Liver

Metacrine Reports Interim Results for MET642 Phase 2a Trial in Patients with NASH and Announces a Strategic Re-Prioritization of Its Clinical Development Programs

Thursday, October 21, 2021 - 9:01pm

In this small interim analysis, the 6 mg cohort displayed a non-normal distribution in liver fat changes, as evidenced by differences between the mean and median results.

Key Points: 
  • In this small interim analysis, the 6 mg cohort displayed a non-normal distribution in liver fat changes, as evidenced by differences between the mean and median results.
  • NASH is a complex and chronic disease that we believe will likely require combination regimes to most effectively treat patients.
  • FXR therapy could potentially change the IBD treatment paradigm by bringing an oral, once-daily, well-tolerated and non-immunosuppressive medicine to patients.
  • All forward-looking statements contained in this press release speak only as of the date on which they were made.

Worldwide Roselle Industry to 2030 - Opportunity Analysis and Industry Forecasts - ResearchAndMarkets.com

Thursday, October 21, 2021 - 5:37pm

The "Roselle Market by Form, End Use and Sales Channel: Global Opportunity Analysis and Industry Forecast, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Roselle Market by Form, End Use and Sales Channel: Global Opportunity Analysis and Industry Forecast, 2021-2030" report has been added to ResearchAndMarkets.com's offering.
  • Growing product demand for soaps, shampoos & conditioners, masks, lotions, and creams is expected to drive the market for Roselle powder.
  • Moreover, health benefits and health consciousness among individuals is expected to fuel the growth of the roselle market.
  • The report provides extensive analysis of the current & emerging trends and opportunities in the Roselle market.

uniQure Announces the Appointment of Rachelle Jacques to its Board of Directors

Thursday, October 21, 2021 - 2:30pm

and AMSTERDAM, Oct. 21, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Rachelle Jacques to its Board of Directors.

Key Points: 
  • and AMSTERDAM, Oct. 21, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Rachelle Jacques to its Board of Directors.
  • "It is with great pleasure that we welcome Rachelle to the uniQure board, stated Matthew Kapusta , chief executive officer of uniQure.
  • I am pleased with this opportunity to join the uniQure Board of Directors at such an exciting time for the Company, stated Ms. Jacques.
  • Since April 2019, Ms. Jacques has served on the Board of Directors of Corbus Pharmaceuticals (NASDAQ: CRBP), and from April 2020 to February 2021, she served on the Board of Directors of Viela Bio.

Drinking Ketones Daily: 10-Day Drink Ketones Challenge Is the Quickest, Easiest Way to Experience Ketosis in Body and Mind

Thursday, October 21, 2021 - 2:00pm

Did you know that you can give your body and mind the experience of ketosis by simply drinking ketones?

Key Points: 
  • Did you know that you can give your body and mind the experience of ketosis by simply drinking ketones?
  • Bill and Erica Schultz are Independent Promoters for the Pruvit nutritional supplements and are excited to invite you to try the 10-Day Drink Ketones Challenge.
  • The challenge, available at Drinking Ketones Daily, provides you with daily Pruvit Ketones supplements that you can mix straight from a packet.
  • As long as the meals are low carb, the supplements will put your body and mind into a state of ketosis.

Silence Therapeutics Provides mRNAi GOLD™ Platform and Pipeline Updates at 2021 R&D Day

Thursday, October 21, 2021 - 1:30pm

21 October 2021, LONDON and NEW YORK -- Silence Therapeutics plc (AIM:SLN and Nasdaq: SLN), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, announces it is hosting its R&D Day in New York City today.

Key Points: 
  • Silence's proprietary mRNAi GOLD platform can be used to create siRNAs (short interfering RNAs) that precisely target and silence disease-associated genes in the liver, which represents a substantial opportunity.
  • Words such as 'anticipates,' 'expects,' 'intends,' 'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions are intended to identify forward-looking statements.
  • Silence will also provide an update on its growing discovery pipeline and plans to deliver 2-3 INDs per year from 2023.
  • Silence remains on-track to deliver 2-3 INDs per year from 2023 through both its proprietary and partnered mRNAi GOLD platform programs.

ReCode Therapeutics Raises Oversubscribed Series B Financing Round of $80 Million

Thursday, October 21, 2021 - 1:00pm

ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the closing of an $80 million Series B financing round co-led by Pfizer Ventures and EcoR1 Capital.

