Category Archives: Genetic Therapy

January was a busy month for the US Food and Drug Administrations precision medicine efforts, as the agency produced guidance on ASO drugs for patients with debilitating or life-threatening genetic disorders and guidance on manufacturing considerations for certain cellular and gene therapy products during the COVID-19 pandemic.

The agency first issued a draft guidance to facilitate the development of individualized antisense oligonucleotide (ASO) drugs for patients with severely debilitating or life-threatening genetic disorders (ASO Guidance). The Food and Drug Administration (FDA) also issued a guidance, with immediate effect, on manufacturing considerations for licensed and investigational cellular and gene therapy products during the COVID-19 public health emergency (Manufacturing Guidance). Sponsors investigating or marketing these products should pay special attention to the discussion in these documents, as FDA outlines its approach to COVID-19 and development considerations with respect to these personalized therapies.

The Manufacturing Guidance supplements FDAs June 2020 guidance on Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing. However, because cell and gene therapy (CGT) manufacturers may face special challenges, FDA recommends that CGT manufacturers perform risk assessments to identify, evaluate, and mitigate factors that may allow for the transmission of SARS-CoV-2 through CGT products. Any plans should take into account FDAs view that allogeneic products may be associated with a higher risk of infection compared to autologous products.

FDA specifically recommends the following:

As always, any adopted risk assessment and mitigation strategies must be documented and approved by the manufacturers quality unit, should include scientific justification and literature references, and should be submitted to FDA.

Turning away from the current COVID-19 crisis, FDA indicated that it is also looking ahead to the continued advancement of personalized therapies, issuing the ASO Guidance to assist sponsor investigators in the development of individualized ASO products for severely debilitating or life-threatening genetic diseases that are tailored to a patients specific genetic variant. As noted by FDA, the ASO Guidance is targeted to academic investigators, who may be less familiar with FDAs requirements and less experienced in interacting with FDA.

While the specific impetus for this guidance is unclear, assumedly FDA is receiving more inquiries regarding individualized ASO drugs from investigators, patients, or those acting on their behalf. Regardless of the reason, healthcare institutions where ASO products are used should familiarize themselves with FDAs requirements and processes to ensure that any use of an investigational ASO product accords with FDAs regulations. It will also be important that manufacturers supporting the use of ASO products or that later intend to work with ASO product investigators ensure that programs comply with FDAs regulations via contractual agreements and, as appropriate, due diligence.

For these programs, FDA recommends the following:

The ASO Guidance is likely a first step in the development of individualized therapies. As stated by FDA, the agency is optimistic that development of [ASO] individualized drug products may spur gene sequencing that leads to the development of additional individualized drug products. Accordingly, through the ASO Guidance, FDA aims to determine the most effective and efficient way to bring personalized drugs to patients, while ensuring the right risk-benefit balance.

[View source.]

Read this article:
FDA Issues More Guidance on Gene and Cell Therapy Products - JD Supra

Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, and Decibel Therapeutics, a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments for hearing and balance disorders, today announced they have signed an agreement for Catalent to provide Decibel with process and analytical development, and CGMP clinical manufacturing services, for Decibel's lead investigational gene therapy product candidate, DB-OTO.

richard@nepr.agency

SOMERSET, N.J. and BOSTON, Feb. 4, 2021 /PRNewswire-PRWeb/ -- Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, and Decibel Therapeutics, a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments for hearing and balance disorders, today announced they have signed an agreement for Catalent to provide Decibel with process and analytical development, and CGMP clinical manufacturing services, for Decibel's lead investigational gene therapy product candidate, DB-OTO.

DB-OTO is a dual-vector adeno-associated virus (AAV) gene therapy that is designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene. Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals. Under the terms of the agreement, Catalent will provide material from its Maryland-based gene therapy facilities to support Decibel's planned IND-enabling studies and Phase 1/2 clinical trial of DB-OTO.

"There are currently no approved therapies that treat congenital deafness caused by genetic deficiencies. In our preclinical studies, we have delivered full-length, functional otoferlin protein under the control of a cell-selective promoter which restored hearing function in a mouse model of otoferlin deficiency, demonstrated cell selective expression in non-human primates, and preliminary safety and tolerability of delivery of DB-OTO," said John Lee, Executive Vice President, Pharmaceutical Development and Interim Chief Scientific Officer of Decibel. "We look forward to working with Catalent on the development of DB-OTO as we progress towards clinical trials."

