Category Archives: Genetic medicine

For Immediate Release

Chicago, IL October 15, 2021 Zacks.com announces the list of stocks featured in the Analyst Blog. Every day the Zacks Equity Research analysts discuss the latest news and events impacting stocks and the financial markets. Stocks recently featured in the blog include: CRISPR Therapeutics AG CRSP, Editas Medicine, Inc. EDIT, Sarepta Therapeutics, Inc. SRPT and Beam Therapeutics Inc. BEAM.

Gene therapy is one of the novel mechanisms of treatment that is attracting several large and small pharma companies. The gene therapies treat a disease by altering or turning off problematic genes and adding genes that help to fight or treat a disease. Scientists have been investigating ways to modify genes or replace faulty genes for the last few decades with a few gene therapies already in the market. Gene therapy is set to become one of the most vital spaces with high prospects in the biotech sector.

These therapies provide the flexibility to develop one-time treatment options for genetic or inherited diseases with limited or no approved therapies available. Moreover, gene-editing can directly affect cells the basic building blocks of living things and may help in developing highly effective therapies.

There are already a few FDA-approved gene therapies targeting different difficult indications. In a historic move, the FDA approved the first gene therapy, Novartis Kymriah, for treating acute lymphoblastic leukemia in 2017. This was followed by the FDA approval of two more gene therapies Gileads Yescarta and Roches Luxturna for oncology and eye disorder indications, respectively, in the same year.

The FDA approved two new gene therapies, Bristol-Myers Breyanzi and Abecma for treating different cancer indications, earlier in 2021. The majority of approved gene therapies have shown strong sales growth since their approval.

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Given the potential of gene therapies to treat complex diseases, the companies developing candidates using gene therapy that are a mix of large and small firms, are in focus. A successful medicine developed by any of these companies can generate annual revenues of $1 billion or more. Here we discuss four biotech stocks with promising gene therapy candidates in their pipeline.

CRISPR Therapeutics

The company is developing its lead pipeline candidate, CTX001, in collaboration with Vertex Pharmaceuticals in mid-stage studies as a potential treatment for transfusion-dependent beta thalassemia and sickle cell disease. The gene-editing therapy candidate previously demonstrated a consistent and sustained response to treatment in the given patient population in an ongoing phase I/II study.

CRISPR Therapeutics is actively seeking collaborations and leveraging its CRISPR/Cas9 gene-editing platform to make therapies for hemoglobinopathies, cancer, diabetes and other diseases.

Editas Medicine

The companys lead pipeline candidate is EDIT-101, which employs CRISPR gene editing to treat LCA10 a rare genetic illness that causes blindness. Editas is currently enrolling in the first pediatric cohort of the phase I/II BRILLIANCE study, which is evaluating EDIT-101 for LCA10. Editas is also pursuing the development of CRISPR candidates for eye diseases other than LCA10 including Usher Syndrome type 2A and recurrent ocular Herpes Simplex Virus type 1.

It is also designing novel medicines for non-malignant hematologic diseases, such as SCD and beta-thalassemia.

Sarepta Therapeutics

Sareptas lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy. The company initiated a pivotal clinical study earlier this year to evaluate it as a one-time treatment for Duchenne muscular dystrophy patients. The promising candidate has also led Roche to sign a collaboration deal with Sarepta.

The company plans to seek FDAs approval to start a pivotal study on its other gene therapy candidate, SRP-9003, in 2021 to evaluate it in patients with Limb-girdle muscular dystrophy (LGMD) type 2E. The company has several other pre-clinical and clinical-stage gene therapy candidates targeting additional indications like Rett Syndrome, cardiomyopathy, Emery-Dreifuss muscular dystrophy type 1, and multiple sclerosis.

Beam Therapeutics

The company has two pre-clinical gene editing candidates, BEAM-101 and BEAM-102, in its pipeline that are being developed as potential treatments for SCD. The company plans to file an investigational new drug application to the FDA seeking approval to start a clinical study on BEAM-101 in the second half of 2021.

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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportBeam Therapeutics Inc. (BEAM) : Free Stock Analysis ReportSarepta Therapeutics, Inc. (SRPT) : Free Stock Analysis ReportEditas Medicine, Inc. (EDIT) : Free Stock Analysis ReportCRISPR Therapeutics AG (CRSP) : Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment Research

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The Zacks Analyst Blog Highlights: CRISPR Therapeutics, Editas Medicine, Sarepta Therapeutics and Beam Therapeutics - Yahoo Finance

CAMBRIDGE, Mass., Oct. 12, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced preliminary financial results for its third quarter ended September 30, 2021.

Sarepta expects its net product revenues for the quarter to be approximately $166.9 million, compared to net product revenues of $121.4 million for the same period of 2020.

The Company had approximately $1.6 billion in cash, cash equivalents and investments as of September 30, 2021, compared to $1.9 billion as of December 31, 2020.

These preliminary financial results are the responsibility of management and have been prepared on a consistent basis with prior periods. The Company has not completed its financial closing procedures for the quarter ended September 30, 2021 and its actual results could be materially different from these preliminary financial results. In addition, the Companys independent registered public accounting firm has not reviewed, compiled, or performed any procedures with respect to these preliminary financial results.

About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

Forward-Looking StatementsIn order to provide Sareptas investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as believes, anticipates, plans, expects, will, may, intends, prepares, looks, potential, possible and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our expected financial results.

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These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we have incurred operating losses since our inception and we may not achieve or sustain profitability; the estimates and judgments we make, or the assumptions on which we rely, in preparing our consolidated financial statements could prove inaccurate; Our revenues and operating results could fluctuate significantly, which may adversely affect our stock price; and those risks identified under the heading Risk Factors in our most recent Annual Report on Form 10-K for the year ended December 31, 2020 and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by the Company which you are encouraged to review.

Source: Sarepta Therapeutics, Inc.

