Category Archives: Gene Medicine

A quick read of whose wallets got thicker in the biopharma industry,from largest to smallest.

D3 Bio

Industry-veteran George Chen left a seven-year career at AstraZeneca to launch his ownShanghai-based biotech, D3 Bio, with the support ofbig-nameinvestorslikeBoyu Capital, Matrix Partners China,SequoiaCapital China, Temasek and Wuxi AppTecs Corporate Venture Fund. At this point D3 is keeping its pipeline close to the chest.But Chensays its the approach thatsunique. The company will startwith insights from clinical development and a market assessment of unmet needs and use that to guide the clinical development path. D3 will use this$200 million Series Ato build out an R&D team focused on precision medicine in the realms of immunology and oncology.

Metagenomi

Unlocking the power of microbial evolution,Metagenomimines the worlds natural microbial environment to rapidly develop effective cures to treat incurable genetic diseases. A recent$65 million Series Awill help accelerate the expansion of its gene editing systems for therapies in oncology and genetic-related diseases.This means developing a vast database of gene editing capabilities to enable unprecedented therapeutic approaches. Working with visionary investors, such as Leaps by Bayer and Humboldt Fund, will allow us to deliver on our promise to partners and fuel the development of our own pipeline of innovative curative medicines, Thomas said in a statement.

AliveCor

AI companydisguisedas a medical device companyAliveCorpicked up$65 millionto ramp up speed on their remote cardiology platform. Amidst a pandemic, telehealth appointments have been increasingly necessary.AliveCorsECG will be strengthened withcardiologicaltelehealthservices as well as with detection and condition management services. To date, its products have served more than one million customers globally, recording over 85 million ECGs.AliveCorsKardiaMobiledevice is FDA-cleared and the most clinically validated personal ECG solution in the world.

Nereid Therapeutics

Birthed fromthe work of Clifford P.Brangwynne, Ph.D., Nereid hopes to translate the therapeutic promise of biomolecular condensates from physics to physicians.The biotech will take the$50 million Series Afunding andBrangwynnesproprietary technology enabling precise measurement, interrogationand control of phase separation in cells to develop their drug discovery platform. The platform holds potential to enable completely new approaches to discovering and developing therapeutics across a wide variety of diseases, focusing first on cancers and neurodegenerative disordersaffected by phase transitions.

KiraPharmaceuticals

Backed by$46 million in financingfrom biotech entrepreneur Peter Wirth and others,Kira launchedwith a mission of pioneering a new generation of complement-targeted therapies to treat immune-mediated diseases. With the financing in hand, Kira is aiming to have three assets in the clinic within the next 18 months. The companys most advanced program, P014, is a first-in-class biologic drug with a unique mechanism of action designed to inhibit both upstream and downstream complement targets.Former Sienna Biopharmaceuticals CEO Frederick Beddingfield will be at the helm.

Adagio Medical

Adagio is singing joyfully to the tune of a $42.5 million Series Eto supportthe commercialization of itsiCLASsystem.iCLASis Adagios intelligent Continuous Lesion Ablation Systempursuing both an Investigational Device Exemption trial and a European VT CE-Mark trial.Cardiac ablation is a large and growing market that faces significant challenges including disappointing clinical outcomes, long procedure times and unsatisfactory profitability for providers, saidTuan Huynh, ofArrowMark, one of the Series E investors joining Adagios board of directors. We believe Adagio represents a unique opportunity to transform ablation therapy and look forward to partnering with Adagios management team to support the companys growth and commitment to addressing challenges faced by physicians and their patients.

IniPharm

Founded in 2018 with a focus on liver disease,IniPharmbrought in$35 million with a Series Afinancing roundto take its lead program through to IND filing and into clinical trials.Theprogram targets the HSD17B13 gene, which according to CEO Brian Farmer, confers pretty amazing protection against liver disease.It doesnt appear to actually prevent the diseases causation, but slows progression to more serious illness by preventing inflammation, fibrosis and cirrhosis of the liver, which are the dangerous effects of liver disease.The potential for therapies that effectively target HSD17B13 activity is significant because it is linked to a broad spectrum of liver and related diseases, said Farmer.

InterVennBiosciences

InterVennlooks to ramp up their ability to discover biomarkersand design clinical trials with the help of a little AI. Funds from a$34 million Series Bwill expand its precision medicine platformfor cancer detection.InterVennstech platform targets carbohydrates known as glycans, looking for aberrant glycosylation of certain proteins, which are implicated in a variety of disease states, including inflammation.The companysVOCALprojectis evaluating a blood test to determine ifan ovarian tumor is benign or malignant.It is also conducting research into colorectal cancer and kidney cancer, hunting for clinically actionable biomarkers that can be used for diagnosis, prognosis, and detection of cancer recurrence, as well as predictive tests to help choose appropriate drugs.

