ivacaftor, sold under the brand names Trikafta and Kaftrio, is a fixed-dose combination medication used to treat cystic fibrosis. Elexacaftor/tezacaftor/ivacaftor is composed of a combination of ivacaftor, a chloride channel opener, and elexacaftor and tezacaftor, CFTR modulators.
Peter Grootenhuis was a Dutch-American Medicinal Chemist. Grootenhuis was the Project Leader and Co-Inventor of Ivacaftor (VX-770), the first CFTR potentiator FDA approved drug to treat the underlying cause of Cystic Fibrosis (CF) in patients with certain mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, who account for 4-5% of CF cases. Grootenhuis also led the Vertex team to subsequent discovery of Orkambi, the combination of Ivacaftor and Lumacaftor(VX-809), approved to treat CF in people with two copies of the F508del mutation. Most recently, Grootenhuis's team discovered Tezacaftor (VX-661) and Elexacaftor (VX-445), which in combination with Ivacaftor are the components of Trikafta, a drug approved by the FDA in 2019 to treat CF in more than 90% of CF patients. For Grootenhuis’ contributions to the discovery of these compounds, he was awarded the 2018 IUPAC Richter Prize, the American Chemical Society’s 2013 Heroes of Chemistry Award, and inducted into the American Chemical Society Division of Medicinal Chemistry Hall of Fame. Grootenhuis has contributed to the discovery of over 11 clinical candidates, co-authored more than 100 peer reviewed papers and is inventor of 65 + U.S Patents, and more than 50 EU Patents.
Paul Adrian Negulescu is an American–Romanian cell biologist. He is a Senior Vice President at American pharmaceutical company Vertex Pharmaceuticals. He received the 2022 Shaw Prize in Life science and medicine, together with Michael J. Welsh, for their work that uncovered the etiology of cystic fibrosis and developed effective medications.
Paul M. Quinton is an American physiologist.