Investor Contact: Ian Estepan, 617-274-4052iestepan@sarepta.com

Media Contact: Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Announces Preliminary Financial Results for the Third Quarter Ended September 30, 2021 - Yahoo Finance

Preimplantation genetic testing (PGT) of embryos during in vitro fertilization (IVF) enlightens patients of possible gene mutations prior to implantation. SGF's in-house team of researchers continues to explore PGT as a counseling tool to optimize care for generations to come.

WASHINGTON, Oct. 14, 2021 /PRNewswire-PRWeb/ -- National Breast Cancer Awareness Month reminds us that an estimated 700,000 people in the U.S. will be diagnosed with breast cancer this year alone, of which, up to 10 percent of cases are linked to an inherited gene mutation. Individuals and couples with known genetic diseases can turn to Shady Grove Fertility (SGF) to undergo in vitro fertilization (IVF) with preimplantation genetic testing for monogenic/single gene defects (PGT-M) to reduce the risk of passing genetic mutations to future offspring.

SGF can screen for over 280 recessive gene mutations, including diseases such as cystic fibrosis, Tay-Sachs disease, and spinal muscular atrophy. Additionally, patients at risk for dominant gene mutations, such as breast cancer or inherited forms of colon cancer, can have individualized testing for these diseases.

In 2019, SGF formed a partnership with breast cancer advocate and Previvor Founder, Allyn Rose, to educate patients of these interventions and the availability of fertility preservation prior to any cancer treatment.

"In the last letter that my mother wrote to me before her passing from metastatic breast cancer at age 50, she warned me of my family's predisposition to cancer and rare diseases, encouraging me to undergo in vitro fertilization (IVF) treatment to eliminate this disease," says Rose, a former Miss USA and Miss America contestant, model, and the recipient of the 21st Annual Congress on Women's Health's Advocacy Award and a Breast Cancer Summit Lifetime Achievement Award.

While Rose is not a carrier for the breast cancer gene, she is a carrier of a rare X-linked genetic mutation called Wiskott-Aldrich syndrome. Because of this genetic mutation, there would be a 50 percent chance that her future children would also become carriers of the disease. In October 2019, Rose started her IVF journey with Kate Devine, M.D., at SGF's K Street location in Washington, D.C., opting to undergo PGT-M to reduce the risk of having a child with an inherited condition. After 10 months of IVF treatment with SGF, Rose announced that she was pregnant in July 2020.

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Nearly two years after beginning their journey with SGF, Rose and her husband Christopher announced the birth of their daughter, Yve, on April 9, 2021.

"It was an incredibly fulfilling experience welcoming my daughter into the world because it felt as if my journey had come full circle," shares Rose. "For the last 10 years, I've worked as an advocate in the breast cancer community. I've spent years speaking on the importance of long-term perspective and highlighting my choice to undergo a preventive mastectomy in order to prolong my life and to conceive via IVF removing my rare genetic disease from my family tree. Now, I can hold my daughter in my arms and see that it was all worth it. Everything that I've worked towards has finally come to fruition."

"Allyn's story is a beacon of hope for women with increased cancer risk and other genetic risk factors," says Dr. Devine. "IVF with PGT-M is a safe and reliable means of fertility treatment for people who want to reduce risk of known genetic mutations in their children. I'm so happy that Allyn underwent treatment to reduce her own familial breast cancer risk and that she and that her baby girl is free of Wiskott-Aldrich gene mutations."

While Rose was proactive with preventive family-building measures, more awareness and education is needed on the topic. SGF is slated to present research findings on genetic testing in the infertility population for those at increased risk at the 2021 ASRM Scientific Congress and Expo, taking place in Baltimore from October 1721.

The study, which was honored with the ASRM 2021 Corporate Member Council In-Training Award, concludes that an overwhelming majority of eligible fertility patients are declining cancer genetic testing despite receiving focused genetic counseling regarding the recommendation to pursue testing as well as the opportunity to potentially minimize the cancer risk of future offspring.

"Prior to initiating fertility treatment, all patients who participated in the study received focused genetic counseling informing them that if one parent has a hereditary cancer-predisposing mutation, there is a 50% risk that any child will have it too," explains Dr. Devine, who also serves as SGF's Director of Research. "We will continue to research the underlying 'why' behind these decisions to evolve future care models that offer the safest and healthiest outcomes for our patients."

"Making the decision to pursue IVF with PGT is a very personal one," adds Rose. "But I am a strong proponent of pursuing all options available in order to give children the best possible opportunity to thrive in life. We are so fortunate to live in a world where we can now reduce the inheritance of deadly genetic diseases via IVF. It's such an incredible gift and I am fortunate to have had the opportunity to give my children a better outlook than I had."

SGF is also dedicated to helping patients with cancer receive fertility preservation care. SGF has a specially trained team that works specifically with people with cancer to ensure the fertility preservation process before cancer treatment can be expedited in order that cancer treatment can quickly begin. The oncofertility team at SGF helps to guide patients through each step of the treatment process, from finding ways to afford treatment to the actual medical procedure.

If you would like to learn more about SGF's oncofertility treatment options or to schedule an appointment, please call the New Patient Center at 1-888-761-1967 or complete this brief online form.

About Shady Grove Fertility (SGF) SGF is a leading fertility and IVF center of excellence with more than 100,000 babies born. With 43 locations, including new locations in Colorado and Norfolk, VA, as well as throughout CO, FL, GA, MD, NY, PA, VA, D.C., and Santiago, Chile, SGF offers patients virtual physician consults, delivers individualized care, accepts most insurance plans, and makes treatment more affordable through innovative financial options, including 100% refund guarantees. More physicians refer their patients to SGF than any other center. SGF is among the founding partner practices of US Fertility, the largest physician-owned, physician-led partnership of top-tier fertility practices in the U.S. Call 1-888-761-1967 or visit ShadyGroveFertility.com.

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Jean Dzierzak, Shady Grove Fertility, 301-545-1375, jean.dzierzak@sgfertility.com

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Advancements in reproductive medicine available at Shady Grove Fertility (SGF) can reduce the inheritance of genetic diseases like breast cancer -...