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Biopharma Money on the Move PharmaLive - PharmaLive

As colleagues at Seattle Childrens Hospital, Andy Scharenberg, M.D., and Michael Jensen, M.D., knew they wanted to build a startup with a mission.

Although cell therapies like Novartis Kymriah and Gileads Yescarta have transformed treatment for certain blood cancers, Scharenberg, a pediatric immunologist by training, was struck by the numbers of patients who cant get those treatments.

I wanted to create something where the impact would be absolutely the biggest possible swing you could take, he said.

That was the driving force behind the pairs decision to found Seattle-based Umoja Biopharma, which debuted Tuesday with a $53 million series A round. Scharenberg envisions Umoja, which means unity in Swahili, as a unification of three approaches developed by scientists at Seattle Childrens and Purdue University.

Umojas multipronged approach relies on three platforms used in sequence.

We're going to give you a first medicine that's going to grow a cancer-fighting cell army in your body, and then we're going to send that cancer-fighting army in your body messages telling it how to kill off your tumor, Scharenberg said.

That first medicine is VivoVec, which generates cancer-fighting CAR-T-cells inside the body, boosting the immune system. This approach is different to that of current treatments Kymriah and Yescarta, which require T cells to be extracted from patients, engineered outside the bodyand then given back to the patient to fight cancer. VivoVec doesnt need pre-conditioninga treatment used to clear a path for cell therapies to work but that tends to suppress the immune systemand it mimics the bodys own immune response, reducing the risk of cytokine release syndrome, a common side effect of CAR-T treatments.

RELATED: Avrobio tracks improvements in first patient treated with Gaucher gene therapy

After two weeks to allow for the number of T-cells to expand in the body, the patient would then receiveTumorTag, which binds to both tumor cells and the shields they use to hide from the bodys natural immune defenses. As its name suggests, the technology tags them as targets for the newly created army of T-cells.

Finally, the patient would receive RACR/CAR, which doctors can use to tune the activity of the engineered T cells in the body with the help of FDA-approved drugs. One of those drugs is rapamycin, an immunosuppressant.

Originally posted here:
Umoja banks $53M to develop triple-threat immunotherapies - FierceBiotech

ALISO VIEJO, Calif.--(BUSINESS WIRE)--Global Genes, a leading rare disease patient advocacy organization, announces eight award recipients of diverse backgrounds and experiences through its annual Champion of Hope Awards, recognizing exceptional leaders and their efforts to inspire, advance progress, and create positive change for rare disease patients worldwide.

The challenges facing the rare disease community are extraordinary, even in the most ordinary of times, and 2020 has tested the resilience and determination of all patients, caregivers, advocates, and leaders in rare disease. This year, Global Genes honors a set of inspirational leaders whom have made a difference despite the odds, to the benefit of patients and caregivers around the world.

Were proud and grateful to celebrate these remarkable champions for the rare disease community, who offer hope to so many, said Craig Martin, interim CEO of Global Genes. We all need a little extra to keep us going these days, and the stories of these leaders will inspire you, whether you are part of the rare disease community or not.

This year, the celebration recognized those who made a significant impact in advocacy, industry, medical care, science, as well as up-and-coming rare disease leaders. With more than a hundred nominees, these awardees were selected:

In addition, this year Global Genes is introducing a RARE Founders Award, honoring an individual for the founding of an impactful organization in the rare disease community. The inaugural recipient of this award is Nicole Boice, founder of Global Genes and cofounder of RARE-X, a nonprofit organization focused on enabling global sharing of rare disease patient data to accelerate diagnosis and progress toward rare disease treatments and cures.

Success for founders comes from the dedicated work of many, especially in rare disease, where collaboration and a team approach are critical, said Boice. I am honored to receive this first Founders Award, and am excited that it will become an award to recognize future recipients that have created value in rare disease by being a trailblazer, by recognizing holes, gaps, and needs and then doing something about it.

It is an honor to be recognized by Global Genes as a RARE Champion honoree, said Timothy Walbert, CEO of Horizon Therapeutics, an advocacy organization that supports rare and rheumatic conditions. I applaud all that the organization has done and continues to do for the rare disease community, and I look forward to our ongoing partnership to address the challenges in the community as well as celebrate its successes.

Alexis Levine said, As someone who has personally been affected by rare disease, I am so intrinsically motivated to further the research that I sometimes forget about the impact of my work. Thank you, Global Genes, for reminding me that I am a RARE Champion of Hope; it is such an honor to have received this award!

