RA Capital Management is an investment advisor based in Boston specializing in the life-sciences and drug development sectors. The company's team has been investing since 2002 and is comprised of professionals with training in biology, chemistry, and medicine and also has industry and business development experience at the executive and board levels. The company invests in companies with promising technologies and products.

RA Capital Management

SNBL USA provides general toxicology, nucleic acid therapeutics and laboratory services.

SNBL USA

Foresite Capital is a healthcare and life sciences investment firm. They take a data-driven approach to invest in startups that employ biology and big data to improve healthcare, such as 10x Genomics, Relay Therapeutics, Element Biosciences, and Inscripta.

Foresite Capital

Fidelity is a privately owned investment management firm that serves a wide range of clients. It offers services including IRAs, rollovers, annuities, discount brokerage, retirement planning, estate planning, wealth management, securities execution and clearance, and life insurance. They serve individual investors, businesses, financial advisors, and institutions. The firm manages equity, fixed-income, and balanced mutual funds and invests in public equity and fixed-income markets. It also conducts in-house research to inform its investment decisions.

Fidelity

New Leaf Venture Partners is a venture capital firm that invests primarily in healthcare technology. The company typically focuses on later stage biopharmaceutical products, early stage medical devices, and laboratory infrastructure technologies.  The New Leaf Ventures (NLV) team has been built over a decade, originating within Sprout Group, the venture capital affiliate of Credit Suisse First Boston. Sprout Group was formed in 1969 and has historically been one of the leading venture capital firms in the country. The team started to invest in healthcare technology in 1993 and since then, has become a leading venture investor in that sector. In 2005, the entire healthcare technology team of Sprout spun out into NLV.

New Leaf Venture Partners

Jennison Associates has earned a reputation for excellence by fulfilling client investment needs since its founding in 1969. Today, the firm manages  over $93 billion for institutions and private clients through separately managed accounts and commingled vehicles, including mutual funds through subadvisory relationships.

Jennison Associates

Redmile Group is a healthcare investment organization that makes venture, growth, and crossover investing across healthcare.

Redmile Group

Cormorant Asset Management is an employee-owned hedge fund sponsor that provides its services to pooled investment vehicles. The firm also focuses on both public and private market innovative companies in the biotechnology and life sciences marketplace.

Cormorant Asset Management

Wave Life Sciences

Wave Life Sciences (Nasdaq: WVE) is a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.

<div>Wave Life Sciences is a biotechnology company focused on developing oligonucleotide therapies for genetic targets. The company's key technology is the PRISM oligonucleotide platform, which is used for the discovery and development of novel treatments. Wave's pipeline includes WVE-006, a preclinical RNA editing therapy for alpha-1 antitrypsin deficiency (AATD), which has the potential to address both liver and lung manifestations of the disease.</div>

GSK and Wave Life Sciences to develop oligonucleotide therapeutics

RNA Therapeutics

Wave Life Sciences to Present at Upcoming Investor Conferences

GlaxoSmithKline

Collaboration brings together Wave’s PRISMTM oligonucleotide platform and GSK’s expertise in genetics and genomics

Reinventing gene therapies with next-generation technologies

Human Gene Editing

Companies that develop novel therapeutics based on other types of gene editing technologies including TALEN and ZFN

Takeda

To develop antisense oligonucleotides for genetically-defined neurological diseases.

<div>
 
 Wave Life Sciences is a biotechnology company focused on developing oligonucleotide therapies for genetic targets. The company's key technology is the PRISM oligonucleotide platform, which is used for the discovery and development of novel treatments. Wave's pipeline includes WVE-006, a preclinical RNA editing therapy for alpha-1 antitrypsin deficiency (AATD), which has the potential to address both liver and lung manifestations of the disease.
 
 
 
 
 In December 2022, Wave Life Sciences entered into a strategic partnership with GSK to advance the development of oligonucleotide therapies. This collaboration combines Wave's PRISM platform with GSK's expertise in genetics and genomics. The initial research period of the alliance is set for four years, during which GSK will progress up to eight programs, while Wave will develop up to three programs. As part of this agreement, GSK acquired the exclusive global license for WVE-006. Wave Life Sciences will conduct all preclinical research for the programs of both companies up to the investigational new drug (IND)-enabling studies, after which GSK will take over the development and commercialization of its partnered programs.
 