News Release

Monday, October 11, 2021

NIH-funded research reveals clinical trial candidate for those with genetic risk.

A commonly available oral diuretic pill approved by the U.S. Food and Drug Administration may be a potential candidate for an Alzheimers disease treatment for those who are at genetic risk, according to findings published in Nature Aging. The research included analysis showing that those whotook bumetanide a commonly used and potent diuretic had a significantly lower prevalence of Alzheimers disease compared to those not taking the drug. The study, funded by the National Institute on Aging (NIA), part of the National Institutes of Health, advances a precision medicine approach for individuals at greater risk of the disease because of their genetic makeup.

The research team analyzed information in databases of brain tissue samples and FDA-approved drugs, performed mouse and human cell experiments, and explored human population studies to identify bumetanide as a leading drug candidate that may potentially be repurposed to treat Alzheimers.

Though further tests and clinical trials are needed, this research underscores the value of big data-driven tactics combined with more traditional scientific approaches to identify existing FDA-approved drugs as candidates for drug repurposing to treat Alzheimers disease, said NIA Director Richard J. Hodes, M.D.

Knowing that one of the most significant genetic risk factors for late-onset Alzheimers is a form of the apolipoprotein E gene called APOE4, researchers analyzed data derived from 213 brain tissue samples and identified the Alzheimers gene expression signatures, the levels to which genes are turned on or off, specific to APOE4 carriers. Next, they compared the APOE4-specific Alzheimers signatures against those of more than 1,300 known FDA-approved drugs. Five drugs emerged with a gene expression signature that the researchers believed might help neutralize the disease. The strongest candidate was bumetanide, which is used to treat fluid retention often caused by medical problems such as heart, kidney, and liver disease.

The researchers validated the data-driven discoveries by testing bumetanide in both mouse models of Alzheimers and induced pluripotent stem cell-derived human neurons. Researchers found that treating mice which expressed the human APOE4 gene reduced learning and memory deficits. The neutralizing effects were also confirmed in the human cell-based models, which led to the hypothesis that people already taking bumetanide should have lower rates of Alzheimers. To test this, the team pared down electronic health record data sets from more than 5 million people to two groups: adults over 65 who took bumetanide and a matching group who did not take bumetanide. The analysis showed that those who had the genetic risk and took bumetanide had a ~35% to 75% lower prevalence of Alzheimers disease compared to those not taking the drug.

We know that Alzheimers disease will likely require specific types of treatments, perhaps multiple therapies, including some that may target an individuals unique genetic and disease characteristics much like cancer treatments that are available today, said Jean Yuan, M.D., Ph.D., Translational Bioinformatics and Drug Development program director in the NIA Division of Neuroscience. The data in this paper make a good case to conduct a proof-of-concept trial of bumetanide in people with genetic risk.

The research team was led by scientists at Gladstone Institutes, San Francisco, the University of California, San Francisco, and the Icahn School of Medicine at Mount Sinai, New York City. This group is one of more than 20 teams supported by NIA through a program encouraging the researcher community to seek, through big data approaches, drugs that could potentially be repurposed.

The research was funded by NIH grants R01AG057683, R01AG048017, F31AG058439, R01AG061150, F31AG057150, R21TR001743, and K01ES028047.

NIA leads NIHs systematic planning, development, and implementation of research milestones to achieve the goal of effectively treating and preventing Alzheimers and related dementias. This research is related to Milestone 7.B, Initiate research programs for translational bioinformatics and network pharmacology to support rational drug repositioning and combination therapy from discovery through clinical development.

About the National Institute on Aging (NIA): NIA leads the U.S. federal government effort to conduct and support research on aging and the health and well-being of older people. Learn more about age-related cognitive change and neurodegenerative diseases via NIAs Alzheimer's and related Dementias Education and Referral (ADEAR) Center website. Visit the main NIA website for information about a range of aging topics, in English and Spanish, and stay connected.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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Precision medicine data dive shows water pill may be viable to test as Alzheimer's treatment - National Institutes of Health

- Oral and poster presentations to highlight preclinical GeneRide platform data in new indications demonstrating strong evidence of selective advantage of the corrected hepatocytes

Published: Oct. 12, 2021 at 3:05 PM CDT

LEXINGTON, Mass., Oct. 12, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today announced that it was selected to present new data from the company's GeneRide platform in a plenary oral presentation and three poster presentations at the upcoming European Society of Gene and Cell Therapy (ESGCT) Virtual Congress 2021, to be held from October 19-22, 2021.

The oral presentation will include new preclinical GeneRide data showing delivery of corrective genes in three different indications with intrinsic liver damage demonstrating strong evidence of selective advantage of the corrected hepatocytes. Poster presentations will highlight GeneRide expression data in preclinical models of tyrosinemia type 1, as well as the company's optimized adeno-associated virus (AAV) process development.

Oral Presentation Details:

Title: Nuclease-free, promoterless recombinant AAV-mediated genome editing restores function of hepatocytes leading to selective advantage and repopulation in mouse models with liver disease (OR40)Presenter:Shengwen Zhang, Director, Pharmacology, LogicBio TherapeuticsSession: 5b: Gene Editing IIISession date/time:October 21, 2021,17:00-17:15 p.m. CEST (11:00-11:15 a.m. ET)

Poster Presentations Details:

Title: A novel endonuclease-free genome editing technology to edit hepatocytes in vivo led to a full molecular liver transplant and cured mice in preclinical models of Tyrosinemia Type 1 (P253)Q. Qiang Xiong, Director, Head of Preclinical Pharmacology, LogicBio Therapeutics

Title: Development of an Anion Exchange Chromatography Method to Assess Percent Full Capsids for Chimeric Capsid AAV-LK03 (P268)William Lee, Research Associate, LogicBio Therapeutics

Title: Modified plasmid and transfection optimization in suspension HEK293 cells lead to scalable high-yield process for AAV manufacturing (P278)Hans Reuter, Upstream Engineer, Process Development, LogicBio Therapeutics

Additional information on the meeting can be found on the ESGCT website.