Global Genes is grateful for the support of event sponsors: Alliance for Regenerative Medicine, Ultragenyx, Taysha Gene Therapies, Ionis Pharmaceuticals, and Horizon Therapeutics.

For more information, visit http://www.globalgenes.org.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization on a mission to connect, empower, and inspire the rare disease community. We provide hope for more than 400 million people affected by rare disease around the globe. To date, weve educated millions of people in more than 100 countries about rare disease, equipped patients and advocates with tools and resources, and provided hundreds of thousands of dollars in support for innovative patient impact programs. If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit the resource hub at Globalgenes.org.

The rest is here:
Global Genes Honors Leaders, Advocates, and Innovators in Rare Disease From Around the World at the 2020 RARE Champion of Hope Celebration - Business...

A quick read of whose wallets got thicker in the biopharma industry,from largest to smallest.

D3 Bio

Industry-veteran George Chen left a seven-year career at AstraZeneca to launch his ownShanghai-based biotech, D3 Bio, with the support ofbig-nameinvestorslikeBoyu Capital, Matrix Partners China,SequoiaCapital China, Temasek and Wuxi AppTecs Corporate Venture Fund. At this point D3 is keeping its pipeline close to the chest.But Chensays its the approach thatsunique. The company will startwith insights from clinical development and a market assessment of unmet needs and use that to guide the clinical development path. D3 will use this$200 million Series Ato build out an R&D team focused on precision medicine in the realms of immunology and oncology.

Metagenomi

Unlocking the power of microbial evolution,Metagenomimines the worlds natural microbial environment to rapidly develop effective cures to treat incurable genetic diseases. A recent$65 million Series Awill help accelerate the expansion of its gene editing systems for therapies in oncology and genetic-related diseases.This means developing a vast database of gene editing capabilities to enable unprecedented therapeutic approaches. Working with visionary investors, such as Leaps by Bayer and Humboldt Fund, will allow us to deliver on our promise to partners and fuel the development of our own pipeline of innovative curative medicines, Thomas said in a statement.

AliveCor

AI companydisguisedas a medical device companyAliveCorpicked up$65 millionto ramp up speed on their remote cardiology platform. Amidst a pandemic, telehealth appointments have been increasingly necessary.AliveCorsECG will be strengthened withcardiologicaltelehealthservices as well as with detection and condition management services. To date, its products have served more than one million customers globally, recording over 85 million ECGs.AliveCorsKardiaMobiledevice is FDA-cleared and the most clinically validated personal ECG solution in the world.

Nereid Therapeutics

Birthed fromthe work of Clifford P.Brangwynne, Ph.D., Nereid hopes to translate the therapeutic promise of biomolecular condensates from physics to physicians.The biotech will take the$50 million Series Afunding andBrangwynnesproprietary technology enabling precise measurement, interrogationand control of phase separation in cells to develop their drug discovery platform. The platform holds potential to enable completely new approaches to discovering and developing therapeutics across a wide variety of diseases, focusing first on cancers and neurodegenerative disordersaffected by phase transitions.

KiraPharmaceuticals

Backed by$46 million in financingfrom biotech entrepreneur Peter Wirth and others,Kira launchedwith a mission of pioneering a new generation of complement-targeted therapies to treat immune-mediated diseases. With the financing in hand, Kira is aiming to have three assets in the clinic within the next 18 months. The companys most advanced program, P014, is a first-in-class biologic drug with a unique mechanism of action designed to inhibit both upstream and downstream complement targets.Former Sienna Biopharmaceuticals CEO Frederick Beddingfield will be at the helm.

Adagio Medical

Adagio is singing joyfully to the tune of a $42.5 million Series Eto supportthe commercialization of itsiCLASsystem.iCLASis Adagios intelligent Continuous Lesion Ablation Systempursuing both an Investigational Device Exemption trial and a European VT CE-Mark trial."Cardiac ablation is a large and growing market that faces significant challenges including disappointing clinical outcomes, long procedure times and unsatisfactory profitability for providers," saidTuan Huynh, ofArrowMark, one of the Series E investors joining Adagios board of directors. "We believe Adagio represents a unique opportunity to transform ablation therapy and look forward to partnering with Adagio's management team to support the company's growth and commitment to addressing challenges faced by physicians and their patients."

IniPharm

Founded in 2018 with a focus on liver disease,IniPharmbrought in$35 million with a Series Afinancing roundto take its lead program through to IND filing and into clinical trials.Theprogram targets the HSD17B13 gene, which according to CEO Brian Farmer, confers pretty amazing protection against liver disease.It doesnt appear to actually prevent the diseases causation, but slows progression to more serious illness by preventing inflammation, fibrosis and cirrhosis of the liver, which are the dangerous effects of liver disease.The potential for therapies that effectively target HSD17B13 activity is significant because it is linked to a broad spectrum of liver and related diseases, said Farmer.

