STAT News

SEPTEMBER 12, 2022

In the life sciences industry, where company names derived from “biology” or “genetics” are a dime a dozen, it helps to have a brand that stands out. The company is developing treatments for diseases caused by broken mitochondria — the main energy source for human cells.

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World of DTC Marketing

JANUARY 24, 2022

Venture capitalists have poured $42 billion into drug development over the past three years. Most small biotech companies rely on venture capitalist funding to develop new drugs but is that a good way to go? billion, which was much lower than previous studies, which have placed the average cost of drug development as $2.8

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Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).

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XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. “They called him ‘The Boy.’

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XTalks

MAY 17, 2023

The Bespoke Gene Therapy Consortium was jointly launched by the NIH and US Food and Drug Administration (FDA) in 2021 to help address common issues that gene therapy developers face. It included formulating a standardized therapeutic development model that would help accelerate clinical trial processes for new treatments.

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XTalks

APRIL 15, 2021

Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. The reverse could also be true whereby the development of coronary artery disease may impact heart rate. HFrEF vs. HFpEF.

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XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases. ” or “Why is my child different?”

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Pharmaceutical Technology

MAY 8, 2023

ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models. According to GlobalData, there are 60+ companies, spanning technology vendors, established pharmaceutical companies, and up-and-coming start-ups engaged in the development and application of zinc-finger nucleases.

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XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

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XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company. Dr. Marinkovich said Vyjuvek “changes all of this” by healing both wounds and preventing skin from re-blistering.

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XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). He added that, “A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe genetic diseases including MLD.

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pharmaphorum

OCTOBER 16, 2020

With large datasets crucial to gaining an understanding of rare diseases and opening the door to drug development, digital technology is proving transformative. Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise.

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XTalks

SEPTEMBER 24, 2024

CDKL5 deficiency disorder (CDD) is a rare genetic condition caused by mutations in the CDKL5 gene, which is essential for brain development and function. With no cure currently available, managing symptoms and improving the quality of life for patients remain critical goals for medical research and treatment.

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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

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The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost.

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XTalks

NOVEMBER 4, 2024

Arrowhead Pharmaceuticals focuses on developing RNAi therapeutics targeting the genetic cause of challenging diseases like FCS. Julie’s presence in our offices reminds us of why we do what we do every day and reaffirms one of Arrowhead’s core values: patients — making people’s lives better.”

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XTalks

AUGUST 29, 2022

Alex Marson, MD, PhD, and senior author of the study states that the research team has developed a method to insert lengthy DNA sequences into a target site in the genome without the use of viral vectors. The results demonstrate potential as a novel, safe and effective approach to cell therapies.

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pharmaphorum

JANUARY 27, 2023

Late last year, pharmaphorum caught up with Dr Karen Mullen, chief medical officer and VP of clinical & medical affairs at global drug development consultancy Boyds. We talk a lot in clinical trials and drug development about benefit,” Dr Mullen said. I still feel that it is very important,” Dr Mullen said. “We’ve

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XTalks

SEPTEMBER 25, 2024

Aqneursa’s efficacy was confirmed in a randomized, double-blind, placebo-controlled, two-period crossover trial involving 60 patients aged four years or older.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic.

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pharmaphorum

DECEMBER 8, 2021

If approved, the drug will be sold by Neuren Pharma, which licensed North American rights to the drug in 2018 and is also developing another drug for Rett blarcamesine, in collaboration with Anavex Life Sciences. Results from a phase 3 trial of blarcamesine are due next year.

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Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

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XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes.

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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR.

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XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Rett syndrome is characterized by a distinctive pattern of symptoms that gradually emerge after a period of seemingly normal development. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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XTalks

NOVEMBER 10, 2023

Acute manifestations include thrombocytopenia, microangiopathic hemolytic anemia, bleeding episodes, headache and abdominal pain, while chronic ones include stroke, cardiovascular disease and damage to organs. As a rare disease, cTTP affects less than 1,000 individuals in the US.

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XTalks

AUGUST 6, 2020

Epidiolex is a plant-derived CBD solution developed by GW Pharmaceuticals. TSC is a rare genetic disease that affects approximately 1 in 6,000 people. Symptoms can range from being mild to more severe or life-threatening. Due to this, the disease can be misdiagnosed or not even recognized for a while.

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XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

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The Pharma Data

OCTOBER 15, 2020

The award honors McHutchison’s work in developing life-changing and curative therapies for patients with the hepatitis C virus. The committee is tasked with supporting BeyondSpring’s business development activities related to its lead asset, Plinabulin, and other pipeline assets. BeyondSpring – BeyondSpring Inc.

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XTalks

AUGUST 16, 2021

Immune systems can be weakened in individuals with HIV/AIDS; cancer and transplant patients taking certain immunosuppressive drugs; and those with genetic diseases affecting the immune system like congenital agammaglobulinemia and congenital IgA deficiency. According to the CDC, approximately 2.7

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XTalks

SEPTEMBER 12, 2022

The FDA also awarded the sponsor (Genzyme, acquired by Sanofi in 2011) a rare pediatric disease priority review voucher, an incentive to encourage development of new drugs and biologics for the prevention and treatment of rare diseases in children, according to the FDA.

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XTalks

MARCH 21, 2024

XTALKS WEBINAR: Cell and Gene Therapy Development: Characterization of Cellular Source Material Live and On-Demand: Tuesday, May 14, 2024, at 11am EDT (4pm BST/UK) Register for this free webinar to learn about characterizing cellular source material for developing and manufacturing cell and gene therapy-based products.

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XTalks

OCTOBER 1, 2020

And although academia and industry have often been pitted against each other in the realm of scientific discovery and development, there may be more similarities and synergies between the two than we may think. WeiQi Lin, MD, PhD, executive vice president of research & development and principal scientist, DURECT Corporation.

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XTalks

SEPTEMBER 17, 2020

While the table lists acceptable surrogate endpoints for trials of more common conditions, such as asthma, type 2 diabetes and various cancer types, the document also provides key information to developers of certain rare disease therapies. Paget’s Disease. Prevalence: 1 in 7,000 to 1 in 10,000.

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XTalks

OCTOBER 8, 2024

Efforts to improve SMA detection and treatment are gaining momentum. In August 2024, Novartis announced the implementation of nationwide newborn screening programs across Canada, enabling SMA detection shortly after birth.

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