Category Archives: Genetic Therapy

The COVID-19 messenger RNA (mRNA) experimental vaccine from Moderna (NASDAQ: MRNA) is one of the most promising vaccine candidates for the disease.

Its success could serve as a proof-of-concept for several other mRNA projects at other publicly-traded companies, such as Pfizer (NYSE: PFE), BioNTech (NASDAQ: BNTX), CureVac (NASDAQ: CVAC) and Translate Bio (NASDAQ: TBIO).

Our genetic information is stored in DNA molecules in the nuclei of our cells. DNA contains encoded instructions for making various proteins used in the creation or maintenance of cells.

But the ribosomes the cell organelles which make proteins cant actually read DNA. They can only read mRNA, an intermediary form of nucleic acid, which transcribes genetic information from DNA and carries it throughout the cell.

In recent years, scientists have started to experiment with using mRNA as a vector to transmit genetic information to cells, which can teach them how to fight a virus.

In other words, theyve been looking to use mRNA as a vaccination method.

According to the University of Cambridges PHG Foundation, mRNA vaccines could be faster, cheaper and safer to produce than conventional vaccines - which typically contain weakened or modified forms of a pathogen which the patients immune system uses as target practice.

There are currently no mRNA vaccines approved for human use in the U.S. Moderna is looking to change that with MRNA-1273, its mRNA-powered COVID-19 vaccine candidate.

MRNA-1273 is widely viewed as the frontrunner in the race for a COVID-19 vaccine. Moderna reported last week that it has completed enrollment for its 30,000-participant late-stage trial of the vaccine candidate. And unlike Johnson & Johnson (NYSE: JNJ) or AstraZeneca (NYSE: AZN), it has not yet had to halt trials over safety concerns.

If Moderna successfully brings a mRNA vaccine to market especially if its among the first COVID-19 vaccines available to the public it could bring a surge of interest to mRNA technology in general.

And its not the only biotech companyworking on an mRNA-based project. There are at least three other publicly-traded firms offering investors exposure to this emerging technology.

Moderna isnt even the only company working on a COVID-19 vaccine candidate which uses mRNA technology.

Pfizer and BioNTech are also developing BNT162, a mRNA-based COVID-19 vaccine candidate which is currently in global Phase 2/3 trials.

BNT162b2 isnt quite as far along in the drug development process as MRNA-1723. But it received Fast Track designation from the U.S. Food and Drug Administration (FDA) in July, making it a strong contender to be the second or third mRNA-based vaccine on the market.

CureVac is also working on a mRNA-based COVID-19 vaccine,as well as a mRNA-based vaccine for rabies.

The biotecg company has been working on an unnamed mRNA-based COVID-19 vaccine platform along with the nonprofit Coalition for Epidemic Preparedness Innovations (CEPI) since the viruss genome was published in early January. It is currently in Phase 2 clinical trials.

It developed that platform based on positive early data from its mRNA-based rabies vaccine candidate, CV7202, which is currently in Phase 1 trials.

A new cystic fibrosis drug candidate from Translate Bio provides an example of a non-vaccine drug built on mRNA technology.

Translate Bio recently presented data from its MRT5005 drug candidate at the 34th Annual North American Cystic Fibrosis Conference (NACFC). Its a mRNA-based gene therapy which seeks to help patients malformed lung cells produce essential proteins correctly.

MRT5005, which is currently in Phase 1/2 clinical trials, has also won Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA.

Moderna shares traded up 8.38% to $71.23 on Thursday afternoon. Translate Bio shares, meanwhile, traded up 4.33% to $13.50 the same day. Pfizer shares traded up 0.19% to $35.51, BioNTech traded up 8.85% to $84.40, and CureVac traded up 6.01% to $49.57.

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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4 Biotech Companies Working On RNA Therapies - Benzinga

Will report updated durability data from Phase 1/2 B-AMAZE trial in Haemophilia B in Q4

Third wholly-owned programme expected to be in the clinic in 2021

Strong cash position with over $250 million at 30 September

LONDON, Oct. 29, 2020 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline), a clinical-stage, fully integrated, next generation, systemic, liver directed, AAV-based gene therapy company today announced its financial results for the first half of 2020 and provided a business update.

