Category Archives: Genetic Therapy

The US subsidiary of French pharma Servier has only been in existence for a touch under three years, but it hasnt wasted a second snapping up collaboration agreements and other deals that have propelled the company toward its main goal of becoming a major player in the oncological therapeutics industry.

David Lee, Servier Pharmaceuticals CEO, toldEndpoints Newsthat biopharma is making a clear shift toward targeting cancer therapies of all kinds, and Servier wants to be a part of it. Lee was named head of Serviers US commercial branch when it launched in July 2018, and his goal was ambitious: double the companys oncology portfolio by 2025.

Serviers already on pace to exceed that benchmark tripling the companys portfolio rather than merely doubling it.

Were able to invest a lot of our revenue in R&D, and then we started committing 50% of that spend in R&D into oncology so were really focusing on oncology, Lee said. And the goal really is to really build out oncology the way we did in cardiovascular and cardiology (therapies). So this is our primary focus.

That focus has spurred three products targeting hematologic malignancies, a portfolio of GI tumor products, biologics, and a series of small molecules from a partnership with Novartis in other words, setting the stage for very consequential pipelines down the road, Lee said.

The goal is to really kind of develop a franchise, he said. To further go deeper into liquid tumors and then to build out (our impact in) solid tumors.

Serviers blitz into the field of oncology therapeutics has included its parent company making a whirlwind series of deals over the past year which have helped speed up Lees admittedly-ambitious goals from when he took the helm of the US branch.

In April, Servier bought up Symphogen, a then-private biotech company focused on recombinant antibodies to be used in oncology treatments. which will enable it to strengthen its antibody capabilities in oncology. And on Dec. 21, the company announced two major deals the first, purchasing Agios Pharmaceuticals, a targeted oncology business, for up to $2 billion; and the second, collaborating with Boston-area biotech Celsius Therapeutics to seek targeted therapies for colorectal cancer.

Five years ago, when Lee was the head of Shires global genetic diseases and oncology franchises, he recalls an industry estimate that investments in cancer therapeutics would grow somewhere between 7% and 11% in the next half-decade. As far as hes aware, Lee said the industry has definitely hit the 11% mark.

The two most exciting areas in our pharmaceutical industry right now are oncology and these rare genetic diseases, he said. (Its) kind of the two areas in pharmacology you really see that are reflected in the valuations and the premiums that people are paying for these deals.

This can be attributed to two main factors, Lee speculates.

Number one is much more targeted therapies coming to oncology. So, you have a lot of these new assets that are paired with biomarkers (and) diagnostic tools that can really get us to more targeted patient populations, he said. Number two is a lot of these assets are going to smaller tumors. So smaller patient populations and more niche tumor types.

Servier is not doing a standalone presentation at JP Morgan this year, but Lee said the company planned to have many meetings with industry stakeholders over the next few days as it continues to expand an increasingly diverse drug portfolio.

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Once aiming to double oncology pipeline, Servier's US branch touts rapid pace in building its foothold in the space - Endpoints News

DetailsCategory: More NewsPublished on Thursday, 07 January 2021 17:39Hits: 531

- The goal of this strategic partnership is to develop ground-breaking drugs for the treatment of debilitating genetic dermatological conditions

- Tyris' expertise in novel non-viral gene therapy technology is synergistic with Almirall's leading expertise in medical dermatology and commitment to deliver breakthrough medicines to patients

BARCELONA, Spain I January 7, 2021 I Almirall, S.A. (BME: ALM), a global biopharmaceutical company based in Barcelona,and Tyris Therapeutics, a next-generation gene therapy company founded by Columbus Venture Partners, have announced a strategic partnership today to tackle orphan dermatological conditions using non-viral gene therapies. By combining Tyris's innovative non-viral based gene therapy technology and Almirall's leading expertise in medical dermatology, the companies aim to develop next-generation gene therapies with transformational potential for the treatment for rare genetic dermatology diseases.

The two partners will collaborate closely via their core expertise. Tyris will be responsible for generating clinical lead candidates and progress them to clinical development. Tyris would receive upfront and research milestone payments along with research funding and Almirall will have exclusive options to acquire gene therapy products and progress them through clinical development to commercialization. The economic terms of this collaboration have not been disclosed.

This new partnership demonstrates Almirall's commitment to deliver breakthrough medicines to patients suffering from debilitating rare genetic dermatological diseases. "We are thrilled to be joining forces with Columbus VP, one of the most experienced investors in the field of gene therapy. This strategic partnership facilitates the development of novel treatments options with the potential to make a significant difference to patients' lives," saidThomas Huber, Research Director at Almirall.

Tyris' non-viral DNA-based technology has the potential to provide new avenues for the treatment of genetic diseases beyond the current viral-based gene therapy. The technology aims to deliver genes without size restrictions to specific tissues with the option for re-administration. Unlike for viral-based gene therapy, this technology may be applicable for all patients, as no preexisting immunity or development of immune response against the vector is expected. In addition, Tyris' technology only requires the DNA sequencenecessary for the therapeutic effect without carrying along any additional unwanted elements.