InterVennBiosciences

InterVennlooks to ramp up their ability to discover biomarkersand design clinical trials with the help of a little AI. Funds from a$34 million Series Bwill expand its precision medicine platformfor cancer detection.InterVennstech platform targets carbohydrates known as glycans, looking for aberrant glycosylation of certain proteins, which are implicated in a variety of disease states, including inflammation.The company'sVOCALprojectis evaluating a blood test to determine ifan ovarian tumor is benign or malignant.It is also conducting research into colorectal cancer and kidney cancer, hunting for clinically actionable biomarkers that can be used for diagnosis, prognosis, and detection of cancer recurrence, as well as predictive tests to help choose appropriate drugs.

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Biopharma Money on the Move: November 11-17 - BioSpace

NEW YORK, Nov. 18, 2020 /PRNewswire/ --Elevation Oncology, a clinical-stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers, announced today a Series B financing of $65 million led by new investors, venBio Partners and Cormorant Asset Management, and the promotion of Shawn M. Leland, PharmD, RPh, the Company's founder, to Chief Executive Officer. Additional participants in the financing include Boxer Capital of Tavistock Group, Janus Henderson, Samsara Biocapital, and Vivo Capital, as well as all of Elevation Oncology's existing investors:Aisling Capital, Vertex Ventures HC, Qiming Venture Partners USA, Driehaus Capital Management, and BVF Partners.

Andrew Phillips, PhD from Cormorant Asset Management, and Richard Gaster, MD, PhD from venBio Partners, will join the Elevation Oncology Board of Directors in conjunction with the new financing.

"We welcome Andy and Rich to our Board of Directors and are encouraged by the support of a highly sophisticated investor group committed to helping us continue to pursue our mission," said Dr. Leland. "At the core of Elevation Oncology is the belief that patients deserve the right clinical team and the right genomic tests to match the right therapeutics to the unique genomic profile of each tumor. We look forward to continuing to work closely with our Board and Scientific Advisors to innovate and accelerate the development of precision oncology therapeutics to realize this vision."

Dr. Lelandfounded Elevation Oncology in July 2019, is a member of its Board of Directors, and previously served as the Company's Chief Business Officer. He has over a decade of experience in medical affairs and business development for the pharmaceutical/biotech industry, with a focus on building collaborations to realize the full potential of targeted and personalized therapeutics. He has been involved in global transactions totaling more than $450 million in upfront payments and milestone payments at Eli Lilly, ARIAD Pharmaceuticals, Argos Therapeutics and Verastem Oncology. Steve Elms, Managing Partner of Aisling Capital, who was serving as Interim CEO of Elevation Oncology, will remain Chair of the Company's Board of Directors.

"Shawn has been instrumental in the founding and success of Elevation Oncology to date," said Mr. Elms. "On behalf of the entire Board of Directors, I express our great confidence in the future of Elevation Oncology under Shawn's leadership. The proceeds raised with the Series B positions the Company well to deliver on our mission of developing precision therapeutics for patients with genomically defined cancers."

Elevation Oncology's lead development program, the Phase 2 CRESTONE study, is evaluating the HER3 monoclonal antibody seribantumab for the treatment of patients with tumors harboring an NRG1 gene fusion. The Company is actively evaluating opportunities for pipeline expansion, prioritizing targeted therapy approaches in tumor types defined by genomic driver alterations.

"The progress that Elevation Oncology has made in the short time since its founding to establish a strong scientific rationale and an accelerated development path for seribantumab in patients with tumors harboring an NRG1 gene fusion is quite impressive," said Dr. Gaster, Partner at venBio. "We see the progress to date as indicative of Elevation Oncology's long-term potential. I look forward to serving on Elevation's Board of Directors as the Company continues to advance its mission to match unique genomic test results with a purpose-built precision medicine approach to enable an individualized treatment plan for each patient."

"Elevation Oncology's commitment to innovation across the drug development lifecycle has drawn a distinguished group of collaborators who are able to broadly conduct genomic testing across the US, rapidly open up clinical trial sites, and ensure exemplary execution of the CRESTONE study," said Dr. Phillips, Managing Director at Cormorant. "I am very pleased to be joining the Company's Board of Directors to support the continued and expanded application of these efforts towards the efficient acquisition, development, and approval of new therapeutics for patients with genomically defined cancers."