2020 has been a transformative year for Freeline, marked by encouraging clinical progress in our Haemophilia B and Fabry programmes, a strengthened management team and the successful completion of our Series C round and Nasdaq initial public offering, said Theresa Heggie, Chief Executive Officer of Freeline Therapeutics. With a strengthened balance sheet, we are now well positioned to achieve upcoming clinical milestones, which include the presentation of updated data from our B-AMAZE trial for Haemophilia B by the end of this year evaluating FLT180a, the only gene therapy candidate showing durable Factor 9 (FIX) activity in the normal range. In 2021 we expect to continue our progress in the clinic with the initiation of a pivotal trial in Haemophilia B, continued progress in our MARVEL1 study in Fabry disease, and the initiation of our Phase 1/2 trial for Gaucher disease. On the preclinical front, we will continue to advance our potentially best-in-class Haemophilia A programme. Importantly, all these programmes are wholly owned and leverage Freelines proprietary AAVS3 capsid which has been engineered for high potency to achieve durable, functional cures by achieving normal levels of protein expression at doses lower than those seen in wild-type AAV constructs.

Business Highlights and Upcoming Milestones

Haemophilia B

Fabry Disease

Gaucher Disease

Haemophilia A

Manufacturing and CMC Platform

Corporate

First Half 2020 Financial Highlights

Total operating expenses increased by approximately $11.4 million from $29.7 million for the six months ended June 30, 2019 to $41.1 million for the six months ended June 30, 2020.

Research and development expenses increased by approximately $5.8 million from $23.6 million for the six months ended June30, 2019 to $29.4 million for the six months ended June30, 2020. The increase in research and development expenses was primarily attributable to an increase in spending on FLT180a specifically related to the ongoing Phase 1/2 B-AMAZE clinical trial and planned pivotal clinical trial as well as unallocated costs for all research and development activities including increased discovery costs.

General and administrative expenses increased by $5.3 million from $6.0 million for the six months ended June30, 2019, to $11.3 million for the six months ended June30, 2020. The increase in general and administrative expenses was primarily attributable to increased legal and professional fees, and personnel costs.

Total other income, net was $7.8 million for the six months ended June30, 2020, compared to $5.1 million for the six months ended June30, 2019. During the six months ended June30, 2020 and 2019, Freeline recognised an R&D tax credit from the U.K. as a benefit within other income for approximately $6.9 million and $5.1 million, respectively.

In June 2020, Freeline received net cash proceeds of approximately $69.0 million from the sale of series C preferred shares. During the six months ended June30, 2020, net cash provided by financing activities was $67.9 million, consisting of net cash proceeds from the sale and issuance of series C preferred shares.

During the six months ended June30, 2020, net cash used in operating activities was $30.7 million, primarily resulting from a net loss of $33.3 million, adjusted for non-cash charges of $0.9 million for depreciation and $0.6 million of non-cash share-based compensation. The net loss was also partially offset by $0.9 million related to changes in components of working capital, including a $9.0 million increase in prepaid expenses and other assets and a $9.9 million increase in accounts payable and accrued expenses, all of which relate to increased research and development spending on preclinical and clinical trials and increased general and administrative spending resulting from increased professional and legal expenses incurred in conjunction with preparation for becoming a public company.

On July 1, 2020, Freeline received an aggregate of approximately $10.0 million in additional proceeds from the sale of series C preferred shares, bringing the total net proceeds of the Series C financing to $79.0 million.

On August 11, 2020, the Company completed a Nasdaq IPO of 8,823,529 American Depositary shares (ADSs) representing the same number of ordinary shares at an IPO price of $18.00, for total net proceeds of approximately $147.7 million. On August 20, 2020, the underwriters of the IPO exercised a portion of their overallotment option by purchasing an additional 1,128,062 ADSs representing the same number of ordinary shares for additional net proceeds of $18.9 million. The total net proceeds from the IPO was $166.6 million.

As of June30, 2020 and September 30, 2020, Freeline had cash and cash equivalents of $106.6 million and $252.6 million, respectively.

As of September 30, 2020, the Company had 35,872,749 ordinary shares outstanding.