Story continues

"Partnering early with innovative companies allows us to develop and optimize robust, scalable manufacturing processes and the analytical methods to assess them," commented Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy. "Our Maryland development centers in Gaithersburg and the University of Maryland BioPark in Baltimore focus on providing process optimization services to meet our customers' needs for early-stage clinical gene therapies."

Catalent has five gene therapy facilities in Maryland that provide services from clinical- to commercial-scale, and house multiple CGMP manufacturing suites, including fill/finish, testing laboratories, warehousing, supply chain capabilities, and other associated services.

About Decibel Therapeutics Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel's pipeline, including its lead gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow @DecibelTx.

About Catalent Cell & Gene Therapy With deep experience in viral vector scale-up and production, Catalent Cell & Gene Therapy is a full-service partner for adeno-associated virus (AAV) and lentiviral vectors, and CAR-T immunotherapies. When it acquired MaSTherCell, Catalent added expertise in autologous and allogeneic cell therapy development and manufacturing to position it as a premier technology, development and manufacturing partner for innovators across the entire field of advanced biotherapeutics. Catalent has a global cell and gene therapy network of dedicated, large-scale clinical and commercial manufacturing facilities, and fill-finish and packaging capabilities located in both the U.S. and Europe. An experienced partner, Catalent Cell & Gene Therapy has worked with industry leaders across 70+ clinical and commercial programs. For more information, visit biologics.catalent.com/cell-gene-therapy/

About Catalent Catalent is the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products. With over 85 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable global clinical and commercial product supply. Catalent employs approximately 15,000 people including around 2,400 scientists and technicians, at more than 45 facilities, and in fiscal year 2020 generated over $3 billion in annual revenue. Catalent is headquartered in Somerset, New Jersey. For more information, visit http://www.catalent.com

More products. Better treatments. Reliably supplied.

Media Contact

Chris Halling, Catalent, +447580041073, chris.halling@catalent.com

Richard Kerns, Northern Exposure Public Relations, +441617285880, richard@nepr.agency

SOURCE Catalent

Go here to read the rest:
Decibel and Catalent Sign Development and Manufacturing Agreement for Dual-Vector Gene Therapy for the Treatment of Congenital Hearing Loss - Yahoo...

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, announced today that it has received $11.6 million from the closing of the sale of Arvelle Therapeutics to Angelini Pharma. Per the terms of the sale, additional payments to Sio Gene Therapies Inc. are expected over time, including a payment of approximately $4.8 million by mid-2021 upon marketing approval of cenobamate by the European Medicines Agency (EMA).

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as expect and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding amounts to be received and income tax liabilities associated with the sale of its stake in Arvelle Therapeutics, as well as the duration of its ability to support its development programs, are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

David NassifSio Gene Therapies Inc.Chief Financial Officer and General Counsel(646) 677-6770investors@siogtx.com

View post:
Sio Gene Therapies Announces Receipt of $11.6 Million from Closing of the Sale of Arvelle Therapeutics - BioSpace

Article reflects Companys leadership and innovation in scalable, reproducible manufacture of adeno-associated virus (AAV)-based gene therapies

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, announced that Sue Washer, President & Chief Executive Officer and Dave Knop, Vice President of Process Development, have been awarded first place in the BioProcess International (BPI) magazine inaugural Readers Choice Awards program, cell and gene therapies category, for their article, Viral-Vectored Gene Therapies: Harnessing Their Potential Through Scalable, Reproducible Manufacturing Processes.

High-productivity approaches to AAV manufacturing processes, like AGTCs HSV-helper based platform, will be crucial if we are to address the unmet clinical need growing across a variety of indications, said AGTC President and CEO, Sue Washer. There is no question that investing in the manufacturing process is imperative and our early commitment in this area has put AGTC in a strong position with respect to the purity and quality needed for late stage development and commercialization.

Concentrating on articles published from September 2019 through June 2020, and using rankings based on views, engagement, and download rates, BioProcess International identified the four most popular articles within each of its six pillars of bioprocessing coverage. The AGTC authors article received the highest number of votes from BPI readers, who ranked the nominees in terms of their innovativeness, presentability and applicability.