The presentation and posters will be available shortly after being presented on the LogicBio Therapeutics website at Presentations | LogicBio Therapeutics, Inc.

AboutLogicBio Therapeutics

LogicBio Therapeuticsis a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. The Company's gene editing platform, GeneRide, is a new approach to precise gene insertion harnessing a cell's natural DNA repair process potentially leading to durable therapeutic protein expression levels. The Company's gene delivery platform, sAAVy, is an adeno-associated virus (AAV) capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues. The Company is based inLexington, MA.For more information, visitwww.logicbio.com, which does not form a part of this release.

Investor Contacts: Laurence WattsGilmartin Group(619) 916-7620laurence@gilmartinir.com

Stephen JasperGilmartin Group(858) 525-2047stephen@gilmartinir.com

Media Contacts:Adam DaleyBerry & Company Public RelationsW:212-253-8881C: 614-580-2048adaley@berrypr.com

Jenna UrbanBerry & Company Public RelationsW: 212-253-8881C: 203-218-9180jurban@berrypr.com

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SOURCE LogicBio Therapeutics, Inc.

The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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LogicBio Therapeutics to Present New GeneRide Data at the European Society of Gene and Cell Therapy Virtual Congress 2021 - WMTV - NBC15

The collaboration will enable production of gene therapies for LEXEOs upcoming clinical trials, future IND-enabling studies and commercialization, according to the parties.

FBD is to provide good manufacturing practice (GMP) production, analytical development, process optimization, and chemistry, manufacturing and controls (CMC) for LEXEOs clinical-stage programs.

Gerry Farrell, COO, FBD, Texas, called the alliance a "synergistic partnership"thatcombines its technical expertise in gene therapy manufacturing with the New York city based company's "cutting-edge" pipeline.

R Nolan Townsend, CEO of LEXEO, outlined how the partnership would help it advance multiple late-stage clinical programs simultaneously.

The partners will initially focus on the supply of LX1004, an adeno-associated virus (AAV)-mediated gene therapy for potential treatment of CLN2 Batten disease, in an upcoming pivotal study.

LEXEO will utilize single-use suspension bioreactor technology, creating a common manufacturing platform and technology base to support vector demand across its pipeline.

The company's portfolio targets rare and non-rare disease indications; it also has an APOE2+ Alzheimers Disease program.

Founded on the basis of a gene therapy research legacy at Weill Cornell Medicines Department of Genetic Medicine with the goal of developing disease-modifying treatments for genetic cardiovascular conditions and genetic conditions of the central nervous system (CNS), itnow has more than 15 AAV-mediated gene therapy programs in research and development.

Parent company, Fujifilm Corporation,has spent US$5.5bm since 2011 to support the CDMO business ofFujifilmDiosynth Biotechnologies.

With an US$850m investment announced in June this year, the CDMO is expanding its manufacturing capabilities to support an increased capacity for biologics and gene therapies. The cash infusion is aimed at growing FBDs contract manufacturing capabilities in the US and the UK. Those expansion projects will be fully realized in 2023.

In January 2021, the company invested US$40m to establish its third viral vector contract development operation in Boston. The third site, it said, would enable it to respond to customers needs for the viral vector manufacturing process from the early clinical stage through commercialization.

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Fujifilm Diosynth in tie-up with US AAV gene therapy developer - BioPharma-Reporter.com

Gene therapy is one of the novel mechanisms of treatment that is attracting several large and small pharma companies. The gene therapies treat a disease by altering or turning off problematic genes and adding genes that help to fight or treat a disease. Scientists have been investigating ways to modify genes or replace faulty genes for the last few decades with a few gene therapies already in the market. Gene therapy is set to become one of the most vital spaces with high prospects in the biotech sector.

These therapies provide the flexibility to develop one-time treatment options for genetic or inherited diseases with limited or no approved therapies available. Moreover, gene-editing can directly affect cells the basic building blocks of living things and may help in developing highly effective therapies.

There are already a few FDA-approved gene therapies targeting different difficult indications. In a historic move, the FDA approved the first gene therapy, Novartis Kymriah, for treating acute lymphoblastic leukemia in 2017. This was followed by the FDA approval of two more gene therapies Gileads Yescarta and Roches Luxturna for oncology and eye disorder indications, respectively, in the same year. The FDA approved two new gene therapies, Bristol-Myers Breyanzi and Abecma for treating different cancer indications, earlier in 2021. The majority of approved gene therapies have shown strong sales growth since their approval.

Given the potential of gene therapies to treat complex diseases, the companies developing candidates using gene therapy that are a mix of large and small firms, are in focus. A successful medicine developed by any of these companies can generate annual revenues of $1 billion or more. Here we discuss four biotech stocks with promising gene therapy candidates in their pipeline.

CRISPR Therapeutics CRSP

The company is developing its lead pipeline candidate, CTX001, in collaboration with Vertex Pharmaceuticals in mid-stage studies as a potential treatment for transfusion-dependent beta thalassemia and sickle cell disease. The gene-editing therapy candidate previously demonstrated a consistent and sustained response to treatment in the given patient population in an ongoing phase I/II study.

Story continues

CRISPR Therapeutics is actively seeking collaborations and leveraging its CRISPR/Cas9 gene-editing platform to make therapies for hemoglobinopathies, cancer, diabetes and other diseases.

Editas Medicine EDIT

The companys lead pipeline candidate is EDIT-101, which employs CRISPR gene editing to treat LCA10 a rare genetic illness that causes blindness. Editas is currently enrolling in the first pediatric cohort of the phase I/II BRILLIANCE study, which is evaluating EDIT-101 for LCA10. Editas is also pursuing the development of CRISPR candidates for eye diseases other than LCA10 including Usher Syndrome type 2A and recurrent ocular Herpes Simplex Virus type 1.

It is also designing novel medicines for non-malignant hematologic diseases, such as SCD and beta-thalassemia.