 
 
</div>

<div>
 
 PYC Therapeutics is a clinical-stage biotechnology company focused on developing RNA therapies for genetic diseases. The company utilizes a proprietary drug delivery platform based on Cell-Penetrating Peptides (CPPs) to enhance the potency of precision medicines within the RNA therapeutic class. PYC's technology aims to overcome the delivery challenges associated with traditional RNA therapeutics, allowing for more effective treatment of genetic ailments.
 
 
 
 
 PYC's lead drug candidate, VP-001, is designed to treat Retinitis Pigmentosa type 11 (RP11), a blinding eye disease that begins in childhood. VP-001 targets the U4/U6 small nuclear ribonucleoprotein (PRPF31) gene and is administered through intravitreal injection. In August 2023, the US Food and Drug Administration (FDA) granted Fast Track designation to VP-001, potentially expediting its development and review process. In May 2024, PYC reported encouraging first clinical data for VP-001 in RP11 patients.
 
 
 
 
 The company's pipeline includes other preclinical programs for Inherited Retinal Diseases and Central Nervous System disorders. In May 2024, PYC received orphan drug designation from the FDA for PYC-001, a drug candidate targeting autosomal dominant optic atrophy (ADOA). PYC is also developing VP-002 and exploring multiple candidates for eye and CNS indications.
 
 
 
 
 In January 2024, PYC announced plans to collaborate with Google Cloud to develop new precision medicines. This project aims to utilize machine learning models, merging AlphaFold and its successors, to predict ideal peptide sequences and structures for delivering precision medicine cargoes to cells in the human body.
 
 
 
 
 For the half-year ended December 31, 2023, PYC reported revenue of AUD 9.12 million, up from AUD 3.49 million in the previous year. The company's net loss for the period was AUD 15.09 million. As of March 31, 2024, PYC had a pro-forma cash balance of approximately AUD 84 million, providing runway into the second half of 2025 for its ongoing clinical trials.
 
</div>

PYC Therapeutics

Flamingo Therapeutics is a biotechnology company specializing in RNA-targeted therapies for oncology. Founded in 2019, the company focuses on developing treatments for difficult-to-treat cancers using state-of-the-art chemistries and a clinical-stage pipeline targeting undruggable transcription factors and splice variants. Flamingo's lead program is danvatirsen, an antisense oligonucleotide (ASO) that selectively targets STAT3 and has shown clinical activity in two Phase II studies. Danvatirsen binds to STAT3 mRNA, inhibiting translation of the transcript, resulting in reduced tumor cell growth and activation of anti-tumor immunity. Flamingo initiated the PEMDA-HN Phase II trial evaluating danvatirsen in combination with pembrolizumab for patients with recurrent/metastatic head and neck squamous cell carcinoma (HNSCC). The company is also advancing FTX-001, its most advanced long non-coding RNA (LncRNA) program targeting MALAT-1, through readiness for Phase 1 trials in solid tumors. Flamingo's proprietary discovery engine, FLAME (Flamingo LncRNA Antisense Mining Engine), addresses lncRNAs, a large and untapped class of disease-causing targets within the "dark matter" of the human genome. In March 2023, Flamingo merged with Dynacure to create a leading RNA therapeutics company focused on clinical oncology, combining their pipelines and expertise.
Key customers and partnerships
Flamingo Therapeutics has established strategic alliances and partnerships to advance its RNA-targeted therapies. The company has an expanded alliance with Ionis Pharmaceuticals, a world leader in RNA-targeted antisense oligonucleotide (ASO) therapeutics. This partnership, announced in September 2021, combines three clinical-stage programs from Ionis with Flamingo's novel lncRNA discovery engine and preclinical lncRNA program. The alliance leverages Flamingo's expertise in oncology and long non-coding RNAs with Ionis' proficiency in RNA-targeted drug discovery and development. Additionally, Flamingo collaborates with academic partners at VIB, KU Leuven, Ghent University, and the University of Michigan, whose pioneering work in non-coding RNA genes in cancer contributed to the company's foundation. In June 2023, Flamingo received a EUR 1.7 million research grant from Flanders Innovation & Entrepreneurship (VLAIO) to advance its RNA-targeting oncology portfolio, further validating its approach in creating a pipeline of programs targeting undruggable transcription factors and non-coding RNA.