Freeline anticipates transitioning to quarterly reporting of financial results in 2021.

About FreelineFreeline is a clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Its vision is to create better lives for people suffering from chronic, systemic diseases using the potential of gene therapy as a one-time treatment to provide a potential functional cure. Freeline is headquartered in the U.K. and has operations in Germany and the U.S.

About HaemophiliaHaemophilia is a genetic bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor. In Haemophilia A, there is a deficiency of the clotting factor VIII (eight) protein and in Haemophilia B, there is a deficiency of the clotting factor IX (nine) protein. Haemophilia mainly affects boys and men; however, women can be carriers of the affected gene and may experience symptoms. Haemophilia A is the most common type of Haemophilia affecting about one in every 5,000 males, while Haemophilia B affects about one in every 30,000 males. Haemophilia is classified as mild, moderate or severe, depending on the level of clotting factor VIII or IX in the blood and is diagnosed through blood tests.

About FLT180aThe Freeline Haemophilia B programme, FLT180a, uses a synthetic AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is currently being studied in a Phase 1/2 trial, B-AMAZE, with the goal of normalising FIX activity in patients with moderate and severe Haemophilia.

About Fabry DiseaseFabry Disease is an inherited, X-linked disease characterised by the progressive accumulation of glycosphingolipids in lysosomes throughout the body. It is caused by mutations in the gene encoding of the -galactosidase A enzyme (GLA) responsible for the breakdown of globotriaosylceramide (Gb3), a type of glycosphingolipid.

The condition ranges from mild to severe and may appear anytime from childhood to adulthood. The progressive accumulation of Gb3 leads to organ damage, major disability and often early mortality. Symptoms and signs include neuropathic pain, impaired sweating, gastrointestinal symptoms, renal failure, heart disease and increased risk of stroke. Current treatment consists of Enzyme Replacement Therapy and chaperone therapy to temporarily clear Gb3 accumulation and alleviate symptoms.

About FLT190FLT190 is an investigational liver-directed adeno-associated viral (AAV) gene therapy for the treatment of Fabry Disease. We believe the programme is the first clinical-stage AAV gene therapy international study in Fabry Disease. FLT190 is an in-vivo gene therapy administered by a one-time intravenous infusion.

The study, named MARVEL-1, is a multi-centre, international, dose-finding Phase 1/2 study in adult males with classic Fabry Disease. The study is focused on assessing the safety of FLT190 and its ability to transduce liver cells to produce continuous high levels of GLA. In addition to safety, endpoints in the study include clearance of Gb3 and LysoGb3 from the plasma and urine, baseline renal and skin biopsies (repeated in long term follow up), renal and cardiac function, GLA immune response, viral shedding and quality of life.

About Gaucher DiseaseGaucher Disease is a genetic disorder in which a fatty substance called glucosylceramide accumulates in macrophages in certain organs due to the lack of functional GCase. The disorder is hereditary and presents in various subtypes. Freeline is currently focused on Type 1 Gaucher Disease, the most common type, which impacts the health of the spleen, liver, blood system and bones. The current standard of care is intravenous infusion of ERT every two weeks, which is a significant treatment burden on the patient. The aim of Freelines investigational gene therapy programme is to deliver a one-time treatment of a long-lasting gene therapy that will provide a consistent and therapeutically relevant level of constant endogenous GCase, thus eliminating the need for ERT.

About FLT201FLT201 is an investigational liver-directed adeno-associated viral (AAV) gene therapy in preclinical development for the treatment of Type 1 Gaucher Disease. By using Freelines high-transducing AAVS3 capsid, our goal is to deliver GBA endogenously at normal levels with a one-time treatment. FLT201 contains a liver-specific promoter and a GBA sequence which expresses our novel, proprietary GBA variant with a greater than 20-fold longer half-life than wild-type GBA protein in purified systems. To our knowledge, we are the only company to date that has announced a programme for the development of an AAV gene therapy for the treatment of Type 1 Gaucher Disease and plan to file an IND for this programme in 2021.