Sarepta Therapeutics SRPT

Sareptas lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy. The company initiated a pivotal clinical study earlier this year to evaluate it as a one-time treatment for Duchenne muscular dystrophy patients. The promising candidate has also led Roche to sign a collaboration deal with Sarepta. The company plans to seek FDAs approval to start a pivotal study on its other gene therapy candidate, SRP-9003, in 2021 to evaluate it in patients with Limb-girdle muscular dystrophy (LGMD) type 2E. The company has several other pre-clinical and clinical-stage gene therapy candidates targeting additional indications like Rett Syndrome, cardiomyopathy, Emery-Dreifuss muscular dystrophy type 1, and multiple sclerosis.

Beam Therapeutics BEAM

The company has two pre-clinical gene editing candidates, BEAM-101 and BEAM-102, in its pipeline that are being developed as potential treatments for SCD. The company plans to file an investigational new drug application to the FDA seeking approval to start a clinical study on BEAM-101 in the second half of 2021.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportBeam Therapeutics Inc. (BEAM) : Free Stock Analysis ReportSarepta Therapeutics, Inc. (SRPT) : Free Stock Analysis ReportEditas Medicine, Inc. (EDIT) : Free Stock Analysis ReportCRISPR Therapeutics AG (CRSP) : Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment Research

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4 Biotechs to Watch Amid Rising Prominence of Gene Therapies - Yahoo Finance

-- New data in patients with POMC or LEPR deficiency obesity show setmelanotide led to clinically meaningful improvements in health related quality of life measures and hunger -- -- Updated results from Uncovering Rare Obesity testing program suggest up to 64.5 percent of individuals with early-onset, severe obesity may carry variants linked to rare genetic diseases of obesity -- -- Additional posters include encore presentations of data from Phase 3 trial evaluating setmelanotide in BBS and Phase 2 trial in HET obesity, as well as Phase 1b trial evaluating once-weekly setmelanotide --

BOSTON, Oct. 14, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today presented the first-ever data on the health related quality of life (HRQOL) and experience of patients with obesity due to POMC or LEPR deficiency and updated results from the Uncovering Rare Obesity (URO) genetic testing program at the Obesity Medicine Associations Overcoming Obesity 2021 Conference and its Digital Experience (DX) Oct. 14-23.

The Company and its collaborators delivered four poster presentations, including:

New HRQOL data from post-hoc analyses of Phase 3 trials evaluating setmelanotide in patients with POMC or LEPR deficiency obesity that showed setmelanotide treatment led to sustained, clinically meaningful HRQOL improvements in a majority of patients;

New results from a study based on in-depth patient interviews conducted in patients with POMC and LEPR deficiency obesity enrolled in Rhythms pivotal Phase 3 trials, which highlighted that the reduced hunger and improved satiety resulting from setmelanotide treatment substantially and meaningfully changed patients lives; and

Two presentations detailing updated results from Rhythms URO genetic testing of approximately 8,500 people in the United States with early-onset, severe obesity project that:

64.5% of individuals who had genetic sequencing performed may carry variants associated with rare genetic diseases of obesity, including 54.6% with variants in the melanocortin-4 receptor (MC4R) pathway that may qualify them for enrollment in Rhythms EMANATE or DAYBREAK trials or for treatment with IMCIVREE; and

1.96% of individuals who had genetic sequencing performed may carry biallelic variants in one of 22 Bardet-Biedl Syndrome (BBS)-associated genes or the ALMS1 gene, of which up to 0.34% carried variants considered pathogenic or likely pathogenic.

These new data contribute to the growing body of evidence that supports setmelanotides potential to deliver clinically meaningful weight loss and clinically meaningful improvements in patient reported hyperphagia, as well as HRQOL, reinforcing the value of setmelanotides potential for the treatment of rare genetic diseases of obesity of the MC4R pathway, said Linda Shapiro, M.D., Ph.D., Chief Medical Officer of Rhythm. As we prepare to initiate our next wave of clinical trials, we are encouraged by these new results from the URO genetic testing program, which suggest the potential prevalence of genetic variants among people living with early-onset, severe obesity. These results reinforce the importance of genetic testing for clinical decision making in individuals with early-onset, severe obesity and hyperphagia and support our plans to initiate the Phase 3 EMANATE and Phase 2 DAYBREAK trials later this year, which will expand our clinical development of setmelanotide into patients with variants in any of 36 genes that may impair the MC4R pathway.

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New HRQOL Data and Patient-reported Experience of Hunger from Phase 3 Trials in POMC and LEPR Deficiency ObesitiesInvestigators presented two posters with data generated from post-hoc analyses of the Companys Phase 3 trials in POMC and LEPR deficiency obesities.

In a presentation entitled, Quality of Life in POMC or LEPR Deficiency: Setmelanotide Phase 3 Trials, Peter Khnen, M.D., Charit - Universittsmedizin Berlin, Corporate Member of Freie Universitt Berlin und Humboldt-Universitt zu Berlin, Institute for Experimental Pediatric Endocrinology presented data on the HRQOL burden improvements before and after treatment with setmelanotide.1 Highlights include:

Setmelanotide resulted in clinically meaningful improvement in HRQOL in eight of 13 (73%) of patients after 52 weeks of treatment, with improvements in HRQOL that were 2 to 3 times larger than the relevant meaningful threshold; and

Meaningful HRQOL improvements were observed as early as Week 5 on therapy, and these improvements were maintained throughout the study as they mirrored clinically meaningful hunger reductions and weight loss in these patients.

Martin Wabitsch, M.D. Division of Pediatric Endocrinology and Diabetes, Center for Rare Endocrine Diseases, Department of Pediatrics and Adolescent Medicine, University of Ulm, Ulm, Germany, presented qualitative data from a series of patient interviews in a poster entitled, Patient Experience of Hunger in POMC or LEPR Deficiency. Patients reported that hyperphagia and the constant inability to feel satiety negatively affects their HRQOL, while reduced hunger and improved satiety achieved on setmelanotide therapy substantially and meaningfully changed their lives, profoundly improving their ability to function at school or work. Additionally, these patients reported that discontinuing treatment with setmelanotide would be devastating.