Proceeds from the Series B financing will be used to fund the completion of enrollment in the CRESTONE study and other corporate development activities.

About Elevation OncologyElevation Oncology is founded on the belief that every patient with cancer deserves to know what is driving the growth of their disease and have access to therapeutics that can stop it. We make genomic tests actionable by selectively developing drugs to inhibit the specific alterations that have been identified as drivers of disease. Together with our peers we work towards a future in which each unique test result can be matched with a purpose-built precision medicine to enable an individualized treatment plan for each patient. Our lead candidate, seribantumab, inhibits tumor growth driven by NRG1 fusions and is currently being clinically tested in the Phase 2 CRESTONE study for patients with tumors of any origin that have an NRG1 fusion. Details on CRESTONE are available at http://www.NRG1fusion.com. For more information visit http://www.ElevationOncology.com.

About Seribantumab and NRG1 Gene FusionsSeribantumab is a fully human IgG2 monoclonal antibody that binds to human epidermal growth factor receptor 3 (HER3). HER3 is traditionally activated through binding of its primary ligand, neuregulin-1 (NRG1). The NRG1 gene fusion is a rare genomic alteration that combines NRG1 with another partner protein to create chimeric NRG1 "fusion proteins". The NRG1 fusion protein is often also able to activate the HER3 pathway, leading to unregulated cell growth and proliferation. Importantly, NRG1 gene fusions are mutually exclusive with other known driver mutations and are considered a unique oncogenic driver event essential for tumor cell survival.

NRG1 fusions have been identified in a variety of solid tumors, including lung, pancreatic, gallbladder, breast, ovarian, colorectal, neuroendocrine, and sarcomas. In preclinical experiments, seribantumab prevents the activation of HER3 signaling in cells that harbor an NRG1 gene fusion. In addition to extensive nonclinical characterization and testing, seribantumab has been administered to 847 patients across 12 Phase 1 and 2 studies, both as a monotherapy and in combination with various anticancer therapies. Seribantumab is currently being clinically tested in the Phase 2 CRESTONE study for patients with solid tumors of any origin that have an NRG1 fusion.

About the CRESTONE StudyClinical Study of Response to Seribantumab in Tumors with Neuregulin-1 (NRG1) Fusions. CRESTONE is a Phase 2 tumor-agnostic "basket trial" of seribantumab in patients with any solid tumor that harbor an NRG1 fusion. The primary objective of the study is to describe the anti-tumor activity and safety of seribantumab specifically in patients with an NRG1 gene fusion. CRESTONE offers a clinical trial opportunity for patients with advanced solid tumors who have not responded or are no longer responding to treatment. Patients are encouraged to talk to their doctor about genomic testing of their tumor. CRESTONE is open and enrolling today in the US. For more information visit http://www.NRG1fusion.com.

Media ContactsElevation OncologyName: David Rosen, Argot PartnersPhone: +1 (716) 371-1125Email: [emailprotected]

SOURCE Elevation Oncology

elevationoncology.com

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Elevation Oncology Announces $65M Series B Financing and Promotion of Founder Shawn M. Leland to Chief Executive Officer - PRNewswire

Helixmith said Thursday the first enrolled patient has received its new gene therapy, Engensis (VM202), in phase 3-2 study of diabetic peripheral neuropathy (DPN).

The company plans to continue administering the drug to other participants, as it is screening many additional patients at five clinical trial centers. It is conducting DPN phase 3-2 trials, REGAIN-1A, at 15 clinical centers with 152 enrolled patients in the U.S.

Helixmith aims to meet two endpoints in the study.

The trial's primary efficacy endpoint is to compare changes in average daily pain scores between VM202 and placebo. The secondary endpoint is to reduce pain by 50 percent or more.

DPN is one of the most common complications of diabetes as 30 million U.S. adults have the disorder. Around 28.5 percent of diabetic patients develop DPN, and up to half of them may advance to painful DPN (PDPN).

Dr. Miguel Trevino, head of clinical trials at Innovative Research in which the first patient received VM202, noted that the existing treatments for DPN only relieve pain and show various adverse events with limited efficacy, inevitably leaving half of the patients in painful life.

"The delay of this phase 3 study was due to the preparation of clinical operation system and examining numerous items during the patient's screening process," Helixmith CEO Kim Sun-young said. "Our latest study will add considerable knowledge and evidence to support the optimal use of VM202 in patients needing therapeutic options."

The U.S. Food and Drug Administration recognized the scientific and clinical results of VM202 and designated it as RMAT (regenerative medicine advanced therapy) in 2018.

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Helixmith injects DPN therapy into 1st patient of P3 study - Korea Biomedical Review