Forward-Looking StatementsThis press release contains statements that constitute forward looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the Companys opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. Examples include discussion of the Companys strategies, financing plans, and clinical trial plans. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project or expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work; the Companys ability to design and implement successful clinical trials for its product candidates; the potential for a pandemic, epidemic or outbreak of an infectious diseases in the U.S., U.K. or EU, including the COVID-19 pandemic, to disrupt the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience which could result in delays in the development or commercialisation of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalise on programmes or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law.

For further information, please reference the Companys reports and documents filed with theU.S. Securities and Exchange Commission. You may get these documents by visiting EDGAR on theSECwebsite atwww.sec.gov.

Further information

JW CommunicationsJulia Wilson+44 (0) 7818 430877juliawilsonuk@gmail.com

The following tables summarize our results of operations for the six months ended June 30, 2020 and 2019, as well as certain other financial information (U.S. dollar amounts in thousands):

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Freeline Reports Half Year 2020 Financial Results and Recent Business Highlights - GlobeNewswire

The original is unfaithful to the translation, Jorge Luis Borges

Investing in new innovative RNA technologies can lead to rapid value creation, and Europe is a fertile ground to hunt for them. The discovery of DNA and the gene underlies much of modern medical therapy, and with the recent advent of successful gene therapies, is undergoing a renaissance. Only in the last couple of decades, however, has the translator of DNARNA, gained appreciation as a potential inroad for new therapies and led to the creation of several multibillion-dollar companies. While the field of RNA therapy has started to blossom recently, it, much like biotechnology companies in Europe, has underappreciated potential.

The main purpose of RNA, known as messenger RNA or mRNA, is to convert (or translate) the genetic information of DNA into proteins. There are many other forms of RNA now recognized, and each a potential target for therapies to combat disease and illness in ways considered undruggable until now. A short list would include the protein synthesis RNAs: messenger RNA (mRNA, Moderna- Covid-19 Vaccine), transfer RNA (tRNA), ribosomal RNA (rRNA), and small nuclear RNAs (snRNA), and regulatory RNAs: micro RNA (miRNA) and short interfering RNA (siRNA). The applications of these RNA species is for therapeutics for various treatment applications, including cancer, cardiovascular disease, kidney disease, infectious disease, metabolic disease and more.

Examples of RNA therapies already approved include: Macugen (approved 2004; Pfizer) for the treatment of AMD, Exondys 51 (approved 2016; Sarepta Therapeutics Inc) for the treatment of Duchenne muscular dystrophy; Spinraza (December 2016; Biogen) approved for the treatment of spinal muscular atrophy in children and adults, and Onpattro (August 2018; Alnylam Pharmaceuticals) approved for the treatment of hATTR in adults.

The major advantages of targeting RNA are specificity and therapeutic leverage. One of the major barriers to developing any new drug is the safety profile and potential side effects. While a given drug may affect the target you want, there is always potential that it will also affect other targets. This is less true for RNA as its made of a specific sequence of 1 of 4 nucleic acids, the longer the matching sequence the lower the chance of targeting the wrong RNAand mathematically, this occurs very rapidly as each nucleic acid is added to the molecule. This is essentially the same technique that another new breakthrough technology such as CRISPR (also an RNA-guided technology) relies on for targeting. Further, as mRNA is upstream of much of the activity of making a protein, one does not need as much material to achieve a large effect.

The major obstacle in the development of these technologies, DNA, RNA, gene therapy, etc., turns out to be getting the oligonucleotide (DNA or RNA) to its intended target cell. The development of oligonucleotide therapeutics is challenging since DNA/RNA is inherently unstable and prone to degradation, immunogenic and rapidly cleared, and requires safe and effective delivery. To date, therefore, the greatest successes have been in easy to reach or target organsspecifically the liver, eye and muscle.

In my recent diligence, I have come across two new RNA technologies, being pioneered for clinical use by European and/or Israeli companies that seek to overcome the above-stated challenges.

The first technology is known as DNAzymeusing sequences of DNA that contain two factors: first, a sequence of oligonucleotides that will match a specific sequence of RNA (specificity) and the second, when this sequence is matched, it activates an internal loop of the DNA that can act to cleave the target RNA, and it can do this repeatedly, like an enzyme (therapeutic leverage). Instead of using oligonucleotides to match a given sequence of RNA, bind to it, and thus turn off its activity, DNAzyme, because it can be used repeatedly to target and cleave the same sequence of RNA has an increased advantage in therapeutic leverage. This allows for smaller amounts needed to achieve an effect, and, therefore, potentially improves the range of delivery technologies available and potential targets.