The results of these studies underscore the tremendous burden of POMC and LEPR deficiency obesity, better characterizing the experience of people living with these conditions and highlighting the significant impact of severe obesity and insatiable hunger on their daily lives, said Dr. Khnen. Importantly, these data suggest setmelanotide can deliver clinically meaningful HRQOL benefits, reducing the significant burden of disease by providing patients with improvements in their feelings of insatiable hunger, in addition to substantial weight loss. These results further reinforce the value of setmelanotide as the first U.S., EU and UK-approved precision medicine for chronic weight management in POMC and LEPR deficiency obesities.

Updated Data from the URO Testing ProgramIda Moeller, ScD, ScM, MMSc, Director of Biomedical Informatics at Rhythm, presented, Variants in Obesity-related Genes in a Population with Early-onset Obesity. Rhythms URO testing program is designed to expand access to genetic testing for patients with suspected rare genetic diseases of obesity in the U.S. As of July 12, 2021, Rhythm had collected genetic sequences from approximately 8,500 individuals with early-onset, severe obesity, including 788 individuals who had genetic sequencing performed on the Companys updated genetic panel with 79 genes and the full chromosomal region 16p11.2.

Based on an integrated yield weighted by the number of individuals sequenced for each gene, utilizing the data from the two panels (the original panel and the updated expanded panel), Rhythm projects that 64.5% of individuals sequenced may carry actionable variants linked to rare genetic diseases of obesity.2 Rhythm also estimates that 54.6% of individuals sequenced carry variants in the MC4R pathway that may qualify them for enrollment in Rhythms EMANATE or DAYBREAK trials or commercial treatment with IMCIVREE.

Additionally, Dr. Robert Haws, M.D., Marshfield Clinical Research Institute, presented, Frequency of BBS and ALMS1 Variants in a Cohort With Early-onset Obesity. Based on updated URO results, the Company projects that 0.34% of individuals with early-onset, severe obesity may carry pathogenic or likely pathogenic variants in genes known to be associated with BBS or Alstrom syndrome (or 0.24%, excluding patient samples from a leading BBS clinic where higher frequency of BBS-related variants would expected). Including patients with variants of unknown significance (VUS), Rhythm estimates that 1.96% of individuals with early-onset, severe obesity may carry biallelic variants in one of 22 known BBS-associated genes or ALMS1.3

Also at the Overcoming Obesity 2021 Conference, Rhythm and its collaborators presented three additional abstracts as posters, all of which detailed previously reported clinical data:

Efficacy and Safety of the Melanocortin-4 Receptor Agonist Setmelanotide in Obesity Due to Bardet-Biedl Syndrome: a Phase 3 Trial, as presented by Dr. Robert Haws, M.D., Marshfield Clinical Research Institute;

Setmelanotide in POMC, PCSK1, or LEPR Heterozygous Deficiency Obesity (Phase 2), as presented by Sadaf Farooqi, Ph.D., Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, University of Cambridge;

Trial of a Once-Weekly Setmelanotide Formulation in Patients with Obesity, as presented by Annette Valles-Sukkar, Director Clinical Operations, Rhythm Pharmaceuticals.

All Rhythms presentations from Obesity Medicine Associations Overcoming Obesity 2021 Conference and its Digital Experience will be available on the Publication and Presentations section of its website: https://www.rhythmtx.com/publications/ .

About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. Rhythms precision medicine, IMCIVREE (setmelanotide), was approved in November 2020 by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing and in July and September 2021, respectively, by the European Commission (EC) and Great Britains Medicines & Healthcare Products Regulatory Agency (MHRA) for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE is the first-ever FDA-approved and EC- and MHRA-authorized therapy for patients with these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity, and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 37,500 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythms headquarters is in Boston, MA.

IMCIVREE (setmelanotide) IndicationIn the United States, IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

In the EU and Great Britain, IMCIVREE is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.

Limitations of UseIMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;

Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

Important Safety Information

WARNINGS AND PRECAUTIONS

Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

ADVERSE REACTIONS

The most common adverse reactions (incidence 23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

USE IN SPECIFIC POPULATIONSDiscontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

Treatment with IMCIVREE is not recommended for use while breastfeeding.

To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

See Full Prescribing Information, EU SmPC and MHRA SmPC for IMCIVREE.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, our expectations surrounding potential regulatory submissions, approvals and timing thereof, our business strategy and plans, including regarding commercialization of setmelanotide, and our participation in upcoming events and presentations. Statements using word such as expect, anticipate, believe, may, will and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2021 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@rhythmtx.com

Investor Contact:Hannah DeresiewiczStern Investor Relations, Inc.212-362-1200hannah.deresiewicz@sternir.com

Media Contact:Adam DaleyBerry & Company Public Relations212-253-8881adaley@berrypr.com

_______________________________1 Assessments were taken for patients in Rhythms Phase 3 trials who experienced weight loss of at least five kilograms (or at least five percent of baseline body weight for the patients who weighed less than 100 kg). QOL was assessed using the Impact of Weight on Quality of Life-Lite (IWQOL-Lite) scale for patients 18 years or older, and using the Pediatric Quality of Life Inventory (PedsQL) for children and adolescents aged 8 to 12 and 13 to 17 years, respectively.2 The 64.5% figure represents a weighted yield from 8,599 URO samples collected as of July 12, 2021. Prior to May 2021, Rhythms URO panel tested for variants in 40 obesity-related genes, including 11 genes eligible for the DAYBREAK or EMANATE trials; data for those 11 genes is available in all 8,599 samples. Rhythm launched URO 2.1/3.0 in early May 2021, which now sequences 79 obesity-related genes and the 16p11.2 chromosomal region, including 25 additional DAYBREAK/EMANATE genes. Data on all 79 genes (including all 36 DAYBREAK/EMANATE genes) was available for 788 patients, and then used to calculate a weighted yield across the total study population.3 Data on the frequency of BBS and ALMS1 variants were collected from URO samples as of July 5, 2021, which included a smaller sample size of 8,459 sequenced individuals.

