Episode 11 (March 1, 2024): After being diverted by the SARS-CoV-2 pandemic to develop and manufacture COVID-19 vaccines, Moderna gets back to advancing rare disease mRNA therapeutics; a look at rare autoimmune diseases that fly below the public radar; Form Bio announced a partnership with Ginkgo Bioworks to bring their AI out of the computer and into gene therapy production by optimizing genetic payloads.

Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base:

Rare Disease Day: Moderna Gets Back to Its Roots

By Jonathan D. Grinstein, GEN, February 28, 2024

Taming the Immune System When Rare Diseases Strike

By Kathy Liszewski, GEN, February 27, 2024

Form Bio’s AI Ensures Gene Therapies Pass Muster, with Ginkgo’s Help

GEN, February 28, 2024

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Episode 10 (February 23, 2024): Researchers from Francis Collins’ lab developed an efficient CRISPR prime editing protocol to generate isogenic-induced pluripotent stem cell (iPSC) lines carrying heterozygous or homozygous alleles for putatively causal single nucleotide variants. An RNA-based system known as PRINT can create and introduce site-specific, safe, and stable transgenes into the human genome. Astellas Pharma and Kelonia announced a nearly $800 million research collaboration and license agreement to develop innovative universal, off-the-shelf in vivo CAR T cell therapies. Fourth-quarter earnings and revenue fell short of expectations as Biogen worked to eliminate 1,000 jobs (11.5% of its workforce), among other cost-cutting actions, and restructured its pipeline after a slow start.

Plus, an interview with Andrea Choe, founder and chief executive officer at Holoclara.

Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base:

Generation of Human Isogenic Induced Pluripotent Stem Cell Lines with CRISPR Prime Editing

The CRISPR Journal, February 14, 2024

Addressing Scientific Misconduct: An Interview with Elisabeth Bik

By Uduak Thomas, GEN Biotechnology, February 15, 2024

StockWatch: Biogen’s Q4 Results Disappoint Analysts

By Alex Philippidis, GEN Edge, February 18, 2024

Astellas and Kelonia Partner for $800M In Vivo CAR T Cell Therapies

By Jonathan D. Grinstein, PhD, GEN Edge, February 20, 2024

PRINT: Precise RNA-Mediated Insertion of Transgenes

GEN, February 21, 2024

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Episode 9 (February 16, 2024): Using CRISPR-Cas9 technology, British animal genetics firm Genus has generated a population of pigs that are resistant to the deadly porcine reproductive and respiratory syndrome (PRRS) virus. Exscientia has fired its founder, CEO, and principal executive officer Andrew Hopkins, PhD, and removed him from the company’s board, after it concluded that he engaged in “inappropriate” relationships with two employees. Julianna LeMieux, PhD, Deputy Editor in Chief at GEN, provides a recap of her time at AGBT 2024.

Plus, an interview with Christian Henry, president and chief executive officer at PacBio.

Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base:

CRISPRed Pigs: Precision Porcine Gene Editing Combats PPRS Virus Threat

By Uduak Thomas, GEN, February 14, 2024

Exscientia Fires CEO for “Inappropriate” Relationships with Two Employees

GEN, February 13, 2024

Sun, Spatial, and Sequencing: A Report from the First Day of AGBT

By Julianna LeMieux, PhD, and Jonathan D. Grinstein, PhD, GEN, February 7, 2024

NanoString CSO Joe Beechem Insists the Company “Not Going Anywhere”

By Julianna LeMieux, PhD, GEN, February 8, 2024

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Episode 8 (February 9th, 2024): Intellia and Ultragenyx gave updates on their clinical trials for gene therapies for inherited rare diseases. Novo Holdings, which manages the assets of the foundation that controls Novo Nordisk, agreed to buy Catalent for $16.5 billion to keep up with the high demand for its hit drugs Wegovy and Ozempic. Spatial biology tools developer NanoString Technologies filed for bankruptcy, blaming the $31 million jury award assessed against it last November in a patent infringement case filed by rival 10x Genomics. Plus, an interview with Simon Barnett, research director at Dimension.

Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base:

Gene Therapy for Hereditary Angioedema Shows Success in Patients

·        GEN, February 5, 2024

Ultragenyx’s Gene Therapy Ameliorates Pediatric Neurodegenerative Disorder 

·        By Jonathan Grinstein, GEN Edge, February 7, 2024

CRISPR-Repaired T Cells May Treat Fatal Inflammatory Diseases 

·        GEN, February 5, 2024

NanoString Files for Chapter 11 Bankruptcy, Launches Strategic Review 

·        By Alex Philippidis, GEN, February 5, 2024

Novo Holdings Buys Catalent for $16.5B; Sells Three Sites to Novo Nordisk for $11B 

·        By Alex Philippidis, GEN, February 5, 2024

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Episode 7 (February 2, 2024): Simplify Genomics launches genome interpretation platform, COUR’s nanoparticles for autoimmunity, gene replacement therapy for heart condition, CG Oncology and ArriVent IPOs; an interview with Nautilus Biotechnology co-founders, Sujal Patel and Parag Mallick.