The eBook featuring the first-place article by Washer and Knop, as well as summarized versions of the second- and third-place articles, are available by visiting: https://bioprocessintl.com/wp-content/uploads/2020/11/18-11-eBook-RCA-CellGeneTherapies.pdf.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Companys most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Companys industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS:David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

Corporate Contact:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

View post:
AGTC Executives Awarded First Place in the BioProcess International Reader's Choice Awards, Cell & Gene Therapies Category - GlobeNewswire

TipRanks

Weve got a full month of 2021 behind us now, and a few trends are coming clearer. The coronavirus crisis may still be with us, but as vaccination programs expand, the end is in sight. With President Trump out of the picture, and the Democrats holding both Houses of Congress and the White House, politics is looking more predictable. And both of those developments bode well for an economic recovery this year. Looking back, at the year that was, we can also see some trends that stayed firm despite the pandemic, the shutdowns, and the supercharged election season. One of the most important is the ongoing rollout of 5G networking technology. These new networks bring with them a fuller realization of the promises inherent in the digital world. Faster connections, lower latency, higher online capacity, clearer signals all will strongly enhance the capabilities of the networked world. And it wont just be mundane things like telecommuting or remote offices that will benefit 5G will allow Internet of Things and autonomous vehicles to further develop their potential. There is even talk of medical applications, of remotely located doctors performing surgery via digitally controlled microsurgical tools. And these are just the possibilities that we can see from now. Who know what the future will really bring? To this end, we pulled up TipRanks database to learn more about three exciting plays in the 5G space. According to the Street, we are likely to see further interesting developments in the next few years as this technology takes over. Skyworks Solutions (SWKS) The first 5G name were looking at, Skyworks, is a semiconductor chip manufacturer that brought in $3.4 billion in total revenues for FY2020. Skyworks, which is a prime supplier of chips for Apples iPhone series, saw a massive 68% year-over-year increase in 1QFY21 revenues the top line reached $1.51 billion, a company record, and also much higher than analysts had forecast. Much of Skyworks fiscal Q1 sales success came after Apple launched the 5G-capable iPhone 12 line. Strong sales in the popular handset device meant that profits trickled down the supply line and Skyworks channels a disproportionate share of its business to Apple. In fact, Apple orders accounted for 70% of Skyworks revenue in the recent quarter. iPhone wasnt the only 5G handset on the receiving end of Skyworks chips, however the company is also an important supplier to Koreas Samsung and Chinas Xiaomi, and has seen demand rise as these companies also launch 5G-capable smartphones. Finally, Skyworks supplies semiconductor chip components to the wireless infrastructure sector, specifically to the small cell transmission units which are important in the propagation network of wireless signals. As the wireless providers switch to 5G transmission, Skyworks has seen orders for its products increase. In his note on Skyworks for Benchmark, 5-star analyst Ruben Roy writes: SWKS significantly beat consensus estimates and provided March quarter guidance that is also well ahead of consensus estimates as 5G related mobile revenue and broad-based segment revenue continued to accelerate In addition to continued strength of design win momentum and customer activity, we are encouraged with SWKS confident tone relative to the overall demand environment and content increase opportunities. In line with his comments, Roy rates SWKS a Buy along with a $215 price target. At current levels, this implies an upside of 20% for the coming year. (To watch Roys track record, click here) Roy is broadly in line with the rest of Wall Street, which has assigned SWKS 13 Buy ratings and 7 Holds over the past three month -- and sees the stock growing about 15% over the next 12 months, to a target price of $205.69.(See SWKS stock analysis on TipRanks) Qorvo, Inc. (QRVO) Qorvos chief products are chipsets used in the construction of radio frequency transmission systems that power wifi and broadband communication networks. The connection of this niche to 5G is clear as network providers upgrade their RF hardware to 5G, they also upgrade the semiconductor chips that control the systems. This chip maker has a solid niche, but it is not resting on its laurels. Qorvo is actively developing a range of new products specifically for 5G systems and deployment. This 5G radio frequency product portfolio includes phase shifters, switches, and integrated modules, and contains both infrastructure and mobile products. Qorvo posted $3.24 billion in total revenues for fiscal 2020. That revenue represents a 4.8% year-over-year increase and the companys sales have been accelerating in fiscal 2021. The most recent quarterly report, for the second fiscal quarter, showed $1.06 billion in revenues, a 31% yoy increase. Rajvindra Gill, 5-star analyst with Needham, is bullish on Qorvos prospects, noting: Qorvo reported strong sales and gross margins as 5G momentum rolls into CY21 on atypical seasonality... The company is planning for 500M 5G handsets to be manufactured in 2021, with an incremental $5-7 of content/unit from 4G to 5G. Management believes that ultra-wideband adoption will be a key growth driver in for smartphones going forward..." To this end, Gill puts a $220 price target on QRVO shares, suggesting room for 31% upside in 2021. Accordingly, he rates the stock a Buy. (To watch Gills track record, click here) What do other analysts have to say? 13 Buys and and 6 Holds add up to a Moderate Buy analyst consensus. Given the $192.28 average price target, shares could climb ~15% from current levels. (See QRVO stock analysis on TipRanks) Telefonakiebolaget LM Ericsson (ERIC) From chipsets, well move on to handsets. Ericsson, the Swedish telecom giant has long been a leader in mobile tech, and is well known for its infrastructure and software that make possible IP networking, broadband, cable TV, and other telecom services. Ericsson is the largest European telecom company, and the largest 2G/3G/4G infrastructure provider outside of China. But that is all in the background. Ericsson is also a leader in the rollout of Europes growing 5G networks. Ericsson is involved in 5G rollout in 17 countries in Europe, the Americas, and Asia, and its product line includes infrastructure base units and handsets, giving the company an interest in all aspects of the new 5G networks. Ericssons revenue performance in 2020 was not notably distressed by the corona crisis. Yes, the top line dipped in Q1, but that was in line with the companys historical pattern of rising revenue from Q1 through Q4. While the companys 1H20 revenues showed small yoy declines, the 2H20 gains were higher. In Q3, the $6.48 billion top line was up 8.7% yoy, and Q4s $8.08 billion revenue was up 17% from the prior year. The companys shares have also performed well during the corona year, and show a 12 month gain of 64%. Raymond James 5-star analyst Simon Leopold bluntly assigns Ericssons recent gains to its participation in 5G rollouts. Japan's awaited 5G roll-out has started. Share gains continue as Ericsson benefits from challenges facing its biggest competitors and more operators embrace 5G it seems obvious that Ericsson should be gaining market share... Competitor Nokia shunned the Chinese 5G projects, citing profitability challenges, yet Ericsson appears to be profiting in the challenging region. Leopold rates this stock an Outperform (i.e. Buy), and his $15 price target implies an upside potential of ~14% for the year ahead. (To watch Leopolds track record, click here) The Raymond James analyst, while bullish on ERIC, is actually less so than the Wall Street consensus. The stock has a Strong Buy consensus rating, based on a unanimous 5 reviews, and the $16.50 average price target indicates 25% growth potential from the share price of $13.19. (See ERIC stock analysis on TipRanks) To find good ideas for 5G stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