Excerpt from:
Rhythm Pharmaceuticals Presents New Data on Experience of People Living with Rare Genetic Diseases of Obesity and Provides Updates on Uncovering Rare...

image:By creating a genome-proteome map scientists have uncovered hundreds of novel connections between different human diseases. view more

Credit: Omicscience https://www.omicscience.org/. This figure has been generated with BioRender.com.

Hundreds of connections between different human diseases have been uncovered through their shared origin in our genome by an international research team led by scientists from the Medical Research Council (MRC) Epidemiology Unit at the University of Cambridge, challenging the categorisation of diseases by organ, symptoms, or clinical speciality.

A new study published in Science today generated data on thousands of proteins circulating in our blood and combined this with genetic data to produce a map showing how genetic differences that affect these proteins link together seemingly diverse as well as related diseases.

Proteins are essential functional units of the human body that are composed of amino acids and coded for by our genes. Malfunctions of proteins cause diseases across most medical specialties and organ systems, and proteins are also the most common target of drugs that exist today.

The findings published today help explain why seemingly unrelated symptoms can occur at the same time in patients and suggest that we should reconsider how diverse diseases can be caused by the same underlying protein or mechanism. Where a protein is a drug target, this information can point to new strategies for treating a variety of conditions, as well as minimising adverse effects.

In the study using blood samples from over 10,000 participants from the Fenland study, the team led by senior author Dr Claudia Langenberg at the MRCEpidemiology Unit and Berlin Institute of Health at Charit Universittsmedizin, Germany, demonstrated that natural variation in 2,500 regions of the human genome is very robustly associated with differences in abundance or function of 5,000 proteins circulating in the blood.

This approach addresses an important bottleneck in the translation of basic science to clinically actionable insights. While large scale studies of the human genome have identified many thousands of variants in our DNA sequence that are associated with disease, underlying mechanisms remain often poorly understood due to uncertainties in mapping those variants to genes. By linking such disease-related DNA variations to the abundance or function of an encoded protein, the team produced strong evidence for which genes are involved, and identified novel mechanisms by which proteins mediate genetic risk into disease onset.

For example, multiple genome-wide association studies (GWAS) have linked a region of the human genome known as KAT8 with Alzheimers disease but failed to identify which gene in this region was involved. By combining data on both proteins and genes the team was able to identify a gene in the KAT8 region named PRSS8, which codes for the protein prostasin, as a novel candidate gene in Alzheimers disease. Similarly, they identified a novel risk gene for endometrial cancer (RSPO3).

The authors used these new insights to systematically test which of these protein-encoding genes affected a large range of diseases. They discovered more than 1,800 examples in which more than one disease was driven by variations in an individual gene and its protein products. What emerged was a network-like structure of human diseases, because many of the genes connected a range of seemingly diverse as well as related conditions in different tissues. This provides strong evidence that the respective protein is the origin, and points to new potential strategies for treatment.

Dr Langenberg explained:

An extreme example we discovered of how one protein can be connected to several diseases is the protein Fibulin-3, which we connected to 37 conditions, including hypermobility, hernias, varicose veins, and a lower risk of carpal tunnel syndrome. A likely explanation is atypical formation of elastic fibres covering our organs and joints, leading to differences in elasticity of soft and connective tissues. This is also in line with features that others have observed in mice where this gene was deleted.

Dr Maik Pietzner, a researcher at the MRC Epidemiology Unit and co-lead author of the study, added:

Using our genome as the basis was key to the success of this study. Because we know that most of the proteins detected in blood have their origin in cells from other tissues, we integrated different biological layers, like gene expression, to enable us to traceproteins back to disease-relevant tissues. For example, we found that higher activity of the enzyme bile salt sulfotransferase was associated with an increased risk of gall stones through a liver specific mechanism. We linked around 900 proteins to their tissue of origin in this way.

In collaboration with colleagues at the Helmholtz Centre in Munich, Germany, the authors have developed a bespoke web application (www.omicscience.org) to enable immediate dissemination of the results, and allow researchers worldwide to dive deeply into information on genes, proteins and diseases they are most interested in.

Dr Eleanor Wheeler, also at the MRC Epidemiology Unit and co-lead author of the study, concluded:

For most genomic regions associated with disease risk, the underlying causal gene and mechanism are not known. Our work demonstrates the distinctive value of proteins to zoom in on the causal gene for a disease and helps us to understand the mechanism through which genetic variation can cause disease. We envisage that the large amount of information we are sharing with the scientific community will help ongoing and emerging efforts to connect genes to diseases more directly via the encoded protein, thus facilitating accelerated identification of drug targets.

Reference

Pietzner M., Wheeler E., et al. Mapping the proteo-genomic convergence of human diseases. Science 2021;14 Oct 2021; DOI:10.1126/science.abj1541

ENDS

About the MRC Epidemiology Unit

The MRC Epidemiology Unit is a department at the University of Cambridge. It is working to improve the health of people in the UK and around the world.

Obesity, type 2 diabetes and related metabolic disorders present a major and growing global public health challenge. These disorders result from a complex interplay between genetic, developmental, behavioural and environmental factors that operate throughout life. The mission of the Unit is to investigate the individual and combined effects of these factors and to develop and evaluate strategies to prevent these diseases and their consequences. http://www.mrc-epid.cam.ac.uk

About the University of Cambridge

The University of Cambridge is one of the worlds top ten leading universities, with a rich history of radical thinking dating back to 1209. Its mission is to contribute to society through the pursuit of education, learning and research at the highest international levels of excellence.

The University comprises 31 autonomous Colleges and 150 departments, faculties and institutions. Its 24,450 student body includes more than 9,000 international students from 147 countries. In 2020, 70.6% of its new undergraduate students were from state schools and 21.6% from economically disadvantaged areas.