Key Points: 
  • ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the closing of an $80 million Series B financing round co-led by Pfizer Ventures and EcoR1 Capital.
  • Existing investors who participated included OrbiMed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures, L.P., and Osage University Partners (OUP).
  • Oleg Nodelman, founder and portfolio manager of EcoR1 Capital also joined ReCodes Board of Directors in connection with the financing.
  • ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful LNP delivery technology to target organs and tissues beyond the liver.

Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis

Thursday, October 21, 2021 - 12:30pm

This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body.

Key Points: 
  • This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body.
  • Orphan drug designation underscores the FDAs recognition of NTLA-2001s potential promise as a single-dose, novel therapy for the treatment of ATTR amyloidosis, said Intellia President and Chief Executive Officer John Leonard, M.D.
  • NTLA-2001 is currently being studied in a Phase 1 trial in adults with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).
  • The decision by the FDA follows a March 2021 decision by the European Commission (EC) to also grant NTLA-2001 orphan drug designation for the treatment of ATTR amyloidosis.

Homology Medicines Announces Presentations Across Gene Therapy and Gene Editing Programs, including GTx-mAb, at European Society of Gene & Cell Therapy Meeting

Thursday, October 21, 2021 - 12:00pm

BEDFORD, Mass., Oct. 21, 2021 (GLOBE NEWSWIRE) --  Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today four presentations of preclinical data spanning its clinical-stage gene therapy program for mucopolysaccharidosis type II (MPS II, or Hunter syndrome), clinical-stage gene editing program for phenylketonuria (PKU), GTx-mAb program for paroxysmal nocturnal hemoglobinuria (PNH) and assays to evaluate levels of pre-existing antibodies to the Company’s adeno-associated viral vectors (AAVHSCs) during the 2021 European Society for Gene & Cell Therapy Virtual Conference (ESGCT).

Key Points: 
  • gene therapy candidate for MPS II, and showcased gene editing data in the PKU and humanized murine models, including on- and off-target assessment confirming the precision of our nuclease-free in vivo gene editing.
  • Encouraging data from our GTx-mAb platform showed a single dose resulted in sustained and robust expression of full-length antibodies from the liver consistent with anti-C5 therapeutics.
  • Further, we presented details of the methods of our neutralizing antibody assays used in our clinical trial screening.
  • Both methods are used in the screening phase of Homologys HMI-102 gene therapy pheNIX clinical trial to determine patient eligibility based on pre-existing Nabs.

Verve Therapeutics to Participate in the Jefferies Gene Therapy/Editing Summit

Thursday, October 21, 2021 - 11:30am

A live webcast will be available in the investor section of the company's website at www.vervetx.com .

Key Points: 
  • A live webcast will be available in the investor section of the company's website at www.vervetx.com .
  • The webcast will be archived for 60 days following the presentation.
  • Verve Therapeutics, Inc. (Nasdaq: VERV) is a genetic medicines company pioneering a new approach to the care of cardiovascular disease, transforming treatment from chronic management to single-course gene editing medicines.
  • VERVE-101, currently in IND-enabling studies, is being developed initially for the treatment of patients with heterozygous familial hypercholesterolemia, a potentially fatal genetic heart disease.

Positive Data from Phase IIa Can-Fite NASH Study Published in Leading Peer Reviewed Scientific Journal

Thursday, October 21, 2021 - 12:00pm
Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20211021005526/en/
    Positive Data from Phase IIa Can-Fite NASH Study Published in Leading Peer Reviewed Scientific Journal (Photo: Business Wire)
    The article includes highlights from Can-Fites Phase IIa NASH study of Namodenoson which achieved its study endpoints including significant anti-steatotic, anti-fibrotic, and anti-inflammatory effects.
  • Namodenoson is advancing into a Phase IIb NASH trial which is expected to commence patient enrollment in Q4 2021.
  • Principal Investigator of the Phase IIa study, Prof. Rifaat Safadi, commented, The acceptance and publication of the article presenting our Phase IIa results in this prestigious journal demonstrates the high value of the data and the potential of Namodenoson as safe and effective treatment for NASH.
  • As of 2016, NASH was the leading cause for liver transplants among women and the second leading cause for liver transplants overall.