See more here:
Paragon Biosciences Adds CiRC Biosciences to Expand Cell and Gene Therapy Platform - Yahoo Finance

Feb. 3, 2021 12:30 UTC

ST. LOUIS--(BUSINESS WIRE)-- M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced its scientific advisory board (SAB) that will support the Companys mission of translating its innovative bicistronic-S1S3 technology platform into best-in-class therapies that address unmet needs within the LSD community. The Companys platform enables improved biodistribution of recombinant enzymes to target tissues and efficient cross-correction for gene therapies.

As we work to advance our robust pipeline, we seek the input and support of a world-class team of scientific advisors with deep expertise in genetics, rare diseases, and lysosomal storage and metabolic disorders in particular, said Pawel Krysiak, president and chief executive officer of M6P Therapeutics. The collective insights, knowledge, commitment, and expertise of our scientific advisory board will help us translate this high science into potential medical benefit for the individuals affected by these serious conditions.

By combining the substantial expertise of the SAB with the expertise of the Companys internal R&D team in recombinant enzyme and gene therapies, M6P Therapeutics is well positioned to rapidly advance its deep pipeline of LSD programs. The members of the SAB are:

M6P Therapeutics bicistronic-S1S3 technology platform enhances mannose 6-phosphate content on lysosomal enzymes for both recombinant enzyme and gene therapies, which improves enzyme uptake across target tissues, said Stuart Kornfeld, MD, M6P Therapeutics co-founder and chairman of its SAB. With promising pre-clinical data across numerous LSD programs, this innovation can potentially translate into new and more efficacious treatments, reduced immunogenicity, and more efficient dosing regimens.

About M6P Therapeutics

M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary bicistronic-S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its bicistronic-S1S3 platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: http://www.m6ptherapeutics.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210203005284/en/

Visit link:
M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board - BioSpace