Cambridge research spans almost every discipline, from science, technology, engineering and medicine through to the arts, humanities and social sciences, with multi-disciplinary teams working to address major global challenges. Its researchers provide academic leadership, develop strategic partnerships and collaborate with colleagues worldwide.

The University sits at the heart of the Cambridge cluster, in which more than 5,300 knowledge-intensive firms employ more than 67,000 people and generate 18 billion in turnover. Cambridge has the highest number of patent applications per 100,000 residents in the UK.

http://www.cam.ac.uk

About the Medical Research Council

The Medical Research Council is at the forefront of scientific discovery to improve human health. Founded in 1913 to tackle tuberculosis, the MRC now invests taxpayers money in some of the best medical research in the world across every area of health. Thirty-three MRC-funded researchers have won Nobel prizes in a wide range of disciplines, and MRC scientists have been behind such diverse discoveries as vitamins, the structure of DNA and the link between smoking and cancer, as well as achievements such as pioneering the use of randomised controlled trials, the invention of MRI scanning, and the development of a group of antibodies used in the making of some of the most successful drugs ever developed. Today, MRC-funded scientists tackle some of the greatest health problems facing humanity in the 21st century, from the rising tide of chronic diseases associated with ageing to the threats posed by rapidly mutating micro-organisms. The Medical Research Council is part of UK Research and Innovation. https://mrc.ukri.org/

Observational study

People

Mapping the proteo-genomic convergence of human diseases

14-Oct-2021

Robert A. Scott and Adrian Cortes are current employees and/or stockholders of GlaxoSmithKline. ERG receives an honorarium from the journal Circulation Research of the American Heart Association as a member of the Editorial Board. Stephen O'Rahilly has received remuneration for consultancy services provided to Pfizer Inc, Astra Zeneca, ERX Pharmaceuticals, GSK, Third Rock Ventures and LG Life Sciences. All other authors declare that they have no competing interests.

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Filling the gaps: connecting genes to diseases through proteins - EurekAlert

PALO ALTO, Calif., Oct. 12, 2021 /PRNewswire/ --BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical companythat focuses on genetic diseases and cancers, is announcing meaningful progress in its KRAS cancer portfolio, new programs in gene therapy, and advancements in cardiorenal and early-stage Mendelian programs at its second annual R&D Day today. BridgeBio will also discuss how it is broadening the scope of its R&D engine with the launch of its new early-stage research institute, BridgeBioX.

BridgeBio's R&D Day will feature presentations by Neil Kumar, Ph.D., BridgeBio founder and CEO; Richard Scheller, Ph.D., chairman of R&D at BridgeBio; Charles Homcy, M.D., chairman of pharmaceuticals at BridgeBio; Uma Sinha, Ph.D., chief scientific officer at BridgeBio; and scientists and physicians leading BridgeBio's drug discovery and development programs.

BridgeBio has more than 30 programs in its pipeline for patients living with genetic diseases and genetically-driven cancers. Fourteen of those programs are being advanced in the clinic or commercial setting, and earlier this year BridgeBio received its first two U.S. Food and Drug Administration (FDA) drug approvals.

R&D Day pipeline news and updates:

BridgeBio Precision Oncology

BridgeBio Gene Therapy

BridgeBio Cardiorenal

BridgeBioX

BridgeBio Mendelian

BridgeBio's R&D Day will be held today from 8:30 am ET 11:00 am ET and it will be webcast, with a link available in the event calendar on BridgeBio's investor website, https://investor.bridgebio.com/. A replay of the webcast will be available for one year following the event.

To register for BridgeBio's R&D Day, please sign uphere.

Agenda:

About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio's pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company's two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking StatementsThis press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements include statements relating to expectations, plans and prospects regarding the preclinical and clinical development plans, clinical trial designs, clinical and therapeutic potential, and strategy of our product candidates, including, but not limited to: the unknown future impact of the COVID-19 pandemic delay on our ongoing development and/or our operations or operating expenses; the potential for our next-generation G12C dual inhibitors to be the first known compounds designed to directly bind and inhibit KRAS in both its active (GTP bound) and inactive (GDP bound) conformations driven by insights from its molecular dynamics platform; the potential of our precision oncology program and the timing of our selection of a RAS development candidate; the potential of BBP-818 to enable production of the GALT enzyme and to enable the body's natural ability to metabolize galactose in clinical trials; the potential and success of our Gene Therapy platform; the timing and success of BBP-711 for the treatment of primary hyperoxaluria type 1 and hyperoxaluria caused by hepatic overproduction of oxalate in recurrent kidney stone formers; the timing and success of acoramidis; the timing and success of our regulatory strategy for acoramidis; the timing and success of our planned meetings with regulatory health authorities, including the U.S. Food and Drug Administration (FDA), to discuss potential paths to registration prior to initiation of a Phase 3 registrational study of encaleret in patients with ADH1; the ability of encaleret to be the first approved therapy option indicated specifically for the treatment of ADH1; the success of BridgeBioX to test earlier scientific hypotheses in discovery research and target large, complex genetic diseases with high unmet need; the continuing success of our partnership with Stanford University; the timing and success of our Phase 2 trial of BBP-418; the potential for BBP-589 to be the only potential systemic treatment option being developed for patients with RDEB;reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to: initial and ongoing data from our preclinical studies and clinical trials not being indicative of final data; the potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated; the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales; despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; the continuing success of our collaborations; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those risks set forth in the Risk Factors section of our most recent annual report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and our other SEC filings. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio's management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:Grace Rauh[emailprotected](917) 232-5478

BridgeBio Investor Contact:Katherine Yau[emailprotected](516) 554-5989

SOURCE BridgeBio

https://bridgebio.com/

Original post:
BridgeBio Pharma Announces Progress in its KRAS Portfolio, New Gene Therapy Programs, and Updates on Advancements Across its R&D Pipeline Targeting...