Damia Tormo, General Partner of Columbus VP and CEO of Tyris, stated, "We are delighted to enter into this partnership and leverage Almirall' s expertise in dermatology to accelerate the development of the most effective gene therapy solutions for many patients with dermatological genetic diseases currently without treatment."

SOURCE: Almirall

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Almirall and Tyris Therapeutics enter into strategic partnership to develop next generation gene therapies | More News | News Channels -...

BOSTON, Jan. 11, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (Akouos) (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, and Blueprint Genetics, a Quest Diagnostics company, today announced the Resonate program. The program offers genetic testing to eligible individuals with auditory neuropathy at no cost to participants, their insurance, or their healthcare providers.

Every year, thousands of children are born with disabling hearing loss and the majority of cases of congenital hearing loss are due to an underlying genetic cause. However, few individuals receive a genetic diagnosis and a key barrier is the availability and accessibility of genetic testing. The Resonate program seeks to overcome the barriers to genetic testing for individuals with auditory neuropathy, empower them to make informed choices, and provide potentially valuable insight to inform medical management.

Akouos is committed to deepening the community's understanding of the genetic forms of auditory neuropathy, a type of sensorineural hearing loss. Today, there are no pharmacologic therapies approved for the treatment of hearing loss. The development of new therapeutic options for underserved patient populations has become increasingly reliant on identification of the underlying genetic cause of a genetic disease, disorder, or condition, said Manny Simons, Ph.D., founder, president, and CEO of Akouos. We hope the Resonate program, along with our efforts to better understand the genetic causes and natural history of multiple forms of sensorineural hearing loss, will enable the research and development of novel genetic medicines for individuals with disabling hearing loss.

There are many different genetic changes that cause hearing loss. These changes can be complex and difficult for routine genetic testing to detect. Understanding the genetic cause of hearing loss requires a high-quality genetic testing strategy that includes the mitochondrial genome, difficult-to-sequence genes, and deep intronic variants in addition to copy number variants in order to increase the diagnostic potential for the program participant. We are excited to work with Akouos to offer our comprehensive genetic testing approach to individuals with auditory neuropathy, says Tero-Pekka Alastalo, M.D., Ph.D., executive medical director of Blueprint Genetics.

The Resonate program is available in the United States and plans to expand to additional geographic regions throughout 2021. To be eligible for the program, individuals can be any age, and must have a current or prior clinical diagnosis of auditory neuropathy, or a medical history consistent with auditory neuropathy. Auditory neuropathy is a hearing disorder in which the inner ear successfully detects sound, but has a problem with sending sound from the ear to the brain.In many cases, genetic mutations cause auditory neuropathy. The Resonate program provides access to the Blueprint Genetics Comprehensive Hearing Loss and Deafness Panel that includes more than 230 genes associated with genetic forms of hearing loss. To help understand their results, participants in the United States also have access to genetic counseling provided by the program at no cost to participants, their insurance, or their healthcare providers.

For more information on the Resonate program, individuals and families please visit http://www.akouosresonate.com and healthcare providers please visit http://www.blueprintgenetics.com/resonate-program.

About AkouosAkouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy. http://www.akouos.com

About Blueprint GeneticsBlueprint Genetics, a Quest Diagnostics company, is a leading specialty genetics and bioinformatics company focused on providing genetic testing for inherited diseases. The company is based in Helsinki and Seattle, with a customer base spanning over 70 countries.www.blueprintgenetics.com

Cautionary Note Regarding Akouos Forward-Looking StatementsStatements in this press release about Akouoss future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the expectations for the Resonate program and the roll-out of the Resonate program. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: Akouoss plans to develop and, if approved, subsequently commercialize Akouoss product candidates; the timing of and Akouoss ability to submit applications for, and obtain and maintain regulatory approvals for, Akouoss product candidates; Akouoss expectations regarding Akouoss regulatory strategy; Akouoss expectations regarding Akouoss ability to fund Akouoss operating expenses and capital expenditure requirements with Akouoss cash, cash equivalents and marketable securities; the potential advantages of Akouoss product candidates; the rate and degree of market acceptance and clinical utility of Akouoss product candidates; Akouoss estimates regarding the potential addressable patient population for Akouoss product candidates; Akouoss commercialization, marketing and manufacturing capabilities and strategy; Akouoss expectations regarding Akouoss ability to obtain and maintain intellectual property protection for Akouoss product candidates; Akouoss intellectual property position; Akouoss ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with Akouoss commercial objectives; the impact of government laws and regulations; Akouoss competitive position and expectations regarding developments and projections relating to Akouoss competitors and any competing therapies that are or become available; developments and expectations regarding developments and projections relating to Akouoss competitors and Akouoss industry; the impact of the COVID-19 pandemic on Akouoss business, results of operations, and financial condition; Akouoss ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the Risk Factors included in Akouoss Quarterly Report on Form 10-Q for the three months ended September 30, 2020 filed with the Securities and Exchange Commission, and in other filings that Akouos makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Akouos expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Akouos

Media:Katie Engleman, 1ABkatie@1abmedia.com

Investors:Courtney Turiano, Stern Investor RelationsCourtney.Turiano@sternir.com

Blueprint Genetics

Tero-Pekka Alastalo, executive medical directortpa@blueprintgenetics.com

Saara Salonoja, communication specialistsaara.salonoja@blueprintgenetics.com

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Akouos and Blueprint Genetics Announce the Resonate Program to Improve Access to Genetic Testing for Individuals with Auditory Neuropathy -...