A second innovative RNA technology that has reached the clinic is short activating RNA. Short interfering RNA has been used by pioneering companies like Alnylam. Essentially, a given short RNA sequence can be used to convince cells to reduce or turn off the production of a protein. It turns out that similar short sequences of RNA can also convince a cell to turn on or produce more of a protein. Thus, short activating RNAs (saRNA) are a new approach being used to turn on targeted proteins enabling an increase in translation, if you will. saRNA opens a whole new landscape of potential therapies by turning on genes and increasing protein production.

European and Israeli companies are pioneering the clinical application of these technologies partly due to the fact that the intellectual property around earlier technologies is already well established and thus there is a need to find new technologies. Regardless, the fact that Israel and Europe are innovating in this area presents an interesting dynamic for investors. On the one hand, these companies often need greater management depth and might require more investor time and effort, on the other hand, the valuations can be lower and there is less capital available for later-stage investing, allowing for greater deployment at better terms.

As companies continue to demonstrate success with RNA therapeutics, especially as Moderna or BioNtech may soon have an approved Covid-19 vaccine using mRNA, I believe that we will see rapid expansion of RNA-targeted therapeutics and value creation. Additionally, as the arsenal of RNA technologies increases, we will see greater ability to target yet-undruggable proteins with these young companies providing a large platform for patient cures and investor returns.

Photo: Abscent84, Getty Images

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Gains in tRNAslation: It's time for investors to realize the potential of European RNA technologies - MedCity News

Psychologist Michael Siegrist and nutrition scientist Christina Hartmann have compiled research on the psychological and societal factors that influence consumer acceptance of food technologies. According to a 2015 survey of more than 30,000 consumers, the most desirable food attributes are freshness, naturalness and minimal processing Many consumers perceive the use of food technologies as contradictory to healthy, nutritious food, which may be a challenge for the industry, says Siegrist.

When scaled up, lab-grown meat may offer a more sustainable and animal-friendly alternative to livestock rearing. But, once again, a 2018 survey showed that the public isnt quite ready for it. Survey respondents had a low understanding of the technology and a low level of acceptance. When the production process was explained to them, it actually reinforced the acceptance of traditionally reared meat.

Cultured meat evokes a disgust response in many people because its perceived as artificial, says Siegrist. He adds that evolutionary psychology might help to explain this. Instead of relying only on our immune system to fight infections in our body, our behavioural immune system has evolved to evoke a feeling of disgust or a neophobic response to unknown foods that may have harmed us in the past. The very same behavioural constructs may be impacting peoples willingness to accept novel food technologies in the modern day.

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Neophobia: The psychological barrier that inspires knee-jerk rejection of GMOs and other food technology - Genetic Literacy Project

Newswise Mass Eye and Ear is proud to announce the successful conclusion of its historic campaign, Bold Science. Life-Changing Cures. which raised $252M from philanthropy to advance research to treat and cure diseases of vision, hearing, and the head and neck.

The campaign was led by co-chair Wyc Grousbeck, Boston Celtics CEO and Lead Owner and former Chairman of Mass Eye and Ear. Grousbeck was elected Chairman of the Foundation and Board of Directors of the Massachusetts Eye and Ear in 2010, and stepped down from the position earlier this year. He will continue to serve as a trustee and contribute to advancing Mass Eye and Ears mission, with a lasting legacy that includes two endowed chairs and a gene therapy research center he and his family established.

The campaigns finale culminated in a virtual celebration as part of the hospitals annual Sense-ation! Gala on Wednesday, October 28, 2020 at 7:30 PM EST. This years gala was held online due to the COVID-19 pandemic, and was hosted by longtime Mass Eye and Ear friends, Billy Costa, (Co-Host of KISS 108s Matty in the Morning and NESNs Dining Playbook) and founding gala chair Joey McIntrye (New Kids on the Block).

[Watch the entire 11th Annual Sense-ation Gala!]