Flamingo Therapeutics

Korro Bio is a biotechnology company focused on developing RNA editing therapeutics for both rare and common diseases. Founded in 2018, the company is headquartered in Cambridge, Massachusetts. Korro's proprietary platform, called OPERA (Oligonucleotide Promoted Editing of RNA), leverages the body's natural ADAR (adenosine deaminase acting on RNA) system to make precise edits to RNA transcripts. This approach allows for the correction of genetic mutations without permanently altering DNA, potentially offering a safer alternative to traditional gene editing techniques.
The company's lead program targets alpha-1 antitrypsin deficiency (AATD), a genetic disorder affecting the lungs and liver. Korro's AATD candidate aims to repair inherited mutations and restore production of normal functional alpha-1 antitrypsin (A1AT) protein in the liver. preclinical studies have demonstrated an increase of normal A1AT protein to 85% of total protein in circulation. Korro plans to submit a regulatory filing to initiate clinical trials for this candidate in the second half of 2024.
Beyond AATD, Korro is developing RNA editing therapies for conditions such as Parkinson's disease, severe alcoholic hepatitis, amyotrophic lateral sclerosis (ALS), subsets of pain, and cardiometabolic diseases. The company's initial focus is on liver-targeted therapies, with plans to expand into central nervous system indications and other tissue types.
In July 2023, Korro Bio announced plans to go public through a reverse merger with Frequency Therapeutics. The combined company will operate under the Korro Bio name and trade on the Nasdaq stock exchange under the ticker symbol KRRO. This transaction, along with a concurrent USD 117 million financing, is expected to provide Korro with approximately USD 170 million in cash upon closing, which is anticipated to fund operations into 2026.

Korro Bio

Halo-Bio is a biopharmaceutical company focused on developing innovative therapies. The company has a licensing agreement with Tekmira Pharmaceuticals related to certain lipid nanoparticle (LNP) technologies.

Halo-Bio RNAi Therapeutics

Oncotelic Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing RNA therapeutics and small molecule drugs for cancer and infectious diseases. The company's lead drug candidate is OT-101, a first-in-class RNA therapeutic designed to target TGF-β2, a protein that suppresses immune cell response and contributes to tumor growth in cancer tissues. OT-101 has demonstrated notable single-agent activity in clinical trials for relapsed/refractory cancer patients, with overall survival increasing in a dose-dependent manner at levels of 140mg/m2 and higher.
Oncotelic is currently conducting a Phase 2 billion/Phase 3 clinical trial called STOP-PC to evaluate OT-101 in combination with mFOLFIRINOX compared to mFOLFIRINOX alone for patients with advanced and unresectable or metastatic pancreatic cancer. The company believes this trial could deliver a decisive win against pancreatic cancer based on previous data showing improved survival in patients with low TGF-β2 expression treated with irinotecan.
In addition to its oncology pipeline, Oncotelic has explored OT-101's potential against SARS-CoV-2, completing a Phase 2 trial for Covid-19 in November 2021 with positive top line data for safety and efficacy. The company has also acquired AL-101 for intranasal delivery of apomorphine, which it intends to develop for conditions including Parkinson's Disease, erectile dysfunction, and female sexual disorders.

Oncotelic

Companies developing treatments using molecules like antisense oligonucleotides (ASOs) and phosphorodiamidate morpholino oligomers (PMOs). These molecules target specific genes to treat genetic diseases by either silencing or modifying gene expression.

Companies using RNA interference (RNAi) to target and silence specific genes involved in diseases for treating various conditions by blocking harmful gene activity.

Companies involved in altering RNA molecules to modify gene expression or correct genetic defects. This can include substitutions, insertions, or deletions of nucleotides, leading to alterations in the RNA sequence and the regulation of RNA molecules to control gene expression or cellular functions.

Companies focused on other RNA-based therapies using approaches such as tRNA, circRNA, lncRNA, and saRNA to develop treatments targeting specific RNA molecules to regulate gene expression.

RNA-based developments form a foundation for precision medicine.

RNA plays essential roles in gene expression, regulation, and protein synthesis, making it a fundamental focus in genomic research and understanding genetic mechanisms.

Wave Life Sciences

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