Almost two years after Biogen swooped again into the ophthalmic gene therapy field with an $800 million buyout of eye-focused Nightstar Therapeutics, its turning to a young player for a new program.

Germanys ViGeneron will be deploying its next-generation AAV vectors to generate candidates for a target Biogen isnt disclosing which exactly that would treat inherited eye disease. The big biotech partner has another target in mind, for which it has the option to add within two years.

Although the Alzheimers drug aducanumab has been shoved to a central, almost existential position at Biogen, the company has been placing measured bets on ophthalmology, which CEO Michel Vounatsos has called an emerging area and part of the second wave of growth to follow aducanumab in the mid-2020s.

With the new deal, its buying into a platform that boasts of superior transduction efficiency in retinal cells and intravitreal administration.

Once its come up with the AAV capsids, ViGeneron will play a role in the in vivo proof of concept, but leave it all to Biogen after that point. Biogen is paying an undisclosed upfront and footing the R&D bill.

This collaboration exemplifies our strategy to develop in-house programs for selected retinal targets, while maximizing our proprietary technology platforms with additional collaboration programs for other targets in ophthalmology and further indications, said Caroline Man Xu, co-founder and CEO of ViGeneron.

Theres no word yet on when the candidates might materialize, but they will likely come long after Biogen reads out the Phase III study of BIIB111 (timrepigene emparvovec), the lead asset from the Nightstar buyout. Designed to replace the gene thats mutated in patients with choroideremia, the program represents our next pivotal readout and our next potential commercial product after aducanumab, Vounatsos said back in July.

The data are expected to come in the first half of 2021.

Aside from that and a second Nightstar experimental treatment for retinitis pigmentosa caused by mutations in the RPGR gene, Biogen has also picked up a preclinical gene therapy from Massachusetts Eye and Ear for inherited retinal degeneration due to mutations in the PRPF31 gene.

It all marks a second try at developing one-shot cures for rare eye diseases, after pulling out from a deal with Applied Genetic Technologies following a failed trial.

Continued here:
Edging toward a pivotal readout, Biogen picks a new discovery partner for ophthalmic gene therapy - Endpoints News

Jan. 12, 2021 11:45 UTC

First four investments of new program created to support clinical-stage biotechnology companies

New investments include companies focused on Oncology, Rare Disease, and Inflammation & Immunology

NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) today announced that, during the second half of 2020, it invested a total of $120 million in four clinical-stage biotech companies as part of the Pfizer Breakthrough Growth Initiative (PBGI). Through PBGI, Pfizer is investing up to $500 million in biotechnology companies, providing funding and access to Pfizers scientific expertise to help ensure the continuity of promising clinical development programs of potential future strategic interest to Pfizer.

Pfizers initial PBGI investments include:

Pfizer has a long history of collaborating across the healthcare ecosystem with the shared goal of turning great science into innovative new medicines, said Debbie Baron, Senior Vice President of Business Development, Pfizer. Our investments in Homology, Vedanta, Trillium, and ESSA reflect our commitment to find new and creative ways to leverage Pfizers resources to deliver breakthroughs to patients.

Established in June 2020, the Pfizer Breakthrough Growth Initiative focuses on non-controlling equity investments primarily in public companies with small- to medium-sized market capitalizations and mature private companies that are developing clinical-stage assets aligned with Pfizers core areas of focus: Internal Medicine, Inflammation & Immunology, Oncology, Rare Disease, Vaccines, and Hospital. In addition to equity, Pfizer looks to strengthen collaboration through participation on company and scientific advisory boards, as well as securing certain strategic rights when of mutual fit.

Biotechnology companies interested in learning more may contact pbgi@pfizer.com.

About Pfizer: Breakthroughs That Change Patients Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice: The information contained in this release is as of January 12, 2021. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about the Pfizer Breakthrough Growth Initiative (PBGI) and Pfizers initial PBGI investments in ESSA Pharma, Trillium Therapeutics, Vedanta Biosciences and Homology Medicines, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development; uncertainties regarding the ability of the Pfizer Breakthrough Growth Initiative to identify investment candidates; uncertainties regarding the success of investments by the Pfizer Breakthrough Growth Initiative; uncertainties and variables inherent in business operating and financial performance, including, among other things, competitive developments and general economic, political, business, industry, regulatory and market conditions; future exchange and interest rates; changes in tax and other laws, regulations, rates and policies; uncertainties regarding the impact of COVID-19; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

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Pfizer Invests $120 Million in Biotechnology Innovation Through the Pfizer Breakthrough Growth Initiative - BioSpace