Bold Science. Life-Changing Cures. Ends with Enduring Legacy

The Bold Science. Life-Changing Cures. campaign was launched with the goal of expediting cures for blindness, deafness and diseases of the head and neck by investing in three key areas: people, programs and places (our facilities). Through this campaign, the generosity of donors and organizations infused significant financial resources into Mass Eye and Ears cutting-edge research programs, and supported the facilities needed by the leading physicians and scientists in Ophthalmology and Otolaryngology-Head and Neck Surgery to conduct their world-class work.

The campaign resulted in a powerful and far-reaching impact on research at Mass Eye and Ear by faculty, including:

Additionally, new Research Centers were launched in gene therapy, tinnitus, and head and neck cancer and two world-class surgical training laboratories were built.

The campaign also will leave a lasting legacy of fourteen endowed Chairs for leading physicians and scientists that will remain for generations. The endowment of a Chair provides financial support for researchers, allowing them to focus on their efforts to develop treatments and cures.

Wyc Grousbeck Honored for Dedication to Mass Eye and Ear During 10 years as Chairman

Grousbeck, who served as Board Chairman at Mass Eye and Ear for the past decade, was honored at this years Sense-ation! Gala for his many contributions. During his tenure, he led the organization through remarkable growth, both clinically and as a world-renowned research center. Mass Eye and Ear surgical volume doubled and the number of clinical locations grew from 9 to 21 throughout the region. Grousbecks leadership played critical roles in two highly successful mergers: Schepens Eye Research Institute joining Mass Eye and Ear in 2012 and Mass Eye and Ear joining Mass General Brigham (formerly Partners HealthCare) in 2018. He also initiated and served as co-chair of the Bold Science. Life-Changing Cures. campaign.

Wycs leadership during the campaign has been nothing short of magnificent; he has showed us anything is possible, said John Fernandez, President of Mass Eye and Ear. When we first reached our $100M goal in the campaign, Wyc called on us to double that amount by 2020. With Wycs guidance, we were able to exceed that ambitious goal by more than $50M by the campaigns end.

Grousbeck first became involved with Mass Eye and Ear because his family has been touched by blindness, and since then, they have been devoted to research and educational initiatives relating to blindness and other vision-related conditions for many years.

Grousbeck and his familys philanthropic support established the Grousbeck Center for Gene Therapy at Mass Eye and Ear as well as the Grousbeck Family Chair in Gene Therapy, held by Luk H. Vandenberghe, PhD. Additionally, Wyc and his wife, Emilia Fazzalari, funded the Fazzalari-Grousbeck Chair in Otolaryngology-Head and Neck Surgery held by Tessa Hadlock, MD. Most recently, they seeded Dr. Vandenberghes work to develop an experimental and novel genetic vaccine for COVID-19.

Grousbeck was a driving force around establishing the first Sense-ation Gala 11 years ago, which brought celebrities, musicians, philanthropists, and scientists together to help raise funds for Mass Eye and Ear research and patient care. The gala to date has raised more than $12M for the Mass Eye and Ear Curing Kids Fund.

About Mass Eye and Ear

Massachusetts Eye and Ear, founded in 1824, is an international center for treatment and research and a teaching hospital of Harvard Medical School. A member ofMass General Brigham, Mass Eye and Ear specializes in ophthalmology (eye care) and otolaryngologyhead and neck surgery (ear, nose and throat care). Mass Eye and Ear clinicians provide care ranging from the routine to the very complex. Also home to the world's largest community of hearing and vision researchers, Mass Eye and Ear scientists are driven by a mission to discover the basic biology underlying conditions affecting the eyes, ears, nose, throat, head and neck and to develop new treatments and cures. In the 20202021 Best Hospitals Survey,U.S. News & World Reportranked Mass Eye and Ear #4 in the nation for eye care and #6 for ear, nose and throat care.For more information about life-changing care and research at Mass Eye and Ear, visit our blog,Focus, and follow us onInstagram,TwitterandFacebook.