Listed below are key references to the GEN stories, media and other items discussed in this episode of Touching Base:

Human Longevity Spin-Out Aims to Tap into Variant Interpretation Market

By Uduak Thomas, GEN, January 31st, 2024

Light My Fire: COUR Raises $105M Series A to Advance Tolerogenic Nanoparticles for Autoimmunity

By Jonathan Grinstein, GEN Edge, January 30th, 2024

Gene Replacement Therapy Halts Fatal Arrhythmogenic Condition in Mice

GEN, January 30th, 2024

StockWatch: IPO Revival? CG Oncology, ArriVent Raise a Combined $555M

By Alex Philippidis, GEN Edge, January 28th, 2024

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Episode 6 (January 26, 2024): 11-year-old regains hearing after gene therapy; Kyverna uses blood draw instead of apheresis for CAR T manufacturing; DNA production technology with GSK and Elegen, StockWatch with Nvidia and Recursion; an interview with renowned cardiologist, Eric Topol, MD

Listed below are key references to the GEN stories, media and other items discussed in this episode of Touching Base:

 Gene Therapy Restores Hearing in 11-Year-Old After Just One Month

GEN, January 24th, 2024

 GSK and Elegen Team Up to Develop DNA-Based Medicines and Vaccines

By Jonathan Grinstein, GEN Edge, January 24th, 2024

Blood Brothers: Kyverna Reinvents Autologous CAR T Manufacturing from Blood Draw

By Jonathan Grinstein, GEN Edge, January 24th, 2024

StockWatch: Nvidia Jumps 24% on Recursion, Amgen AI Partnerships 

By Alex Philippidis, GEN Edge, January 21st, 2024

Five Deaf Children Have Hearing Restored by AAV-Based Gene Therapy

GEN, January 25th, 2024

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Episode 5 (January 19, 2024); RNAs to address Alzheimer’s disease; monkey cloned via somatic cell nuclear transfer; Cytokinetics stock roller coaster; an interview with founder and CEO of 1910 Genetics, Jen Nwankwo, PhD.

Listed below are key references to the GEN stories, media and other items discussed in this episode of Touching Base:

· Alzheimer’s Disease Linked to Toxic Short RNAs

GEN, January 18, 2024.

·   Rhesus Monkey Cloned via Somatic Cell Nuclear Transfer

GEN, January 16, 2024.

·   StockWatch: M&A Talk Fuels Cytokinetics Stock Roller Coaster

By Alex Philippidis, GEN Edge, January 16th, 2024.

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Episode 4 (January 12, 2024): Takeaways from the J.P. Morgan Health Care conference: Illumina, Danaher, 10x, Empress Therapeutics; an interview with Liberate Bio’s Shawn Davis and Walter Strapps. 

Listed below are references to relevant GEN stories discussed in this episode of Touching Base: 

• JPM: Illumina’s new CEO spells out 2024 priorities, presents preliminary Q4 2023 results 

By Alex Philippidis, GEN, January 10, 2024. 

• JPM: Prime Medicine eyes 2024 IND for first prime editing therapy 

By Alex Philippidis, GEN, January 9, 2024 

• JPM 2024: Veracyte’s C2i genomics acquisition adds AI readouts to liquid biopsy testing 

By Jonathan D. Grinstein, GEN, January 8, 2024 

• JPM: More M&A “firepower” in store for 2024, EY predicts 

By Alex Philippidis, GEN, January 8, 2024 

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Episode 3 (January 5, 2024): Interview with Co-founder and CEO of Moonwalk Biosciences and former CTO of Illumina, Alex Aravanis; Neuro gene therapy with Novartis and Voyager; GEN reporters look forward to the 2024 J.P. Morgan Healthcare Conference

Listed below are key references to the GEN stories, media and other items discussed in this episode of Touching Base:

• Novartis, Voyager Ink Up-to-$1.3B Expansion of Neuro Gene Therapy Collaboration

By Alex Philippidis, GEN, January 2, 2024.

• Moonwalk Biosciences Raises $57M to Edit the Epigenome

By Uduak Thomas, GEN, January 4, 2024.

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Episode 2 (December 22, 2023): Interview with President and CEO of Tome Biosciences, Rahul Kakkar; intelligent lab partner using LLMs; AI for antimicrobials; mRNA vaccine production in Africa; Moderna’s positive cancer vaccine data.

Listed below are key references to the GEN stories, media and other items discussed in this episode of Touching Base:

• The Next Chapter in Genome Editing? Tome Emerges from Stealth with $213M By Alex Philippidis, GEN, December 19, 2023.
• Artificial Intelligence-Based Lab Partner Designs, Runs Experiments in the Lab GEN, December 20, 2023.
• BioNTech Advances Plans for mRNA Vaccine Production in Africa by 2025 GEN, December 19, 2023.
• Explainable AI Directs Small Molecule Structural Class Drug Discovery By Jonathan Grinstein, GEN, December 20, 2023.
• StockWatch: Positive Cancer Vaccine Data Wows Moderna Investors By Alex Philippidis, GEN Edge, December 18, 2023.

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