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Mass Eye and Ear Capital Campaign Concludes with $252 Million Raised to Advance Research and Patient Care - Newswise

Vertex Pharmaceuticals(NASDAQ:VRTX) is a pharmaceutical company that has utilized its portfolio of medicines for cystic fibrosis (CF) to grow consistently, expanding its trailing revenues by more than 423% over the last five years. But Vertex's stock has underperformed the market this year. The company's heavy focus on CF treatments has historically paid off, but the strategy may carry longer-term risks in part because Vertex's drugs will eventually lose their patent exclusivity. Is Vertex approaching the end of its run as a stalwart growth stock, or does it still have some gas in the tank for new investors?

I expect that Vertex will continue to grow for quite some time, and that it will return to outperforming the market relatively soon. But the factors driving that future success probably won't be quite the same as those that have carried it to this point. Here are three reasons that Vertex could be worth buying and holding for the long term -- and one reason why investors might want to be cautious.

Image source: Getty Images.

Vertex earned practically all of its $5.4 billion in revenue over the past year from sales of its life-saving medicines for CF -- a relatively rare and difficult illness. About 100,000 people around the world have CF, and in the next five years, the company aims to expand its reach to at least 90% of those patients. The company is already within spitting distance of that goal thanks to the reach of its drugs as they are currently being marketed, and regulatory approvals for new combinations of those treatments are expected in the near term. Vertex's integration into the CF community is substantial, and its ability to market products within the group is quite impressive. Within a year of launching its drug Trikafta, the company claimed that the "vast majority" of the 18,000 eligible patients in the U.S. had enrolled in treatment.

Furthermore, Vertex has an even larger ambition to reach 100% of people with CF by creating new genetic therapies that will help the estimated 10% of patients with variants of the disease that can't be effectively treated with its current offerings. At present, Vertex sells four different medications for CF, three of which are combination therapies utilizing different drugs like lumacaftor and ivacaftor, which were developed in-house. By combining its successful therapies into new formulations, Vertex can address different needs within CF patients while also extracting the maximum amount of value from its prior research and development (R&D) efforts. This also allows Vertex to avoid some of the revenue damage inflicted by patent expiration. On the whole, this has proven a profitable strategy. Without a doubt, Vertex is poised to maintain its competitive position in the CF market.

Through a strategy of pursuing expanded regulatory approvals that make its drugs available to ever-wider swaths of the CF patient population, Vertex has achieved impressive revenue growth. Year over year, its quarterly revenue for the period ended June 30 rose by 62%, and its earnings expanded by a shocking 213.1%. Importantly, the company has been consistently profitable, and sports a margin of 38.51%. It could pay off its small debt burden instantly with cash on hand if it so chose. All of those factors make it quite a favorable investment in comparison to many of its pharmaceutical industry peers.

Vertex also has a pipeline treatment -- CTX001 -- that's undergoing phase 1 clinical studies for both sickle cell disease (SCD) and beta thalassemia. It's something of a departure from the company's typical fare both in terms of the diseases targeted and the therapeutic modality. While Vertex's therapies for CF are based on traditional pharmacology, CTX001 uses genetic editing technology and is being developed in collaboration with CRISPR Therapeutics (NASDAQ:CRSP).

Investors should be pleased with Vertex's efforts to break into new markets with long-term growth potential. By working with CRISPR Therapeutics, Vertex can also gain experience with genetic therapies, which have been proposed as a potential route to advanced treatments for CF, and even a cure for the disease. In other words, Vertex's new gene therapy projects might help it develop a groundbreaking treatment to offer its core market.

With all of that said, investors should temper their expectations for Vertex Pharmaceuticals. One significant reason to be wary of the stock is that the road to diversifying into new markets will be a long and expensive one, and the company has only taken the first few steps. While its efforts in the CF market have been wildly successful, it may not have the organizational resources necessary to replicate that success in different contexts. Over the last five years, it has boosted its R&D spending sharply, and even heavier investments will be required when and if its early stage projects reach the later clinical trial stages.

Given that its projects outside of CF are in their infancy, investors should anticipate it taking as long as a decade for those efforts to yield results and revenues. In the meantime, Vertex Pharmaceuticals may still be able to grow its sales within its formidable CF catalog, but it's also possible that chasing the last 10% of that patient population will cost more than it returns.

Link:
3 Reasons to Buy Vertex Pharmaceuticals and 1 Reason Not To - The Motley Fool